CLINSQUARE: CLINICAL RESEARCH COURSES
Sunday, December 22, 2024
CLINSQUARE: Clinical Rsearch Courses by LIFEPRONOW
Home Blog Page 4

FDA Approves First Over-the-Counter Naloxone Nasal Spray

admin
-
0
FDA Approves First Over-the-Counter Naloxone Nasal Spray

March 29, 2023: “The U.S. FDA approved Narcan, 4 milligram (mg) naloxone hydrochloride nasal spray for over-the-counter (OTC), nonprescription, use – the first naloxone product approved for use without a prescription.

Naloxone is a medication that rapidly reverses the effects of opioid overdose and is the standard treatment for opioid overdose.

Today’s action paves the way for the life-saving medication to reverse an opioid overdose to be sold directly to consumers in places like drug stores, convenience stores, grocery stores and gas stations, as well as online. 

The timeline for availability and price of this OTC product is determined by the manufacturer.

The FDA will work with all stakeholders to help facilitate the continued availability of naloxone nasal spray products during the time needed to implement the Narcan switch from prescription to OTC status, which may take months. Other formulations and dosages of naloxone will remain available by prescription only. 

Drug overdose persists as a major public health issue in the United States, with more than 101,750 reported fatal overdoses occurring in the 12-month period ending in October 2022, primarily driven by synthetic opioids like illicit fentanyl. 

“The FDA remains committed to addressing the evolving complexities of the overdose crisis.

As part of this work, the agency has used its regulatory authority to facilitate greater access to naloxone by encouraging the development of and approving an over-the-counter naloxone product to address the dire public health need,” said FDA Commissioner Robert M. Califf, M.D.

“Today’s approval of OTC naloxone nasal spray will help improve access to naloxone, increase the number of locations where it’s available and help reduce opioid overdose deaths throughout the country.

We encourage the manufacturer to make accessibility to the product a priority by making it available as soon as possible and at an affordable price.”   

Narcan nasal spray was first approved by the FDA in 2015 as a prescription drug.

In accordance with a process to change the status of a drug from prescription to nonprescription, the manufacturer provided data demonstrating that the drug is safe and effective for use as directed in its proposed labeling.

The manufacturer also showed that consumers can understand how to use the drug safely and effectively without the supervision of a healthcare professional.

The application to approve Narcan nasal spray for OTC use was granted priority review status and was the subject of an advisory committee meeting in February 2023, where committee members voted unanimously to recommend it be approved for marketing without a prescription. 

The approval of OTC Narcan nasal spray will require a change in the labeling for the currently approved 4 mg generic naloxone nasal spray products that rely on Narcan as their reference listed drug product.

Manufacturers of these products will be required to submit a supplement to their applications to effectively switch their products to OTC status.

The approval may also affect the status of other brand-name naloxone nasal spray products of 4 mg or less, but determinations will be made on a case-by-case basis and the FDA may contact other firms as needed. 

The use of Narcan nasal spray in individuals who are opioid dependent may result in severe opioid withdrawal characterized by body aches, diarrhea, increased heart rate (tachycardia), fever, runny nose, sneezing, goose bumps, sweating, yawning, nausea or vomiting, nervousness, restlessness or irritability, shivering or trembling, abdominal cramps, weakness and increased blood pressure.

“Naloxone is a critical tool in addressing opioid overdoses and today’s approval underscores the extensive efforts the agency has undertaken to combat the overdose crisis,” said Patrizia Cavazzoni, M.D., director of the FDA’s Center for Drug Evaluation and Research.

“The FDA is working with our federal partners to help ensure continued access to all forms of naloxone during the transition of this product from prescription status to nonprescription/OTC status.

Further, we will work with any sponsor seeking to market a nonprescription naloxone product, including through an Rx to OTC switch, and encourage manufacturers to contact the agency as early as possible to initiate discussions.”

The FDA has taken a series of measures to help facilitate access to naloxone products.

In November 2022, the agency announced its preliminary assessment that certain naloxone products, such as the one ultimately approved today, have the potential to be safe and effective for over-the-counter use and encouraged sponsors to submit applications for approval of OTC naloxone products.

The agency previously announced in 2019 that it had designed, tested, and validated a model naloxone Drug Facts Label (DFL) with easy-to-understand pictograms on how to use the drug to encourage manufacturers to pursue approval of OTC naloxone products.

The model DFL was used to support the approved application along with the results of a simulated use Human Factors validation study designed to assess whether all the components of the product with which a user would interact could be used safely and effectively as intended.

Through the

 FDA Overdose Prevention Framework, the agency remains focused on responding to all facets of substance use, misuse, substance use disorders, overdose and death in the U.S. The framework’s priorities include: supporting primary prevention by eliminating unnecessary initial prescription drug exposure and inappropriate prolonged prescribing; encouraging harm reduction through innovation and education; advancing development of evidence-based treatments for substance use disorders; and protecting the public from unapproved, diverted or counterfeit drugs presenting overdose risks.
 
The FDA granted the OTC approval of Narcan to Emergent BioSolutions.”

https://www.fda.gov/news-events/press-announcements/fda-approves-first-over-counter-naloxone-nasal-spray

License agreement with KYM Biosciences for CMG901, a Claudin-18.2 antibody drug conjugate, completed

admin
-
0
License agreement with KYM Biosciences for CMG901, a Claudin-18.2 antibody drug conjugate, completed

March 30, 2023: “AstraZeneca has completed an exclusive global license agreement with KYM Biosciences Inc.i for CMG901, a potential first-in-class antibody drug conjugate (ADC) targeting Claudin 18.2, a promising therapeutic target in gastric cancers.

CMG901 is currently being evaluated in a Phase I trial for the treatment of Claudin 18.2-positive solid tumours, including gastric cancer. Preliminary results from the trial have shown an encouraging profile for CMG901, with early signs of anti-tumour activity across the dose levels tested.

Financial considerations
AstraZeneca has entered into an exclusive global license for the research, development, manufacture and commercialisation of CMG901 for an upfront payment of $63m, with potential development and sales-related milestone payments of up to $1.1bn and tiered royalties up to low double digits.

The transaction does not impact AstraZeneca’s financial guidance for 2023.

i. KYM Biosciences is a joint venture established by affiliates of Keymed Biosciences and Lepu Biopharma.

Notes

CMG901
CMG901 is a novel antibody drug conjugate targeting Claudin 18.2, and consists of an anti-Claudin 18.2 monoclonal antibody, a protease-degradable linker, and a cytotoxic small molecule monomethyl auristatin E (MMAE).

CMG901 is being developed for the treatment of solid tumours that express the cell surface protein Claudin 18.2, including gastric cancers.

CMG901 is owned by KYM Biosciences Inc. (KYM), a joint venture established by affiliates of Keymed Biosciences (70% of KYM ownership) and Lepu Biopharma (30% of KYM ownership).

AstraZeneca in gastrointestinal cancers
AstraZeneca has a broad development programme for the treatment of gastrointestinal (GI) cancers across several medicines and a variety of tumour types and stages of disease.

In 2020, GI cancers collectively represented approximately 5.1 million new cancer cases leading to approximately 3.6 million deaths.1

Within this programme, the Company is committed to improving outcomes in gastric, liver, biliary tract, oesophageal, pancreatic and colorectal cancers.

Imfinzi (durvalumab) is approved in the US in combination with chemotherapy (gemcitabine plus cisplatin) for advanced biliary tract cancer and in combination with Imjudo in unresectable hepatocellular carcinoma. 

Imfinzi is being assessed in combinations, including with Imjudo in liver, oesophageal and gastric cancers in an extensive development programme spanning early to late-stage disease across settings.

Enhertu (trastuzumab deruxtecan), a HER2-directed antibody drug conjugate, is approved in HER2-positive advanced gastric cancer and is being assessed in colorectal cancer. Enhertu is jointly developed and commercialised by AstraZeneca and Daiichi Sankyo.”

https://www.astrazeneca.com/media-centre/press-releases/2023/astrazeneca-completes-agreement-with-kym-for-cmg901.html

IOnctura Awarded UK’s MHRA Innovation Passport For Entry ILAP

admin
-
0
IOnctura Awarded UK’s MHRA Innovation Passport For Entry ILAP

MARCH 28, 2023: “iOnctura, a clinical stage biotechnology company developing breakthrough therapies for patients suffering with cancer announces that the innovative medicine designation, the Innovation Passport, has been awarded for roginolisib, for the treatment of metastatic uveal melanoma by the Medicines & Healthcare products Regulatory Agency (MHRA).

The Innovation Passport is the entry point to the Innovative Licensing and Access Pathway (ILAP) which aims to accelerate time to market and thereby patient access to novel treatments in the UK.

Reserved for innovative therapies for life-threatening or seriously debilitating conditions, ILAP provides applicants with a toolkit to support all stages of the design, development, and approval process.

Roginolisib is a first-in-class, non-ATP-competitive, allosteric modulator of PI3Kd which prevents tumor proliferation and breaks immune tolerance in patients with solid and hematological tumors.

Catherine Pickering, Chief Executive Officer of iOnctura, said: “The Innovation Passport is an exciting step in the clinical development programme for roginolisib, a drug with a game-changing clinical safety and activity profile.

Being awarded this passport will allow us to work closely with the MHRA and its partner agencies to chart out a roadmap for regulatory and key development milestones with the primary goal of achieving early patient access to roginolisib.”

PI3Kδ inhibition in solid tumors has recently emerged as a novel approach to treating cancer because of its potential in targeting multiple tumor survival pathways.

First-generation PI3Kδ inhibitors are used to treat hematological tumors, but safety concerns and limited target selectivity have curbed their clinical usefulness. These concerns are even more aggravated in patients with solid malignancies where rapid onset of toxicities have been observed.

In contrast, roginolisib has a favorable toxicity profile with less than 5% Grade 3/4 toxicities at the biologically effective dose in clinical studies. Importantly, these toxicities have to-date been transient in nature without the need for dose reductions.

Clinical activity, including partial and complete responses, are being seen in patients with both solid and hematologic malignancies.

Further details on clinical responses will be released at a future international clinical conference in 2023.

Fourteen of 43 patients (including 12 of 28 uveal melanoma patients) are still on treatment, with two patients having been on treatment for more than two years.

The one-year OS rate is currently 70%; median OS has not been reached.

A research paper recently published in Cancer Research Communications highlights that roginolisib has immune-modulatory properties that can be exploited in solid tumors and further reinforced the conclusion that roginolisib inhibits regulatory T cell proliferation while having limited anti-proliferative effects on conventional CD4+ T cells and no effect on CD8+ T cells; both immune cell types key to a robust immune response to tumors.”

https://www.ionctura.com/news/

EnteroBiotix Initiates Phase 2 study of microbiome drug EBX-102 in liver cirrhosis and hepatic encephalopathy

admin
-
0
EnteroBiotix Initiates Phase 2 study of microbiome drug EBX-102 in liver cirrhosis and hepatic encephalopathy

March 28, 2022: “EnteroBiotix Limited (“EBX”), a clinical stage biotechnology company focussed on developing best-in-class full-spectrum microbiome therapeutics announced the initiation of a multi-centre randomised double-blind placebo controlled Phase 2 clinical trial.

The trial has been designed to evaluate EBX-102, the company’s lead product candidate, for the treatment of liver cirrhosis and hepatic encephalopathy (“HE”).

EBX-102 is presented in the form of an orally administered capsule that contains diverse full-spectrum microbial ecosystems that are intended to restore microbial ecology and act in concert to target multiple key disease pathways.

The Phase II study, named ‘IMPuLCE’ (Intestinal Microbiota Product in Liver Cirrhosis and Encephalopathy) intends to enrol 56 patients with liver cirrhosis across sites in the United Kingdom. The trial evaluates EBX-102 in patients with cirrhosis and HE being treated with standard of care medications, including Rifaximin.”

https://www.enterobiotix.com/news/phase-2-study-of-microbiome-drug-ebx-102

BMS Sotyktu approves for the Treatment of Moderate-to-Severe Plaque Psoriasis

admin
-
0
BMS Sotyktu approves for the Treatment of Moderate-to-Severe Plaque Psoriasis

March 28, 2023: ” Bristol Myers Squibb announced that the European Commission has approved Sotyktu (deucravacitinib), a first-in-class, selective tyrosine kinase 2 (TYK2) inhibitor, for the treatment of adults with moderate-to-severe plaque psoriasis who are candidates for systemic therapy, representing a new way of treating this chronic immune-mediated disease.

The approval was based on results from the Phase 3 POETYK PSO-1 and POETYK PSO-2 clinical trials, which demonstrated superior efficacy of once-daily Sotyktu compared to placebo and twice-daily Otezla® (apremilast) at both 16 and 24 weeks, with responses maintained through 52 weeks.

Additional data from the POETYK PSO long-term extension trial (LTE) also supported approval.

The POETYK study program demonstrated a consistent safety profile in patients through three years of continuous treatment.

“Today’s approval is a landmark achievement as patients across Europe with moderate-to-severe plaque psoriasis will now have the opportunity to be treated with Sotyktu, the first once-daily oral option to provide significant symptom relief,” said Samit Hirawat, MD, chief medical officer, Bristol Myers Squibb.

“Discovered in our own labs, Sotyktu has a unique mechanism of action and a well-demonstrated safety, efficacy and tolerability profile, representing a potential new oral standard of care, and demonstrating our ability to develop breakthrough, first-in-class treatments with the potential to transform people’s lives.”

Psoriasis is a widely prevalent, chronic, systemic immune-mediated disease that impacts approximately 14 million people in Europe. Up to 90% of patients with psoriasis have psoriasis vulgaris, or plaque psoriasis, which is characterized by distinct round or oval plaques typically covered by silvery-white scales.

Nearly one-quarter of people with psoriasis have cases that are considered moderate to severe.

Current therapies include topicals, orals and biologics. Patients may prefer oral therapies, and many patients and physicians are looking for additional efficacious and tolerable options.

“The approval of Sotyktu is groundbreaking for the psoriasis community because we now have a tolerable, highly efficacious, once-daily oral treatment option that does not require lab monitoring,” said Diamant Thaçi, MD, PhD, director and full professor, Institute and Comprehensive Center for Inflammation Medicine, University Lübeck, Germany.

“The Phase 3 POETYK-PSO clinical trials showed that Sotyktu demonstrated significant, durable efficacy across multiple key endpoints, including skin clearance and symptom burden.

The results are particularly meaningful for dermatologists and patients who have been waiting for a more effective and convenient oral therapy to help manage this serious, chronic, immune-mediated disease.”

The most commonly reported adverse reaction was upper respiratory infections (18.9%), most frequently nasopharyngitis. The incidence of serious infection in the Sotyktu group was 0.6% compared to 0.5% in the placebo group.

The majority of infections were non-serious and mild to moderate in severity and did not lead to the discontinuation of Sotyktu. Infections occurred in 29.1% of patients in the Sotyktu group compared to 21.5% in the placebo group during the first 16 weeks.

The rate of infections and serious infections in the Sotyktu group did not increase through Week 52. The longer-term safety profile of Sotyktu was similar and consistent with previous experience.

“Psoriasis can greatly impact one’s life physically, emotionally and mentally, with symptoms, such as visible plaques and itching, and feelings of self-stigma and isolation,” said Frida Dunger Johnsson, Executive Director, IFPA (International Federation of Psoriasis Associations).

“There has been an acute need for treatment options offering higher levels of efficacy for people living with moderate-to-severe psoriasis, many of whom remain untreated, undertreated or dissatisfied with current medicines.

We are thrilled that we now have the first once-daily oral therapy to help people with plaque psoriasis and physicians as they work together toward the goal of relieving symptoms and improving their disease.”

Bristol Myers Squibb thanks the patients and investigators involved in the POETYK PSO clinical trial program.

About the POETYK PSO Clinical Trial Program

PrOgram to Evaluate the efficacy and safety of Sotyktu (deucravacitinib), a selective TYK2 inhibitor (POETYK) PSO-1 (NCT03624127) and POETYK PSO-2 (NCT03611751) were global Phase 3 studies designed to evaluate the safety and efficacy of Sotyktu compared to placebo and Otezla® (apremilast) in patients with moderate-to-severe plaque psoriasis.

Both POETYK PSO-1, which enrolled 666 patients, and POETYK PSO-2, which enrolled 1,020 patients, were multicenter, randomized, double-blind trials that evaluated Sotyktu (6 mg once daily) compared to placebo and Otezla (30 mg twice daily).

POETYK PSO-2 included a randomized withdrawal and retreatment period after Week 24.

The co-primary endpoints of both POETYK PSO-1 and POETYK PSO-2 were the percentage of patients who achieved Psoriasis Area and Severity Index (PASI) 75 response and those who achieved static Physician’s Global Assessment (sPGA) score of 0 or 1 (clear/almost clear) at Week 16 versus placebo.

Key secondary endpoints of the trials included the percentage of patients who achieved PASI 75 and sPGA 0/1 compared to Otezla at Week 16 and other measures evaluating Sotyktu versus placebo and Otezla.

Across both clinical trials and timepoints, significantly more Sotyktu-treated patients achieved a sPGA score of 0/1, PASI 75 response and PASI 90 response.

Responses persisted through Week 52, as 82% (187/228) of patients who achieved PASI 75 with Sotyktu at Week 24 maintained their response at Week 52 in POETYK PSO-1.

In POETYK PSO-2, 80% (119/148) of patients who continued Sotyktu maintained PASI 75 response compared to 31% (47/150) of patients who were withdrawn from Sotyktu.

Efficacy
endpoints		POETYK PSO-1POETYK PSO-2
Sotyktu
(n=332)
n (%)		Otezla
(n=168)
n (%)		Placebo
(n=166)
n (%)		Sotyktu
(n=511)
n (%)		Otezla
(n=254)
n (%)		Placebo
(n=255)
n (%)
sPGA 0/1
Week 16178 (53.6)54 (32.1)c12 (7.2)a,c253 (49.5)86 (33.9)c22 (8.6)a,c
Week 24195 (58.7)52 (31.0)c–251 (49.8)b75 (29.5)c–
PASI 75
Week 16194 (58.4)59 (35.1)c21 (12.7)a,c271 (53.0)101 (39.8)d24 (9.4)a,c
Week 24230 (69.3)64 (38.1)c–296 (58.7)b96 (37.8)c–
PASI 90
Week 16118 (35.5)33 (19.6)d7 (4.2)c138 (27.0)46 (18.1)e7 (2.7)c
Week 24140 (42.2)37 (22.0)c–164 (32.5)b50 (19.7)c–
Non-responder imputation (NRI) was used; patients who discontinued treatment or the study prior to the endpoint or had missing data were counted as non-responders.
a Co-primary endpoint comparing Sotyktu and placebo
b n=504 accounting for missed assessments due to COVID-19 pandemic
c p ≤0.0001 for comparison between Sotyktu and placebo or Sotyktu and Otezla
d p <0.001 for comparison between Sotyktu and Otezla
e p <0.01 for comparison between Sotyktu and Otezla

Following the 52-week POETYK PSO-1 and POETYK PSO-2 trials, patients could enroll in the ongoing POETYK PSO long-term extension (LTE) trial (NCT04036435) and receive open-label Sotyktu 6 mg once-daily.

In the LTE trial, 1,221 patients were enrolled and received at least one dose of Sotyktu.

Efficacy was analyzed utilizing treatment failure rules (TFR) method of imputation, along with sensitivity analyses using modified non-responder imputation and as-observed analysis, which have been used in similar analyses with other agents.

In addition to POETYK PSO-1, POETYK PSO-2 and POETYK PSO-LTE, Bristol Myers Squibb has evaluated Sotyktu in two other Phase 3 studies in psoriasis: POETYK PSO-3 (NCT04167462) and POETYK PSO-4 (NCT03924427).”

https://news.bms.com/news/corporate-financial/2023/Bristol-Myers-Squibb-Receives-European-Commission-Approval-of-Sotyktu-deucravacitinib-a-Once-Daily-Oral-Treatment-for-Adults-With-Moderate-to-Severe-Plaque-Psoriasis/default.aspx

FDA Outlines Immediate National Strategy to Further Increase the Resiliency of U.S. Infant Formula Market

admin
-
0
FDA Outlines Immediate National Strategy to Further Increase the Resiliency of U.S. Infant Formula Market

March 28, 2023: “The U.S. FDA released a national strategy outlining actions the agency will take immediately to help ensure one of the nation’s most vulnerable populations continue to have access to safe, nutritious infant formula and to increase resiliency of the U.S. infant formula market and supply.

“Safety and supply go hand-in-hand. We witnessed last year how a safety concern at one facility could be the catalyst for a nationwide shortage. That’s why we are looking to both strengthen and diversify the market, while also ensuring that manufacturers are producing infant formula under the safest conditions possible,” said FDA Commissioner Robert M. Califf, M.D.

“Now, with this strategy, we are looking at how to advance long-term stability in this market and mitigate future shortages, while ensuring formula is safe” 

The Food and Drug Omnibus Reform Act of 2022 (FDORA) directed the FDA, in consultation with other federal government partners, to develop this immediate strategy to increase the resiliency of the U.S. infant formula market.

Key elements of the immediate strategy released today include:

  • Ensuring industry is aware of requirements to develop and implement redundancy risk management plans.
    These plans are intended to help industry identify risks to the supply chains of infant formula and medical foods and to develop mitigation plans against potential disruptions that could impact production. 
  • Continuing to enhance inspections of infant formula manufacturers, including by expanding and improving infant formula training for investigators.
  • Expediting review of premarket submissions for new infant formula products to mitigate or prevent shortages.
  • Continuing to monitor the infant formula supply and developing a forecasting model to enable FDA to prepare for and mitigate future supply disruptions.
  • Engaging with U.S. government partners who play a role in mitigating other factors that may influence the infant formula supply, such as tariffs and market concentration, to sustain the safe, continuous production of infant formula.
  • Engaging with the U.S. Department of Agriculture to support efforts to build resiliency within its Special Supplemental Nutrition Program for Women, Infants, and Children (WIC).
  • Continuing to advance the agency’s Strategy to Help Prevent Cronobacter sakazakii Illnesses Associated with Consumption of Powdered Infant Formula.
  • Improving the agency’s consumer education materials relating to infant formula on FDA.gov.
  • Enhancing and leveraging the FDA’s partnerships with health care providers and professionals, particularly infant care professionals, to further expand the agency’s consumer education program. 

These actions are well underway. FDORA also called on the FDA to trace the events that led up to and followed the voluntary recall of infant formula in February 2022 by Abbott Nutrition, the temporary pause in production at the facility in Sturgis, Michigan, and the numerous factors that contributed to the fragility of this particular supply chain. 

“The events that led up to and ultimately resulted in the voluntary recall of certain infant formula products in February 2022 shocked the infant formula supply in the U.S., creating an unparalleled challenge for parents and caregivers,” said Susan Mayne, Ph.D., director of the FDA’s Center for Food Safety and Applied Nutrition.

“Since then, the agency has had ongoing and extensive engagement with the infant formula industry to identify and implement opportunities to strengthen preventive control practices.

The immediate strategy released today will play an important role in increasing the resiliency of the infant formula market as the agency continues its critical work to improve industry’s processes and programs for the protection of those who rely on infant formula while incentivizing additional infant formula manufacturers to enter the market.”

The strategy also describes initial actions the FDA took to address the infant formula shortage and details the agency’s plans for improving the safety and resiliency of the infant formula supply, while noting considerations beyond the purview of the FDA. 

The agency remains committed to improving the infant formula supply and ensuring that consumers have the utmost confidence that infant formula available in the U.S. is safe and nutritious. 

This initial strategy represents a first step toward issuing — with input from the National Academies of Science, Engineering and Medicine (NASEM)— a long-term national strategy, also called for in FDORA, to improve preparedness against infant formula shortages by outlining methods to improve information-sharing, recommending measures for protecting the integrity of the infant formula supply chain and preventing contamination.

That long-term strategy will follow the plan released today and outline methods to incentivize entry of new infant formula manufacturers to increase supply and mitigate future shortages and recommend necessary authorities to gain insight into the supply chain and risks for shortages.

The long-term strategy is due to Congress one year after enactment, or 90 days after the NASEM issues its report. The FDA anticipates issuing this strategy publicly in early 2024.”

https://www.fda.gov/news-events/press-announcements/fda-outlines-immediate-national-strategy-further-increase-resiliency-us-infant-formula-market

Bayer Pharmaceuticals accelerates innovations for patients

admin
-
0
Bayer Pharmaceuticals accelerates innovations for patients

March 24,2023: “At its annual Pharma Media Day 2023, Bayer presented the latest progress in the ongoing transformation of its pharmaceutical business focusing on accelerating innovative healthcare solutions for the benefits of patients and delivering long-term, sustainable business growth.

“The transformation of our Pharma business is progressing well. With bold investments in industry-leading technology platform companies, we have bolstered our R&D capabilities and strengthened our early development pipeline,” said Stefan Oelrich, Member of the Board of Management, Bayer AG and President of Bayer’s Pharmaceuticals Division. “Going forward, we will focus on realizing the full potential of our projected blockbuster candidates Nubeqa™, Kerendia™, asundexian and elinzanetant.

We will specifically allocate resources to grow our oncology and cardiology businesses in the U.S. The implementation of our strategy aims to deliver new medicines to patients faster and drive sustainable growth across our key areas of focus.”

During this year’s Pharma Media Day 2023, Bayer showcased how its investments in strategic technology platforms accelerate its medical and digital innovations and will advance the company’s leadership ambitions in cardiology, oncology, and radiology.

In addition, Bayer provided insights into its innovation strategy, comprising enhanced research and development (R&D) capabilities in key areas.

“Patient needs, precision therapies to address disease complexity as well as advances in science and technology are evolving rapidly.

Therefore, we are evolving our R&D strategy and operating model to focus on differentiated, high-value breakthrough medicines,” said Christian Rommel, Member of the Executive Committee of Bayer’s Pharmaceuticals Division and Head of Research and Development.

“Moving forward, we will focus our early research activities on core strategic areas: oncology, cardiovascular, neurology & rare diseases and immunology, where we believe we can make the biggest possible difference for the patients we serve.”

Delivering significant growth through a leadership position in oncology

The company has doubled its portfolio of oncology treatments over the past five years. With Nubeqa (darolutamide) as a key growth driver it is gearing up to become a leader in prostate cancer, the second most commonly diagnosed cancer in men.

Nubeqa has the potential to become the standard of care for prostate cancer patients from the early- to the late-stage of this disease.

Going forward, growth of Nubeqa will be driven by recent approvals for the treatment of metastatic hormone-sensitive prostate cancer (mHSPC) in the U.S., EU, Japan, and China.

In parallel, the robust Phase III clinical development program with darolutamide aims to broaden its use from the metastatic into the earlier disease stages, e.g. in patients with hormone-sensitive prostate cancer with high-risk of biochemical recurrence and as an adjuvant treatment for patients with localized prostate cancer with very high risk of recurrence.

The company has projected a peak sales potential for Nubeqa of more than three billion euros.

“With Nubeqa’s potential to set a new standard of care in prostate cancer, its compelling clinical program across the spectrum of prostate cancer and our growing early pipeline, we are well placed to deliver on our commitment to bring innovative medicines to cancer patients,” said Christine Roth, Member of the Executive Committee of Bayer’s Pharmaceuticals Division and Head of the Oncology Strategic Business Unit.

“Bayer has clear ambitions to become a top oncology company. We are investing in innovative treatments and modalities that have the potential to be transformative for a broad range of cancer patients, who currently have little or no treatment options.”

Bayer is advancing its oncology R&D efforts in three scientific areas that have the potential to address unmet needs in cancer patients.

This includes targeted radiopharmaceuticals (specifically targeted alpha therapies), next-generation immuno-oncology and precision molecular oncology approaches.

In these areas, the company is advancing truly first-in-class, highly innovative projects in its portfolio. Nearly one third of new molecular entities within Bayer’s development pipeline is dedicated to fighting cancer, spanning all phases of clinical development.

With recent major investments, Bayer has fortified its oncology innovation engine substantially.

The new $140 million Bayer Research and Innovation Center (BRIC) facility, located in the center of thriving Boston-Cambridge, Massachusetts, is home to Bayer’s precision molecular oncology research group, fostering collaborations in this outstanding U.S. innovation hub, including with the Broad Institute of MIT and Harvard.

Vividion Therapeutics, which Bayer acquired for $1.5 billion, comprises a leading chemoproteomics platform capable of identifying previously unknown binding pockets in undruggable targets to generate first-in-class novel drug candidates in indications of high unmet medical need.

This technology and the unique approach of an innovative arms-length operating model have the potential to accelerate the timelines to get assets into the clinic.

For the first two programs, which are addressing high profile cancer targets NRF2 and STAT3, Vividion is expected to file the Investigational New Drug Application (IND) in the U.S. in 2023.

Developing the next generation of cardiovascular therapies

The short- to medium-term growth in the area of cardiovascular (CV) diseases will be driven mainly by Kerendia (finerenone), a non-steroidal, selective mineralocorticoid receptor antagonist, representing a valuable treatment option for patients with cardio-renal disease.

Kerendia is already approved in major markets in chronic kidney disease (CKD) associated with type 2 diabetes.

Bayer has exclusive worldwide marketing rights for this product including the U.S.

The ongoing development program to expand Kerendia into additional indications comprises three Phase III studies: FINEARTS-HF, which investigates the compound in patients with symptomatic heart failure with preserved ejection fraction; FIND-CKD, with the goal to make the medicine available also to patients with non-diabetic CKD etiologies including hypertension and chronic glomerulonephritis (inflammation of the kidneys); and FIONA, which investigates finerenone in pediatric patients with CKD and severely increased proteinuria.

Kerendia is estimated to generate peak sales of more than three billion euros.

Bayer’s investigational treatment of cardiovascular diseases asundexian is currently in Phase III clinical development.

Asundexian is an oral Factor XIa (FXIa) small molecule inhibitor being investigated in patients with atrial fibrillation (AF) as well as in patients with an acute non-cardioembolic ischemic stroke or high-risk transient ischemic attack (TIA), a temporary period of symptoms similar to those of a stroke.

The respective Phase III clinical trial program, OCEANIC, which has recently started enrolling patients, is one of the largest conducted in the company’s history. Asundexian, which belongs to a new class of drugs, has a projected peak sales potential of more than five billion euros.

“Cardiovascular diseases can have a significant impact on patients’ lives. We are advancing highly innovative projects in our portfolio to address patients’ high unmet medical needs and shape the future in cardiology,” said Christian Rommel, Member of the Executive Committee of Bayer’s Pharmaceuticals Division and Head of Research and Development.

“The number of innovative approaches and novel modalities in our portfolio will triple in future. With those we will be able to target pathologies and disease mechanisms that were not druggable before.”

Building on its strong late-stage projects, the company has set a clear directive towards precision cardiology, exploring innovative approaches across a diverse range of modalities, with the aim of pushing the boundaries of innovation for patients with cardiovascular diseases with high unmet needs and potentially reversing life-altering conditions.

Complementing in-house expertise with external collaborations remains a vital part of Bayer’s strategy to expand its R&D expertise and develop robust cardiovascular therapies, with the goal of addressing high unmet medical needs across broad indications, including specialty cardiology and rare indications.

Through Asklepios BioPharmaceutical Inc. (AskBio), Bayer is developing a gene therapy as a potential new approach for treating congestive heart failure.

With the acquisition of AskBio, Bayer gained access to an industry-leading gene therapy platform with demonstrated applicability and world-class manufacturing facilities.

Beyond congestive heart failure, AskBio is also currently advancing four clinical-stage and a number of preclinical candidates addressing medical need indications across a diverse set of diseases such as Parkinson’s disease, Pompe disease and Huntington’s disease.

Bayer’s advancements in cardiovascular disease are enabled by digitalized, decentralized and more diverse clinical trials, as well as partnerships on industry-leading sources of data, such as the Accelerating Medicines Partnership in Heart Failure and the Broad Institute’s Precision Cardiology Laboratory.

These initiatives are helping to accelerate the discovery of new molecules and bring new treatments to patients that need them.

Leveraging digital innovation to support diagnosis and drug discovery

Bayer is implementing its innovation strategy in radiology, with a key focus on digital medical imaging and artificial intelligence (AI). The medical imaging AI market is the fastest growing segment within the global radiology industry.

The estimated compounded annual growth rate is more than 26 percent reaching $1.36 billion in sales by 2026. 

A cornerstone of Bayer’s ambition is its recently launched platform Calantic™ Digital Solutions, which delivers access to digital applications, including those powered by AI.

It provides an ecosystem of third-party and Bayer products to deliver disease-oriented solutions for radiologists and their teams. The platform is designed to support healthcare professionals at various stages of their work, from diagnosis through treatment of their patients.

To bolster its position in the medical imaging AI market, Bayer recently acquired the global strategic imaging AI platform and solutions provider Blackford Analysis Ltd, which will operate under Bayer’s well established “arm’s length” operating model.

Utilizing Blackford’s and Bayer’s combined expertise in healthcare technology, this acquisition fosters Bayer’s engagement to drive digital innovation in healthcare, with the aim to deliver true value to radiologists and their teams for the benefit of their patients.

“Building on a deep medical expertise across a multitude of diagnostic and therapeutic areas, Bayer is a leader in key radiology segments with a comprehensive portfolio that generated close to 2 billion euros sales in 2022,” said Gerd Krueger, President Radiology, Bayer.

“Expanding our offerings into the field of AI further advances our strong position in this market. It follows our mission to deliver disease-oriented solutions for patients and their physicians.

The transformative expansion of our radiology business is a key element of Bayer’s accelerated digital transformation, which strategically leverages data science and advanced analytics across the value chain.”

Bayer is investing to leverage the power of data and rapidly evolving technologies such as artificial intelligence throughout the entire pharma value chain, from discovery to production.

The company is partnering with technology leaders such as Google Cloud and biotech leaders such as Recursion Pharmaceuticals, Inc. to access new areas of innovation.

In addition, Bayer has built up extensive in-house expertise to transform its development, manufacturing and medical operations to better serve the needs of patients, healthcare professionals and healthcare systems with relevant innovative solutions. Bayer works on leveraging all the possibilities of the technologies while fully integrating all regulatory and bioethical requirements.”

https://www.bayer.com/media/en-us/bayer-pharmaceuticals-accelerates-innovations-for-patients/

Macomics and Ono Pharmaceutical partner to discover Cancer therapy

admin
-
0
Macomics and Ono Pharmaceutical partner to discover Cancer therapy

March 23, 2023: “Macomics Ltd, a leader in macrophage drug discovery, announces that it has entered into a worldwide drug discovery collaboration agreement with Ono Pharmaceutical Co., Ltd. (Osaka, Japan; President and CEO: Gyo Sagara; “Ono”) to develop new immuno-oncology antibody drugs against a novel macrophage target of interest in cancer.

Under the terms of the agreement, Macomics will identify and characterise antibody candidates against the novel target of interest using its ENIGMACTM macrophage drug discovery platform.

Ono will have an exclusive option to license global rights to the candidates for further development and commercialization.

Macomics will receive an up-front payment, R&D funding, and success-based milestone payments, as well as tiered royalties based on global net sales.

Macomics is exploiting the potential of macrophage-based approaches to develop novel precision medicines to target disease specific macrophage biology.

Macomics’ ENIGMAC macrophage drug discovery platform integrates large volume human data sets, custom cell models, and proprietary human macrophage genome editing capability to discover novel targets and unlock disease specific target biology.

Macrophages (TAMs) are often the most abundant immune cell many types of cancer and modulating TAMs can enhance the body’s ability to fight cancer.

Toichi Takino, Senior Executive Officer / Executive Director, Discovery & Research of Ono said:

“Targeting macrophages in immune-oncology is emerging as an exciting area with significant opportunity to deliver novel therapeutics to improve cancer outcomes and to change the lives of patients with cancer. Macomics has demonstrated the power of its macrophage platform and drug discovery approach and we are delighted to partner with them on taking this novel target discovery program forwards.”

Stephen Myatt, CEO of Macomics said:

“This global collaboration with Ono is testament to our strong program portfolio, and the unique enablement offered by our ENIGMAC discovery platform, and our world class R&D team. Ono is a leader in immuno-oncology and we are delighted to have a partner in Ono who brings the complementary skills necessary to succeed in this therapeutic area.”

https://macomics.com/2023/03/23/macomics-and-ono-pharmaceutical-partner-to-discover-and-develop-macrophage-targeting-antibody-therapy/

Debiopharm announces launch of galuci study for its Caix-Targeted radiopharmaceutical program

admin
-
0
Debiopharm announces launch of galuci study for its Caix-Targeted radiopharmaceutical program

MARCH 22, 2023: ” Debiopharm, a Swiss-based, global biopharmaceutical company, aiming to establish tomorrow’s standard-of-care to cure cancer and infectious diseases announced the first patient dosed of their first-in-human, phase 1/2 study, GaLuCiTM.

The first patient was screened and dosed at the Australian-based Peter MacCallum Cancer Centre.

This multicenter international trial, evaluating a radioligand theranostic pair will be carried out in three stages: Part A to confirm the safety and reliability of Debio 0328 in detecting CAIX-expressing solid tumors, Part B to assess escalating doses of the therapeutic agent, Debio 0228 in patients, whose tumors show high uptake of Debio 0328 and finally, based on the recommended dose from part B, Part C will further assess safety and preliminary efficacy in selected tumor types.

Currently, Debio 0228/0328 is the only peptide-based theranostic pair targeting CAIX in clinical development, with pan-tumor potential, and developed first for patients with advanced cancers such as renal, pancreatic, and colorectal.

It leverages a theranostic approach to identify and deliver radiation to diseased tissues, allowing the imaging-based pre-identification of patients who have the target proteins necessary to respond to the targeted radioligand.

“The results of the GaLuCiTM trial are highly anticipated considering the therapeutic potential of Debio 0228 as observed in preclinical models.

Using this theranostic pair could pave the way for personalized nuclear medicine, enabling administration of the lutetium coupled radioligand only to patients who are more likely to respond to the therapy.” explained Angela Zubel, Chief Development Officer at Debiopharm.

“We always have immense gratitude for our patients who participate in first time in human trials, but in this case, we are particularly thankful for our patient who agreed to be the first person in the world to have their kidney cancer imaged with Debio 0328 on the GaLuCiTM trial. 

We hope this is the beginning of the theranostics era in kidney cancer!” expressed Dr. Ben Tran, Lead Genito-urinary medical oncologist, Peter MacCallum Cancer Centre. 

“We are excited about this first-in-human study as it is a novel approach for advanced kidney cancer patients,” said Darren R. Feldman, MD, Associate Attending Physician, Genitourinary Oncology Service at Memorial Sloan Kettering Cancer Center. 

“Precision nuclear medicine applied to CA9 could benefit advanced cancer patients who still experience a high unmet medical need. This theranostic pair allows targeted radiation delivery to the cancer cells bearing CAIX, which is largely expressed, over 85%, in clear cell renal cell carcinoma.”  

https://www.debiopharm.com/drug-development/press-releases/debiopharm-announces-launch-of-the-phase-1-2-galucitm-study-for-its-caix-targeted-radiopharmaceutical-program/

AZ’s Eplontersen shpows benefit for hereditary transthyretin-mediated amyloid polyneuropathy through 66 weeks

admin
-
0
AZ’s Eplontersen shpows benefit for hereditary transthyretin-mediated amyloid polyneuropathy through 66 weeks

March 27, 2023: “Positive high-level results from the NEURO-TTRansform Phase III trial in patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN) showed eplontersen met its co-primary endpoints through 66 weeks.

The results were consistent with the positive 35-week findings announced in June 2022.

At 66 weeks, patients treated with eplontersen continued to demonstrate a statistically significant and clinically meaningful change from baseline versus an external placebo group on the co-primary endpoints of modified Neuropathy Impairment Score +7 (mNIS+7), a measure of neuropathic disease progression, and Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN).

The trial also met its third co-primary endpoint demonstrating a statistically significant reduction in serum TTR concentration versus an external placebo group. TTR reductions were consistent with those reported at week 35.

Eplontersen continued to demonstrate a safety and tolerability profile consistent with that observed at 35 weeks.

Sami Khella, M.D., Chief, Department of Neurology, Penn Presbyterian Medical Center and Professor of Clinical Neurology, University of Pennsylvania School of Medicine, said: “The positive results from the 66-week analysis of the Phase III NEURO-TTRansform trial show that eplontersen provided consistent and sustained transthyretin protein reduction and that a substantial number of patients improved in measures of both neuropathy progression and quality of life.

This builds on the favourable 35-week results, which first demonstrated eplontersen’s potential to significantly improve outcomes in this underserved population.”

Mene Pangalos, Executive Vice President, BioPharmaceuticals R&D, AstraZeneca, said: “These results further underscore eplontersen’s potential to be a best-in-class treatment across all forms of transthyretin-mediated amyloidosis, including polyneuropathy and cardiomyopathy which can lead to heart failure.

With limited treatment options currently available, there is an urgent unmet medical need for new therapies and earlier, accurate diagnosis across the different types of this systemic, progressive and fatal condition.”

Eugene Schneider, M.D., Executive Vice President and Chief Clinical Development Officer for Ionis, said: “These latest results from our NEURO-TTRansform study represent an important step towards delivering a potential new therapy for ATTRv-PN patients living with this debilitating and fatal disease. We are encouraged by the sustained benefit demonstrated by eplontersen and what a self-administered treatment could mean for patients and families affected by ATTRv-PN.

Together, with our partner AstraZeneca, we look forward to sharing detailed results from this study at the upcoming American Academy of Neurology Annual Meeting.”

ATTRv-PN is a debilitating disease that leads to peripheral nerve damage with motor disability within five years of diagnosis and, without treatment, is generally fatal within a decade.

As part of a global development and commercialisation agreement, AstraZeneca and Ionis are seeking regulatory approval for eplontersen for the treatment of ATTRv-PN in the US and plan to seek regulatory approval in Europe and other parts of the world.

Earlier this month, the US Food and Drug Administration accepted a New Drug Application for eplontersen for the treatment of ATTRv-PN.

Eplontersen was granted Orphan Drug Designation in the US.

Eplontersen is currently being evaluated in the Phase III CARDIO-TTRansform trial for transthyretin amyloid cardiomyopathy (ATTR-CM), a systemic, progressive and fatal condition that typically leads to progressive heart failure and often death within three to five years from disease onset.

The results from both the 35 and 66-week analyses of the trial will be presented as an Emerging Science presentation at the American Academy of Neurology Annual Meeting in April.

Notes

TTR Amyloidosis
ATTR cardiomyopathy and polyneuropathy are progressive systemic diseases caused by aging or genetic mutations, resulting in misfolded TTR protein and accumulation as amyloid fibrils in the cardiac myocardium and peripheral nerves, respectively.

In patients with ATTR, both hereditary and wild type (non-hereditary), TTR protein builds up as fibrils in tissues, such as the peripheral nerves and heart, gastrointestinal system, eyes, kidneys, central nervous system, thyroid and bone marrow.

The presence of TTR fibrils interferes with the normal functions of these tissues.

As the TTR protein fibrils accumulate, more tissue damage occurs and the disease worsens, resulting in poor QoL and eventually death.8 Worldwide, there are an estimated 300,000 – 500,000 patients with ATTR-CM9 and about 40,000 patients with ATTRv-PN.

NEURO-TTRansform
NEURO-TTRansform is a global, open-label, randomised trial evaluating the efficacy and safety of eplontersen in patients with ATTRv-PN.

The trial has enrolled adult patients with ATTRv-PN Stage 1 or Stage 2 and will be compared to the external placebo group from the TEGSEDI® (inotersen) NEURO-TTR registrational trial that Ionis completed in 2017.

The final analysis comparing eplontersen to external placebo was completed at week 66 and all patients will be followed on treatment until week 85, when they will have the option to transition into an open-label extension study.

The 66-week analysis evaluated percent change from baseline in serum TTR concentration, changes in the mNIS+7 and Norfolk-QOL-DN in the eplontersen group versus an external placebo group.

The mNIS+7 uses highly standardised, quantitative and referenced assessments to quantify muscle weakness, muscle stretch reflexes, sensory loss and autonomic impairment.

The Norfolk QoL-DN is a patient-reported questionnaire capturing neuropathy-related QoL.

Eplontersen
Eplontersen is a ligand-conjugated antisense (LICA) investigational medicine designed to reduce the production of transthyretin, or TTR protein, to treat all types of ATTR, a systemic, progressive and fatal disease.”

https://www.astrazeneca.com/media-centre/press-releases/2023/eplontersen-demonstrated-sustained-benefit-in-phase-iii-trial.html

Novartis Kisqali® Phase III NATALEE trial meets primary endpoint at interim analysis for breast cancer

admin
-
0
Novartis Kisqali® Phase III NATALEE trial meets primary endpoint at interim analysis for breast cancer

March 27, 2023: “Novartis announced positive topline results from an interim analysis of NATALEE, a Phase III trial evaluating Kisqali® (ribociclib) plus endocrine therapy (ET) in a broad population of patients with hormone receptor-positive/human epidermal growth factor receptor 2-negative (HR+/HER2-) early breast cancer (EBC) at risk of recurrence.

The Independent Data Monitoring Committee recommended stopping the trial early as the primary endpoint of invasive disease-free survival (iDFS) has been met.

Kisqali plus ET significantly reduced the risk of disease recurrence, compared to standard adjuvant ET alone, with consistent benefit in patients with stage II and stage III EBC regardless of nodal involvement.

“While most patients are diagnosed and treated early with the aim to cure breast cancer, the risk of cancer returning, often as metastatic disease, peaks within three years after diagnosis, but never goes away completely,” said Dennis J. Slamon, MD, Director of Clinical/Translational Research, University of California, Los Angeles Jonsson Comprehensive Cancer Center and Chairman and Executive Director of Translational Research In Oncology (TRIO) and NATALEE trial lead investigator.

“There is a critical need for new, well-tolerated options that keep patients cancer-free without disrupting quality of life.

The NATALEE trial, where ribociclib was given for three years plus ET, was designed with these unmet needs in mind, and it is extremely encouraging that this study met its primary endpoint.” 

Per the NATALEE study protocol, patient follow-up will continue to evaluate long-term outcomes, including overall survival1.

“The positive topline results from NATALEE represent a major milestone in our ambition to expand the benefits of Kisqali to patients with earlier stages of breast cancer, building on the heritage of this effective treatment in HR+/HER2- metastatic breast cancer,” said Shreeram Aradhye, M.D., President, Global Drug Development and Chief Medical Officer, Novartis.

“These data have the potential to be paradigm-shifting for patients at risk of recurrence, including those with no nodal involvement, who have limited well-tolerated options to prevent recurrence.

Our teams are working on submissions to health authorities around the world with the hope to bring Kisqali to many more patients diagnosed with breast cancer.”

These findings build on the legacy of Kisqali in metastatic breast cancer (MBC), where it has consistently demonstrated overall survival benefit while preserving or improving quality of life across three Phase III trials.

Updates to the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) for breast cancer, released in January 2023, recommend ribociclib (Kisqali) as the only Category 1 preferred CDK4/6 inhibitor for first-line treatment of patients with HR+/HER2- MBC when combined with an aromatase inhibitor (AI).

About NATALEE
NATALEE is a global Phase III multi-center, randomized, open-label trial to evaluate the efficacy and safety of Kisqali with ET as adjuvant treatment versus ET alone in patients with HR+/HER2- EBC, being conducted in collaboration with Translational Research In Oncology (TRIO).

The primary endpoint of NATALEE is iDFS as defined by the Standardized Definitions for Efficacy End Points (STEEP) criteria; secondary endpoints include safety, quality of life, and overall survival, among others. iDFS is a composite endpoint in EBC adjuvant trials, which incorporates locoregional relapse, ipsilateral and contralateral invasive breast cancer, distant recurrence, and types of new cancer events or death from any cause.

Approximately 5,100 adult patients with HR+/HER2- EBC across 20 countries were randomized in the trial, including patients with tumor stages IIA (select patients), IIB or III, regardless of nodal involvement. NATALEE explored a lower starting dose (400 mg) of Kisqali than the dose approved for treatment in MBC (600 mg) with the goal to minimize disruptions to patient quality of life without compromising efficacy1.

About Early Breast Cancer
More than 90% of patients diagnosed with breast cancer have EBC. Approximately 30-60% of people with HR+/HER2- stage II and III EBC treated with ET only remain at risk of breast cancer recurrence.

The risk of recurrence peaks within the first three years after initial diagnosis and continues over decades. For many of these patients, there are currently no targeted therapeutic options outside of the standard chemotherapy and ET.

About Kisqali® (ribociclib) 
Kisqali has consistently demonstrated overall survival benefit while preserving or improving quality of life across three Phase III trials. Updates to the NCCN Guidelines® for breast cancer, released in January 2023, recommend ribociclib (Kisqali) as the only Category 1 preferred CDK4/6 inhibitor for first-line treatment of patients with HR+/HER2- MBC when combined with an AI.

Additionally, Kisqali has the highest rating of any CDK4/6 inhibitor on the ESMO Magnitude of Clinical Benefit Scale, achieving a score of five out of five for first-line premenopausal patients with HR+/HER2- advanced breast cancer.

Further, Kisqali in combination with either letrozole or fulvestrant has uniquely, among other CDK4/6 inhibitors, received a score of four out of five for postmenopausal patients with HR+/HER2- advanced breast cancer treated in the first line19.
        
Kisqali has been approved in 99 countries worldwide, including by the United States Food and Drug Administration (FDA) and the European Commission.

In the U.S., Kisqali is approved for the treatment of adult patients with HR+/HER2- advanced or metastatic breast cancer in combination with an AI as initial ET or fulvestrant as initial ET or following disease progression on ET in postmenopausal women or in men. In the EU, Kisqali is approved for the treatment of women with HR+/HER2- advanced or metastatic breast cancer in combination with either an AI or fulvestrant as initial ET or following disease progression. In pre- or perimenopausal women, the ET should be combined with a luteinizing hormone-releasing hormone agonist.

https://www.novartis.com/news/media-releases/novartis-kisqali-phase-iii-natalee-trial-meets-primary-endpoint-interim-analysis-demonstrating-clinically-meaningful-benefit-broad-population-patients-early-breast-cancer

FDA Issues Draft Guidance Aimed at Improving Oncology Clinical Trials for Accelerated Approval

admin
-
0
FDA Issues Draft Guidance Aimed at Improving Oncology Clinical Trials for Accelerated Approval

March 24, 2023: “The U.S. FDA issued draft guidance, Clinical Trial Considerations to Support Accelerated Approval of Oncology Therapeutics, regarding clinical trial design considerations to support accelerated approval applications.

The accelerated approval pathway is commonly used for approval of oncology drugs in part due to the serious and life-threatening nature of cancer and because of available intermediate clinical endpoints likely to predict clinical benefit.

“The FDA’s accelerated approval program has provided patients with cancer earlier access to novel treatments that can be practice changing,” said Richard Pazdur, MD, Director of the FDA’s Oncology Center of Excellence.

“Today’s draft guidance provides recommendations to sponsors for designing clinical trials to support accelerated approval. Building quality and efficiency into the design of oncology clinical trials is a crucial component in providing maximum benefit to those living with cancer.”

The draft guidance discusses the design of clinical trials, and ways to improve the data available at the time of accelerated approval and reduce clinical uncertainty for patients by initiating postmarketing confirmatory studies in a timely manner.

Specifically, the draft guidance addresses the design, conduct and analysis of data through two randomized clinical trial approaches – conducting two separate randomized controlled clinical trials or using one trial for both accelerated approval and to verify clinical benefit.

The draft guidance also provides considerations for sponsors to determine the adequacy of single-arm studies to support an application.
  
For drugs granted accelerated approval, postmarketing confirmatory trials have been required to verify and describe the anticipated clinical benefit.

The draft guidance discusses a potential advantage of randomized clinical trials–compared to single-arm trials–by highlighting that use of the one-trial approach, in appropriate cases, may not require separate clinical trials because longer term follow-up in the same trial could fulfill a postmarketing requirement to verify clinical benefit.

Moreover, confirmatory trials that are in progress at the time of accelerated approval are more likely to result in a timely verification of clinical benefit, therefore minimizing the period of uncertainty for patients.

In a commentary in the New England Journal of Medicine in 2022External Link Disclaimer, Oncology Center of Excellence staff outlined the concepts included and expanded upon in the draft guidance.

The Oncology Center of Excellence has also launched Project Confirm, as an initiative that promotes the transparency of outcomes related to accelerated approval for oncology indications and  fosters discussion and research on the accelerated approval program.

The project developed a searchable database with information on the status of all oncology accelerated approvals, a model that was then adopted by FDA’s Center for Drug Evaluation and Research for all accelerated approvals.”

https://www.fda.gov/news-events/press-announcements/fda-issues-draft-guidance-aimed-improving-oncology-clinical-trials-accelerated-approval

1...345...156Page 4 of 156

EDITOR PICKS

POPULAR POSTS

POPULAR CATEGORY

ABOUT US

LifeProNow provides guidance and high quality contents to help job seekers to get their dream Job. All the contents are written by experienced folks and peer reviewed by experts.

Contact us: [email protected]

FOLLOW US

© LifeProNow | All Rights Reserved