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FDA Flexibilities, Collaboration to Yield Millions of Bottles of Specialized Medical Infant Formula in Coming Months to Increase U.S. Supply

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FDA Flexibilities, Collaboration to Yield Millions of Bottles of Specialized Medical Infant Formula in Coming Months to Increase U.S. Supply

May 26, 2022: “The U.S. FDA is providing an update on steps it has taken that will lead to millions of bottles of specialized medical infant formula to help address immediate needs for infants with certain allergies or critical health conditions.

This type of specialized medical infant formula is currently in short supply in the U.S.

“We have made tremendous progress, including notable steps in just the past week, which will allow us to immediately begin bringing specialty and infant formula products into the U.S. as quickly as possible,” said FDA Commissioner Robert M. Califf, M.D.

“We continue to work closely with our U.S. government partners and domestic and international manufacturers to identify additional formula product that will be available to parents and caregivers in the weeks and months ahead.

It is our goal to ensure that hospitals, specialty pharmacies, and retail store shelves will begin seeing adequate supplies again in the coming weeks.”

About 500,000 additional cans of specialized medical formula manufactured by Danone’s Nutricia business will be headed to the U.S.

These Neocate amino acid-based formula products — some of which are already manufactured for the U.S. market and are made at facilities in Europe — amounts to more than 5 million full-size, 8-ounce bottles. 

The U.S. Department of Health and Human Services and Danone continue to partner to evaluate options for getting the products to the U.S. as quickly as possible.

The specialized medical formula is expected to be distributed through direct ordering, hospitals, health care professionals, pharmacies, healthcare product distributors and Special Supplemental Nutrition Program for Women, Infants, and Children (WIC) local access points to ensure they get to the infants who rely on these products.

The FDA has been working with Danone’s Nutricia business over the past several months to boost production of these products at its facilities and to identify additional supply of other infant formula products moving forward.

The agency is leveraging a number of flexibilities to bolster the supply of products that serve as the sole source of nutrition for many infants while ensuring the infant formula can be used safely and provides adequate nutrition.

The FDA remains in further discussions with manufacturers and suppliers regarding additional supply to ensure there’s adequate infant formula available wherever and whenever parents and caregivers need it.

On Tuesday, the FDA announced that it informed Kendal Nutricare that the agency is exercising enforcement discretion for the importation of certain infant formula under the Kendamil brand.

Under the agency’s recent increased flexibilities regarding importation of certain infant formula products, the company initially estimates that about 2 million cans of infant formula (over 50 million full-size, 8-ounce bottles) are expected to land on U.S. store shelves beginning in June.

Kendal Nutricare also currently has over 40,000 cans in stock for immediate dispatch.

The FDA also announced that it is not objecting to the release of about 300,000 cans of EleCare amino acid-based infant formula previously produced at Abbott Nutrition’s Sturgis, Michigan, facility to individuals needing urgent, life-sustaining supplies of this specialty formula on a case-by-case basis.

These products will undergo enhanced microbiological testing before release.

Last week, the FDA issued guidance that outlined a process by which the FDA would not object to the importation of certain infant formula products intended for a foreign market or distribution in the U.S. of products manufactured here for export to foreign countries.

This guidance also may provide flexibilities to those who manufacture infant formula products domestically and may be able to increase further the quantity of domestically produced product for the U.S. market. 

The FDA has posted a webpage that will be updated with information about additional products headed to the U.S.

The agency’s around-the-clock work as part of the all-of-government efforts has already begun to improve supply and availability.

The agency expects that the measures and steps it is taking, and the potential for Abbott Nutrition’s Sturgis, Michigan, facility to safely resume production in the near-term, will mean more and more supply is on the way or on store shelves moving forward. 

As more formula becomes available to the general population of infants, the agency understands that availability of specialty products — such as amino acid-based specialty formulas and metabolic products — continues to be of concern.

The FDA has already taken steps with Abbott Nutrition to make product available to those with life-threatening conditions on a case-by-case basis and will continue its efforts to make these products even more readily available as the agency works with the company to implement provisions of a consent decree.

In addition, these products have been an area of focus for discussions with other manufacturers that make comparable products.

As a result of the recall and work with the FDA, other manufacturers have increased production of comparable product lines and in some cases expedited the importation of these products where available.

The FDA continues to advise against making infant formulas at home or diluting formula. Caregivers are encouraged to work with their child’s health care provider for recommendations on changing feeding practices, if needed.

HHS has also released a fact sheet with information to help families find infant formula.

The agency also monitors online marketplaces for fraudulent products and works with major online retailers to remove violative and harmful products offered for sale on their sites.

Additionally, since many of these fraudulent products originate overseas, the agency targets and examines these products at ports of entry.

The FDA also monitors and follows up on various external signals such as consumer complaints about potential counterfeit and fraudulent products.

The FDA will continue to dedicate all available resources to help ensure that infant formula products remain available for use in the U.S. and will keep the public informed of progress updates.”

https://www.fda.gov/news-events/press-announcements/fda-flexibilities-collaboration-yield-millions-bottles-specialized-medical-infant-formula-coming

FDA Flexibilities to Yield Millions of Cans of Additional Infant Formula in Coming Months to Increase Supply Available to U.S. Consumers

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FDA Flexibilities to Yield Millions of Cans of Additional Infant Formula in Coming Months to Increase Supply Available to U.S. Consumers

May 24, 2022: “The U.S. FDA is providing an update on steps it has taken that will lead to millions of cans of additional infant and specialty formula being available to U.S. consumers.

“We continue to do everything in our power as part of the all-of-government efforts to ensure there’s adequate infant formula available wherever and whenever parents and caregivers need it,” said FDA Commissioner Robert M. Califf, M.D.

“Our recent steps will help further bolster supply of infant formula, including through the import of safe and nutritious products from overseas based on our increased flexibilities announced last week.

Importantly, we anticipate additional infant formula products may be safely and quickly imported into the U.S. in the near-term based on ongoing discussions with manufacturers and suppliers worldwide.”

Key Activities

U.K.’s Kendal Nutricare Will Send About 2 Million Cans to U.S. 

The FDA has informed Kendal Nutricare that the agency is exercising enforcement discretion for the importation of certain infant formula under the KendamilExternal Link Disclaimer brand.

Under the agency’s recent increased flexibilities regarding importation of certain infant formula products, the company initially estimates that about 2 million cans of infant formula are expected to land on U.S. store shelves beginning in June.

Kendal Nutricare currently has over 40,000 cans in stock for immediate dispatch and the U.S. Department of Health and Human Services has initiated conversations to evaluate options for getting the products to the U.S. as quickly as possible.

Kendamil has set up a websiteExternal Link Disclaimer for consumers to receive updates and locate product once it arrives in the U.S. 

The agency reviewed applicable information relating to certain Kendamil products and, based on the information provided, does not have concerns that the products can be used safely and that they provide adequate nutrition.

In reaching this decision, the FDA evaluated information pertaining to nutritional adequacy and safety, including microbiological testing, labeling, and additional information about facility production and inspection history. 

Today’s announcement follows guidance issued last week that outlined a process by which the FDA would not object to the importation of certain infant formula products intended for a foreign market or distribution in the U.S. of products manufactured here for export to foreign countries.

This guidance also may provide flexibilities to those who manufacture infant formula products domestically and may be able to increase further the quantity of domestically produced product for the U.S. market. 

The agency remains in further discussions with manufacturers and suppliers regarding additional supply and intends to prioritize submissions for products that can demonstrate safety and nutritional adequacy and have the largest volume of product available and/or those who can get product onto U.S. shelves the quickest.

The FDA has posted a webpage that will be updated with information about additional products headed to the U.S.

Abbott Will Release About 300,000 Cans of EleCare Specialty Formula on Case-By Case Basis

The FDA is announcing that it is not objecting to the release of about 300,000 cans of EleCare amino acid-based infant formula previously produced at Abbott Nutrition’s Sturgis, Michigan, facility to individuals needing urgent, life-sustaining supplies of this specialty formula on a case-by-case basis.

These products will undergo enhanced microbiological testing before release. Although some EleCare product was included in Abbott Nutrition’s infant formula recall, these EleCare products that will be released were in different lots, have never been released and have been maintained in storage under control by Abbott Nutrition.

These EleCare product lots were not part of the recall but have been on hold due to concerns that they were produced under insanitary conditions observed at Abbott Nutrition’s Sturgis, Michigan, facility.

As noted above, these products will undergo enhanced microbiological testing prior to their release. 

Given the critical need of this product for some individuals, the FDA encourages parents and caregivers to consult with their healthcare providers to weigh the potential risk of bacterial infection with this product.

The FDA will continue to work closely with healthcare provider organizations and stakeholders to understand the potential risks and benefits of using this product.

Parents and caregivers seeking access to these products should contact Abbott directly to request that a product be made available to them by calling 1-800-881-0876.

Parents and caregivers should continue to work with their medical provider to consider whether comparable alternative products may be appropriate.

The FDA is also announcing that in response to the agency’s concerns, Abbott has confirmed to the agency that EleCare will be the first formula produced at the Sturgis facility when it restarts production, and other specialty metabolic formulas will closely follow.

Additionally, under the consent decree with the FDA, Abbott is required to retain an independent expert to review the Sturgis facility’s operations to ensure compliance with the law. Additional Ongoing Efforts to Increase Supply of Infant Formula

On Friday, the agency held a webinar with more than 700 attendees to review the recent guidance for the infant formula industry, particularly those manufacturers and processors not currently manufacturing infant formula products for the U.S. market. 

The FDA’s around-the-clock work as part of the all-of-government efforts has already begun to improve supply and availability.

The agency expects that the measures and steps it is taking, and the potential for Abbott Nutrition’s Sturgis, Michigan, facility to safely resume production in the near-term, will mean more and more supply is on the way or on store shelves moving forward. 

As more formula becomes available to the general population of infants, the agency understands that availability of specialty products — such as amino acid-based specialty formulas and metabolic products — continues to be of concern.

The FDA has already taken steps with Abbott Nutrition to make product available to those with life-threatening conditions on a case-by-case basis and will continue its efforts to make these products even more readily available as the agency works with the company to implement provisions of a consent decree.

In addition, these products have been an area of focus for discussions with other manufacturers that make comparable products.

As a result of the recall and work with the FDA, other manufacturers have increased production of comparable product lines and in some cases expedited the importation of these products where available.

The FDA continues to advise against making infant formulas at home or diluting formula. Caregivers are encouraged to work with their child’s health care provider for recommendations on changing feeding practices, if needed.

The U.S. Department of Health and Human Services has also released a fact sheet with information to help families find infant formula.

The agency also monitors online marketplaces for fraudulent products and works with major online retailers to remove violative and harmful products offered for sale on their sites.

Additionally, since many of these fraudulent products originate overseas, the agency targets and examines these products at ports of entry.

The FDA also monitors and follows up on various external signals such as consumer complaints about potential counterfeit and fraudulent products.

The FDA will continue to dedicate all available resources to help ensure that infant formula products remain available for use in the U.S. and will keep the public informed of progress updates.”

https://www.fda.gov/news-events/press-announcements/fda-flexibilities-yield-millions-cans-additional-infant-formula-coming-months-increase-supply

PTC Therapeutics Upstaza™ Receives Positive CHMP Opinion for AADC Deficiency

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PTC Therapeutics Upstaza™ Receives Positive CHMP Opinion for AADC Deficiency

May 20, 2022: “PTC Therapeutics announced that Upstaza™ (eladocagene exuparvovec; PTC-AADC) received a positive opinion from the CHMP of the European Medicines Agency.

Once ratified by the European Commission, Upstaza will be the first approved disease-modifying treatment for aromatic L-amino acid decarboxylase (AADC) deficiency for patients 18 months and older and the first marketed gene therapy directly infused into the brain.

“We are thrilled with the positive opinion from the CHMP, and are eager to bring Upstaza to patients living with AADC deficiency,” said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics. “Upstaza will be the first marketed gene therapy that is directly administered into the brain, the first gene therapy approved in a major market in several years, the third gene therapy that is on the market now, and only the fourth in vivo gene therapy ever approved. It’s important for the biotech community to have gene therapy products achieving approvals at regulatory bodies, as well as it being an important milestone for PTC that will help us build the gene therapy franchise and grow our revenue base.”

The CHMP opinion is based on the findings of clinical studies conducted in Taiwan. In addition, data from the compassionate use treatment of patients in Europe were included in the application. In the clinical studies, patients went from no display of any motor milestone development to developing clinically meaningful motor skills and neuromuscular function from as early as three months following treatment, with transformational improvements shown to continue up to nine years after treatment.1 Cognitive and communication skills improved in all treated patients.1,2

“The difference Upstaza, a one-time gene therapy, can make is life-changing,” said Paul Wuh-Liang Hwu, M.D., Ph.D., Lead Investigator, National Taiwan University Hospital. “AADC deficiency is a devastating neurological disorder with no effective treatment. Before therapy, affected children couldn’t even lift their head, but now many can sit, stand with help, feed themselves and some can walk and talk.”

PTC expects the European Commission to ratify the marketing authorization for Upstaza under exceptional circumstances in approximately two months. The decision will be applicable to all 27 European Union member states, as well as Iceland, Norway and Liechtenstein.

About Upstaza™ (eladocagene exuparvovec)
Upstaza, formerly PTC-AADC, is a one-time gene replacement therapy for the treatment of AADC deficiency. It is a recombinant adeno-associated virus serotype 2 (AAV2)-based gene therapy, containing the human DDC gene.1 It is designed to correct the underlying genetic defect, by delivering a functioning DDC gene directly into the putamen, increasing the AADC enzyme and restoring dopamine production.2,3

The efficacy and safety profile of Upstaza has been demonstrated across clinical trials and compassionate use programs.1The first patient was dosed more than 10 years ago. In the clinical trials, Upstaza demonstrated transformational neurological improvements, which have continued for up to nine years following treatment. The most common side effects were initial insomnia, irritability and dyskinesia. The full indication proposed by the CHMP for ratification is: Upstaza is indicated for the treatment of patients aged 18 months and older with a clinical, molecular, and genetically confirmed diagnosis of aromatic L‑amino acid decarboxylase (AADC) deficiency with a severe phenotype.

Administration of Upstaza occurs through a stereotactic surgical procedure, a minimally invasive neurosurgical procedure used for the treatment of a number of pediatric and adult neurological disorders. The Upstaza administration procedure will be performed by a qualified neurosurgeon in a center specialised in stereotactic neurosurgery.

About aromatic L-amino acid decarboxylase (AADC) deficiency
AADC deficiency is a fatal, rare genetic disorder that typically causes severe disability and suffering from the first months of life, affecting every aspect of life – physical, mental and behavioral. The suffering of children with AADC deficiency may be exacerbated by: episodes of distressing seizure-like oculogyric crises, which can happen daily and last for hours, causing the eyes to roll up in the head, frequent vomiting, behavioral problems, difficulty sleeping, and life-threatening complications such as respiratory infections and gastrointestinal problems.

There is no disease-modifying treatment approved for AADC deficiency and the lives of affected children are severely impacted, and shortened, with the use of many different medications to help manage symptoms. Ongoing physical, occupational and speech therapy, and interventions, including surgery, are also often required to manage potentially life-threatening complications such as infections, severe feeding and breathing problems and scoliosis.

About PTC Therapeutics, Inc.
PTC is a science-driven, global biopharmaceutical company focused on the discovery, development and commercialization of clinically differentiated medicines that provide benefits to patients with rare disorders.

PTC’s ability to innovate to identify new therapies and to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines.

PTC’s mission is to provide access to best-in-class treatments for patients who have little to no treatment options.”

https://ir.ptcbio.com/news-releases/news-release-details/ptc-therapeutics-receives-positive-chmp-opinion-upstazatm

FDA Approves First Treatment for Eosinophilic Esophagitis, a Chronic Immune Disorder

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FDA Approves First Treatment for Eosinophilic Esophagitis, a Chronic Immune Disorder

May 20, 2022: “The U.S. FDA approved Dupixent (dupilumab) to treat eosinophilic esophagitis (EoE) in adults and pediatric patients 12 years and older weighing at least 40 kilograms (which is about 88 pounds).

Today’s action marks the first FDA approval of a treatment for EoE.

“As researchers and clinicians have gained knowledge about eosinophilic esophagitis in recent years, more cases of the disorder have been recognized and diagnosed in the U.S.,” said Jessica Lee, M.D., director of the Division of Gastroenterology in the FDA’s Center for Drug Evaluation and Research.

“Today’s approval will fulfill an important unmet need for the increasing number of patients with eosinophilic esophagitis.”

EoE is a chronic inflammatory disorder in which eosinophils, a type of white blood cell, are found in the tissue of the esophagus. In adults and adolescent patients with EoE, common symptoms include difficulty swallowing, difficulty eating, and food getting stuck in the esophagus.

Dupixent is a monoclonal antibody that acts to inhibit part of the inflammatory pathway. 

The efficacy and safety of Dupixent in EoE was studied in a randomized, double-blind, parallel-group, multicenter, placebo-controlled trial, that included two 24-week treatment periods (Part A and Part B) that were conducted independently in separate groups of patients.

In Part A and Part B, patients received either placebo or 300 milligrams of Dupixent every week.

The two primary measurements of efficacy were the proportion of patients who achieved a certain level of reduced eosinophils in the esophagus at week 24, as determined by assessing patients’ esophageal tissue under a microscope, and the change in the patient-reported Dysphagia Symptom Questionnaire (DSQ) score from baseline to week 24.

The DSQ is a questionnaire designed to measure difficulty swallowing associated with EoE, with total scores ranging from 0 to 84; higher DSQ scores indicate worse symptoms. 

In Part A of the trial, 60% of the 42 patients who received Dupixent achieved the pre-determined level of reduced eosinophils in the esophagus compared to 5% of the 39 patients who received a placebo.

Patients in Part A who received Dupixent experienced an average improvement of 22 points in their DSQ score compared to 10 points in patients who received placebo.

In Part B, 59% of the 80 patients who received Dupixent achieved the pre-determined level of reduced eosinophils in the esophagus compared to 6% of the 79 patients who received a placebo.

Patients in Part B who received Dupixent experienced an average improvement of 24 points in their DSQ score compared to 14 points in patients who received placebo.

Assessments incorporating the perspectives from patients with EoE supported that the DSQ score improvement in patients who received Dupixent in the clinical trial was representative of clinically meaningful improvement in dysphagia. 

The most common side effects associated with Dupixent include injection site reactions, upper respiratory tract infections, joint pain, and herpes viral infections.

Dupixent is contraindicated in patients with known hypersensitivity to dupilumab or any of its inactive ingredients.

Dupixent carries warnings and precautions, including ones addressing potential development of allergic reactions, conjunctivitis, keratitis, or joint pain; use in patients with certain parasitic infections; and use in conjunction with live vaccinations. 

Dupixent received priority review and breakthrough therapy designations for this indication. Dupixent was originally approved in 2017.

It is approved for the treatment of moderate-to-severe atopic dermatitis in adult and pediatric patients aged 6 and older whose disease is not adequately controlled by topical prescription therapies or when those therapies are not advisable.

Dupixent is also approved as an add-on maintenance treatment of adults and pediatric patients aged 6 and older with certain types of moderate-to-severe asthma, as well as an add-on maintenance treatment in adults with inadequately controlled chronic rhinosinusitis with nasal polyposis. 

The FDA granted the approval of Dupixent to Regeneron Pharmaceuticals, Inc.”

https://www.fda.gov/news-events/press-announcements/fda-approves-first-treatment-eosinophilic-esophagitis-chronic-immune-disorder
 

AstraZeneca aims to transform cancer care with practice-changing data at ASCO 2022

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AstraZeneca aims to transform cancer care with practice-changing data at ASCO 2022

May 23, 2022: “AstraZeneca advances its ambition to redefine cancer care with new data to be presented across its diverse and industry-leading portfolio of cancer medicines at the American Society of Clinical Oncology (ASCO) Annual Meeting, 3 to 7 June 2022.

A total of 18 approved and potential new medicines from AstraZeneca will be featured across more than 100 abstracts, including nine oral presentations and a plenary presentation of the DESTINY-Breast04 Phase III trial for Enhertu (trastuzumab deruxtecan) in HER2-low metastatic breast cancer.

Dave Fredrickson, Executive Vice President, Oncology Business Unit, AstraZeneca, said: “Our five leading Oncology medicines have each set new standards for patient outcomes across many cancers.

Our data at ASCO will showcase our continued investment in driving innovation with these medicines as well as their long-term impact in real-world settings.

In particular, the groundbreaking data from DESTINY-Breast04 will show the potential of Enhertu to treat patients with HER2-low metastatic breast cancer who have never before been eligible for HER2-targeted treatments.”

Susan Galbraith, Executive Vice President, Oncology R&D, AstraZeneca, said: “At AstraZeneca, we are pioneering new biomarkers and novel therapeutic modalities in our ambition to attack cancer from every angle and deliver personalised medicines to more patients.

The results from DESTINY-Breast04 support the potential for Enhertu to redefine the classification and treatment of breast cancer across the spectrum of HER2 expression.

We are also excited to share promising clinical data for our bispecific PD1-CTLA4 antibody MEDI5752 in advanced renal cell carcinoma, designed to have both of these clinically validated checkpoint targets in one molecule, delivering efficacy with an improved tolerability profile.”

Leading through disruption in breast cancer
A late-breaking plenary presentation will highlight the potentially practice-changing results of the DESTINY-Breast04 trial of Enhertu in patients with HER2-low metastatic breast cancer.

DESTINY-Breast04 is the first-ever Phase III trial of a HER2-directed therapy to show statistically significant and clinically meaningful benefit in both progression-free survival (PFS) and overall survival (OS) in patients with HER2-low unresectable and/or metastatic breast cancer regardless of hormone receptor status compared to standard-of-care chemotherapy.

Additionally, data from a retrospective study will estimate the prevalence of HER2-low breast cancer and describe its clinical and pathological characteristics, to help identify patients with HER2-low expressing tumours who may benefit from HER2-targeted therapy.

Further results will be shared from dose-finding and dose-expansion studies of Enhertu in combination with other anti-cancer agents in patients with advanced or metastatic HER2-positive breast cancer (DESTINY-Breast07) and HER2-low breast cancer (DESTINY-Breast08).

Data will also be presented from a safety follow-up of the DESTINY-Breast03 Phase III trial of Enhertu in the treatment of patients with unresectable or metastatic HER2-positive breast cancer previously treated with trastuzumab and a taxane. 

Enhertu was recently approved in the US for patients in this setting.

Revealing the full potential of an industry-leading portfolio and pipeline
Beyond breast cancer, AstraZeneca will share results from multiple trials highlighting its focus on delivering life-changing cancer medicines for patients with high unmet need.

Data will also support the Company’s commitment to realising the full potential of its leading medicines with ongoing analyses, real-world data and research into novel combinations.

·       MEDI5752 – An oral presentation will share safety and clinical activity results for MEDI5752 in patients with advanced renal cell carcinoma as a monotherapy treatment.

MEDI5752 is a novel bispecific antibody that simultaneously targets the immune checkpoint proteins PD-1 and CTLA-4.

·       Calquence (acalabrutinib) – Updated data from the ELEVATE-TN and ASCEND Phase III trials will highlight long-term safety and efficacy results of Calquence in patients with chronic lymphocytic leukaemia (CLL) regardless of line of therapy.

Presentations include updated data with approximately five-years of median follow-up from the ELEVATE-TN trial, which has demonstrated sustained clinical benefit of Calquence either in combination with obinutuzumab or as monotherapy compared to obinutuzumab plus chlorambucil, providing flexibility to tailor treatment for adults with treatment-naïve CLL.

Additionally, updated results from the ASCEND Phase III trial with approximately four years of median follow-up will highlight the sustained reduction of disease progression or death for Calquence compared to idelalisib plus rituximab or bendamustine plus rituximab in patients with relapsed or refractory CLL, as well as a maintained safety profile.

·       Imfinzi (durvalumab) – Patient-reported outcomes from the HIMALAYA trial will highlight quality of life for patients treated with a single priming dose of tremelimumab added to Imfinzi in 1st-line unresectable liver cancer (STRIDE regimen).

HIMALAYA is the first Phase III trial to show that a dual immunotherapy regimen has improved OS versus sorafenib in this setting.

Tremelimumab with Imfinzi was recently accepted under Priority Review in the US by the Food and Drug Administration (FDA) based on this trial.

Patient-reported outcomes will also be presented from the TOPAZ-1 trial of Imfinzi plus standard-of-care chemotherapy (gemcitabine plus cisplatin) in 1st-line advanced biliary tract cancer.

TOPAZ-1 is the first Phase III trial to show improved survival with an immunotherapy combination versus chemotherapy alone in this setting.

An additional regional subgroup analysis for the TOPAZ-1 trial will compare efficacy outcomes, including OS, for Asian patients with other geographies. 

Imfinzi plus chemotherapy was recently granted Priority Review in the US by the FDA based on this trial.

Further clinically relevant safety data from the positive POSEIDON Phase III trial of Imfinzi, tremelimumab and chemotherapy in 1st-line metastatic non-small cell lung cancer (NSCLC) will also be presented.

·       Lynparza (olaparib) – Data from the PROpel Phase III trial will further reinforce the safety profile of Lynparza plus abiraterone in the treatment of 1st-line metastatic castration-resistant prostate cancer (mCRPC).

These data build on PROpel efficacy data, which demonstrated that this combination significantly delayed disease progression versus standard-of-care abiraterone in 1st-line mCRPC in patients with or without homologous recombination repair gene mutations. 

Lynparza is the first PARP inhibitor to demonstrate clinical benefit in combination with a new hormonal agent versus abiraterone alone in this setting.

·       Tagrisso (osimertinib) – Results will be shared from the externally sponsored OPAL Phase II trial in previously untreated EGFR-mutated (EGFRm) NSCLC that evaluated whether the addition of platinum-based chemotherapy to Tagrisso can improve patient outcomes.

This combination is also being tested in the ongoing FLAURA2 Phase III trial.

Real-world data will also be presented to better inform unmet needs and treatment strategies among patients with resectable early-stage NSCLC, providing valuable insights into EGFRm disease prevalence and rates of recurrence, despite adjuvant chemotherapy, in this population. 

Tagrisso is approved for the adjuvant treatment of early-stage (IB, II and IIIA) EGFRm NSCLC based on the ADAURA Phase III trial.

Collaboration in the scientific community is critical to improving outcomes for patients.

AstraZeneca is collaborating with Daiichi Sankyo Company Limited to develop and commercialise Enhertu and with MSD (Merck & Co., Inc. in the US and Canada) to develop and commercialise Lynparza.

Key AstraZeneca presentations during ASCO 2022

Lead authorAbstract titlePresentation details
Antibody drug conjugates
Modi, STrastuzumab deruxtecan (T-DXd) versus treatment of physician’s choice (TPC) in patients (pts) with HER2-low unresectable and/or metastatic breast cancer (mBC): Results of DESTINY-Breast04, a randomized, phase 3 study.Abstract #LBA3Plenary Session5 June 20222:17pm (CDT) 
Hamilton, EPTrastuzumab deruxtecan (T-DXd) versus trastuzumab emtansine (T-DM1) in patients (pts) with HER2-positive (HER2+) unresectable and/or metastatic breast cancer (mBC): Safety follow-up of the randomized, phase 3 study DESTINY-Breast03.Abstract #1000Oral Abstract Session Breast Cancer—Metastatic4 June 20221:15pm (CDT)
Andre, FDose-finding and -expansion studies of trastuzumab deruxtecan in combination with other anti-cancer agents in patients (pts) with advanced/metastatic HER2+ (DESTINY-Breast07 [DB-07]) and HER2-low (DESTINY-Breast08 [DB-08]) breast cancer (BC).Abstract #3025Poster Session Developmental Therapeutics—Molecularly Targeted Agents and Tumor Biology5 June 20228:00am (CDT)
Immuno-Oncology
Cho, BCDurvalumab (D) +/- tremelimumab (T) + chemotherapy (CT) in first-line (1L) metastatic (m) NSCLC: AE management in POSEIDON.Abstract #9035Poster Session Lung Cancer—Non-Small Cell Metastatic6 June 20228:00am (CDT)
Sangro, BPatient-reported outcomes from the phase 3 HIMALAYA study of tremelimumab plus durvalumab in unresectable hepatocellular carcinoma.Abstract #4074Poster Session Gastrointestinal Cancer—Gastroesophageal, Pancreatic, and Hepatobiliary4 June 20228:00am (CDT)
Burris III, HAPatient-reported outcomes for the phase 3 TOPAZ-1 study of durvalumab plus gemcitabine and cisplatin in advanced biliary tract cancer.Abstract #4070Poster Session Gastrointestinal Cancer—Gastroesophageal, Pancreatic, and Hepatobiliary4 June 20228:00am (CDT)
Vogel, ARegional subgroup analysis of the phase 3 TOPAZ-1 study of durvalumab (D) plus gemcitabine and cisplatin (GC) in advanced biliary tract cancer (BTC).Abstract #4075Poster Session Gastrointestinal Cancer—Gastroesophageal, Pancreatic, and Hepatobiliary4 June 20228:00am (CDT)
Özgüroğlu, MAdverse events self-reported by patients (pts) with extensive-stage small cell lung cancer (ES-SCLC) treated with durvalumab (D) plus platinum-etoposide (EP) or EP in the CASPIAN study.Abstract #8571Poster Session Lung Cancer—Non-Small Cell Local-Regional/Small Cell/Other Thoracic Cancers6 June 20228:00am (CDT)
Albiges, LSafety and clinical activity of MEDI5752, a PD-1/CTLA-4 bispecific checkpoint inhibitor, as monotherapy in patients (pts) with advanced renal cell carcinoma (RCC): Preliminary results from an FTIH trial.Abstract #107Clinical Science Symposium Bispecifics: Are Two Better Than One?5 June 202210:33am (CDT)
DNA damage response
Pignata, SMaintenance olaparib in patients (pts) with platinum-sensitive relapsed ovarian cancer (PSROC) by somatic (s) or germline (g) BRCA and other homologous recombination repair (HRR) gene mutation status: Overall survival (OS) results from the ORZORA study.Abstract #5519Poster Discussion Session Gynecologic Cancer4 June 20224:30pm (CDT) 
Thiery-Vuillemin, ATolerability of abiraterone (abi) combined with olaparib (ola) in patients (pts) with metastatic castration-resistant prostate cancer (mCRPC): Further results from the phase III PROpel trial.Abstract #5019Poster Discussion Session Genitourinary Cancer—Prostate, Testicular, and Penile6 June 20225:26pm (CDT)
Armstrong, AJOlaparib plus abiraterone as first-line therapy in men with metastatic castration-resistant prostate cancer: Pharmacokinetics data from the PROpel trial.Abstract #5050Poster Session Genitourinary Cancer—Prostate, Testicular, and Penile6 June 20221:15pm (CDT)
Eskander, RNReal-world effectiveness of first-line maintenance olaparib in women with BRCA-mutated advanced ovarian cancer: U.S. retrospective cohort study.Abstract #5518Poster Discussion Session Gynecologic Cancer4 June 20224:30pm (CDT)
Tumour drivers and resistance
Jones, RHFulvestrant plus capivasertib versus fulvestrant plus placebo after relapse or progression on an aromatase inhibitor in metastatic, estrogen receptor–positive breast cancer (FAKTION): Overall survival and updated progression-free survival data with enhanced biomarker analysis.Abstract #1005Oral Abstract Session Breast Cancer—Metastatic4 June 20222:39pm (CDT)
Nakamura, AA phase II study of osimertinib in combination with platinum plus pemetrexed in patients with EGFR-mutated, advanced non–small cell lung cancer: The OPAL study (NEJ032C/LOGIK1801).Abstract #9097Poster Session Lung Cancer—Non-Small Cell Metastatic6 June 20228:00am (CDT)
Haematology
Sharman, JPAcalabrutinib ± obinutuzumab versus obinutuzumab + chlorambucil in treatment-naïve chronic lymphocytic leukemia: Five-year follow-up of ELEVATE-TN.Abstract #7539Poster Session Hematologic Malignancies—Lymphoma and Chronic Lymphocytic Leukemia4 June 20228:00am (CDT)

https://www.astrazeneca.com/content/astraz/media-centre/press-releases/2022/astrazeneca-aims-transform-cancer-care-practice-changing-data-asco-2022.html

Vaxzevria approved in EU as third dose booster against COVID-19

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Vaxzevria approved in EU as third dose booster against COVID-19

May 23, 2022: “AstraZeneca’s COVID-19 vaccine, Vaxzevria (ChAdOx1-S [Recombinant]), has been granted approval in the EU by the European Medicine Agency (EMA) as a third dose booster in adults.

Healthcare professionals can now use Vaxzevria as a third dose booster in patients previously given a primary vaccine schedule of either Vaxzevria or an EU-approved mRNA COVID-19 vaccine.

The authorisation is based on a review by the Committee for Medicinal Products for Human Use (CHMP) of the substantial body of evidence demonstrating an increased immune response after a third dose booster with Vaxzevria following a primary vaccine schedule of either Vaxzevria or an mRNA COVID-19 vaccine.

Although more than 65% of the global population has received at least one dose of a COVID-19 vaccine6, there remains a significant challenge to ensure people receive both their primary vaccine schedule and third dose booster, and healthcare professionals now have greater flexibility in their choice of vaccine.

Mene Pangalos, Executive Vice President, BioPharmaceuticals R&D, AstraZeneca said: “Today’s marketing authorisation for AstraZeneca’s COVID-19 vaccine as a third dose booster is an important step towards our goal of providing continued protection against COVID-19 for all populations.

Ensuring a longer duration of immune protection is essential to the long-term management of COVID-19 globally, and boosters can address the waning of protection over time that has been seen with all primary vaccine schedules to date.”

There is a substantial body of evidence supporting Vaxzevria as a third dose booster following all primary vaccination schedules tested to date including Vaxzevria, mRNA vaccines, and CoronaVac.  

Vaxzevria is already authorised as a homologous booster (patients previously given a primary vaccine schedule of Vaxevria) in the UK, and several countries in Asia and Latin America.

It has also been authorised as a heterologous booster (patients previously given a primary vaccine schedule of either a viral vector vaccine other than Vaxzevria or an inactivated vaccine or an mRNA COVID-19 vaccine) in a number of non-EU countries.

Vaxzevria is estimated to have helped prevent 50 million COVID-19 cases, five million hospitalisations, and saved more than one million lives worldwide, based on model outcomes assessing COVID-19 worldwide.”

https://www.astrazeneca.com/content/astraz/media-centre/press-releases/2022/vaxzevria-approved-eu-third-dose-booster-covid-19.html

CHMP recommends approval of Xenpozyme, first & only treatment for ASMD

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CHMP recommends approval of Xenpozyme, first & only treatment for ASMD

May 20, 2022: “The European Medicines Agency’ CHMP has adopted a positive opinion for Xenpozyme® (olipudase alfa), recommending that this investigational enzyme replacement therapy be approved in the European Union for the treatment of non-central nervous system (non-CNS) manifestations of acid sphingomyelinase deficiency (ASMD) in pediatric and adult patients with ASMD type A/B or ASMD type B.

The positive CHMP opinion is based on data from the ASCEND and ASCEND-Peds clinical trials, demonstrating that Xenpozyme showed robust and clinically relevant improvement in lung function (as measured by diffusing capacity of the lung for carbon monoxide, or DLco) and reduced spleen and liver volumes, with a well-tolerated safety profile in adults and children with ASMD.

The EMA previously awarded olipudase alfa the PRIority MEdicines designation, also known as PRIME, and the application was reviewed under the EMA’s accelerated assessment, intended to aid and expedite the regulatory process for investigational medicines that may offer a major therapeutic advantage over existing treatments, or benefit patients without treatment options.

The European Commission will review the CHMP recommendation and is expected to make a final decision in the coming months.

About Xenpozyme

Xenpozyme® (olipudase alfa) is an enzyme replacement therapy designed to replace deficient or defective acid sphingomyelinase (ASM), an enzyme that allows for the breakdown of sphingomyelin.

Accumulation of sphingomyelin in cells can cause harm to the lungs, spleen, and liver, as well as other organs, potentially leading to early death.

Xenpozyme is currently being investigated in pediatric and adult patients to treat non-CNS manifestations of ASMD.

Xenpozyme has not been studied in ASMD type A patients.

In March 2022, Xenpozyme was approved in Japan under the SAKIGAKE (or “pioneer”) designation, marking the first approval for olipudase alfa anywhere in the world.

In the United States, where olipudase alfa received Breakthrough Therapy designation, the FDA has extended its review of the Biologics License Application (BLA) by three months, with a new target action date for the FDA decision (PDUFA date) of October 3, 2022.

https://www.sanofi.com/en/media-room/press-releases/2022/2022-05-20-07-00-00-2447427

FDA Urges Drug Manufacturers to Develop Risk Management Plans to Promote a Stronger, Resilient Drug Supply Chain

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FDA Urges Drug Manufacturers to Develop Risk Management Plans to Promote a Stronger, Resilient Drug Supply Chain

May 19, 2022: “Drug shortages pose a significant public health threat as they can delay, and in some cases, even deny critically needed care for patients.

Over the past decade, the FDA’s efforts have contributed to fewer new drug shortages and reduced the time to resolve existing drug shortages.

This is due, in part, to authorities the agency now has, including those added by the  Food and Drug Administration Safety and Innovation Act. 

But despite mitigating or preventing hundreds of new drug shortages, disruptions in the U.S. drug supply continue to occur due to drug quality problems, vulnerabilities in the global supply chain, unanticipated increases in demand, market withdrawals of drugs or natural disasters.  

In 2019, in an effort to address the national drug shortage problem, the federal Drug Shortages Task Force released a report that called for the adoption of risk management plans to proactively assess risk and to predict and prevent supply disruptions that could potentially lead to a drug shortage.

Then in 2020, Congress passed the CARES Act to require certain manufacturers to develop, maintain, and implement, as appropriate, risk management plans that identify and evaluate risks to a drug’s supply.

To further assist manufacturers with these requirements, we are issuing a draft guidance, Risk Management Plans to Mitigate the Potential for Drug Shortages, intended to help with the development, maintenance and implementation of risk management plans.

The draft guidance describes a framework for stakeholders to consider when developing risk management plans that aligns with principles stated in the International Council for Harmonisation guidance for industry, Q9 Quality Risk Management, and identifies risk factors to consider when developing the content of risk management plans.

The steps needed to reduce risks of a disruption in drug supply may vary among the different manufacturers in the supply chain for a given drug. 

Today’s draft guidance is an important step in what the nation needs to achieve an increasingly secure and resilient drug supply.

For Americans to have access to an uninterrupted supply of safe, effective and high-quality drugs, all entities involved in the manufacture of a drug should take every step available to reduce risks and threats to the drug supply chain.

Risk management plans can serve as a safeguard, helping manufacturers prepare for and respond to hazards that could lead to drug supply disruptions and shortages.”

https://www.fda.gov/news-events/press-announcements/fda-urges-drug-manufacturers-develop-risk-management-plans-promote-stronger-resilient-drug-supply

New FDA Draft Guidance Aims to Increase Safety Information About Dietary Supplement Marketplace

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New FDA Draft Guidance Aims to Increase Safety Information About Dietary Supplement Marketplace

May 19, 2022: “The U.S. FDA announced the availability of a draft guidance aimed at increasing the amount of safety information the agency has about the dietary supplement marketplace by providing the industry an opportunity to submit late new dietary ingredient (NDI) notifications.

The FDA wants consumers who use dietary supplements to know that today’s draft guidance, if finalized, will advise the dietary supplement industry that the agency intends to exercise enforcement discretion, for a limited time and in limited circumstances, to encourage manufacturers and distributors to correct any past failures to submit a required NDI notification.

By providing industry with an opportunity to correct past failures to submit required safety information, the FDA can gain more safety information about the dietary supplement marketplace and better protect public health. 

“We remain committed to a flexible framework for dietary supplements that ensures the safety of these products for consumers,” said Cara Welch, Ph.D., director of the Office of Dietary Supplement Programs in the FDA’s Center for Food Safety and Applied Nutrition (CFSAN).

“The resulting notifications the agency receives through this period of enforcement discretion will help increase the amount of safety information we have about NDI-containing dietary supplements in the marketplace.” 

The Dietary Supplement Health and Education Act of 1994 (DSHEA) requires manufacturers and distributors who wish to market a dietary supplement containing an NDI to notify the FDA before marketing, unless a legal exception applies.

The notification must contain the safety information that a manufacturer relied upon to conclude the dietary supplement containing the NDI is reasonably expected to be safe.  

The NDI notification process is the FDA’s only chance to evaluate the safety of a dietary supplement before it becomes available to consumers.  

For dietary supplements that do not contain an NDI, the law does not require manufacturers to submit safety information to the FDA before marketing.  

The FDA is aware that in the more than 27 years since the requirement was established, some dietary supplement firms have marketed products for which a premarket NDI notification was required, but never submitted.

The enforcement discretion policy proposed in this draft guidance relates solely to the failure to submit an NDI notification.

For example, it would not extend to NDI-containing dietary supplements that are adulterated for safety reasons or that violate any other regulatory requirements that pertain to dietary supplements.

This temporary policy also should help facilitate enforcement actions against those that remain out of compliance with the NDI notification requirements after the enforcement discretion period ends.

In a February 2019 statement about new efforts to strengthen the regulation of dietary supplements through modernization and reform, the FDA emphasized the need to ensure that our regulatory framework is flexible yet comprehensive enough to effectively evaluate product safety, while promoting innovation.

The FDA also stated that fostering the submission of NDI notifications would be key to this effort. This draft guidance is a critical first step toward encouraging those submissions.

If the draft guidance is finalized without change, the enforcement discretion period to submit a late notification would start when the guidance is published, would last 180 days, and would apply only to products on the market when the Federal Register notice announcing the draft guidance was published.

Along with this draft guidance, the FDA is also developing a new submission type through the CFSAN Online Submission Module to provide a dedicated pathway for stakeholders to electronically submit their late notifications.”

https://www.fda.gov/news-events/press-announcements/new-fda-draft-guidance-aims-increase-safety-information-about-dietary-supplement-marketplace

Lilly to Participate in UBS Global Healthcare Conference 2022

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Lilly to Participate in UBS Global Healthcare Conference 2022

May 16, 2022: “Eli Lilly and Company will attend the UBS Global Healthcare Conference on Tuesday, May 24, 2022. Michael Mason, senior vice president, president of Lilly Diabetes, will participate in a fireside chat at 8:30 a.m., Eastern time.

A live audio webcast will be available on the “Webcasts & Presentations” section of Lilly’s Investor website at https://investor.lilly.com/webcasts-and-presentations. A replay of the presentation will be available on this same website for approximately 90 days.”

https://investor.lilly.com/news-releases/news-release-details/lilly-participate-ubs-global-healthcare-conference-2022

FDA Expands Eligibility for Pfizer-BioNTech COVID-19 Vaccine Booster Dose to Children 5 through 11 Years

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FDA Expands Eligibility for Pfizer-BioNTech COVID-19 Vaccine Booster Dose to Children 5 through 11 Years

May 17, 2022: “The U.S. FDA amended the emergency use authorization for the Pfizer-BioNTech COVID-19 Vaccine, authorizing the use of a single booster dose for administration to individuals 5 through 11 years of age at least five months after completion of a primary series with the Pfizer-BioNTech COVID-19 Vaccine. 

“While it has largely been the case that COVID-19 tends to be less severe in children than adults, the omicron wave has seen more kids getting sick with the disease and being hospitalized, and children may also experience longer term effects, even following initially mild disease,” said FDA Commissioner Robert M. Califf, M.D.

“The FDA is authorizing the use of a single booster dose of the Pfizer-BioNTech COVID-19 Vaccine for children 5 through 11 years of age to provide continued protection against COVID-19.

Vaccination continues to be the most effective way to prevent COVID-19 and its severe consequences, and it is safe.

If your child is eligible for the Pfizer-BioNTech COVID-19 Vaccine and has not yet received their primary series, getting them vaccinated can help protect them from the potentially severe consequences that can occur, such as hospitalization and death.”   

On Jan. 3, the FDA authorized the use of a single booster dose of the Pfizer-BioNTech COVID-19 Vaccine for administration to individuals 12 through 15 years of age after completion of primary vaccination with the Pfizer-BioNTech COVID-19 Vaccine.

Today’s action expands the use of a single booster dose of the vaccine for administration to individuals 5 through 11 years age at least five months after completion of a primary series of the Pfizer-BioNTech COVID-19 Vaccine.

The FDA has authorized the Pfizer-BioNTech COVID-19 Vaccine for use in individuals 5 years of age and older and has approved Comirnaty (COVID-19 Vaccine, mRNA) for use in individuals 16 years of age and older.

“The Pfizer-BioNTech COVID-19 Vaccine is effective in helping to prevent the most severe consequences of COVID-19 in individuals 5 years of age and older,” said Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research.

“Since authorizing the vaccine for children down to 5 years of age in October 2021, emerging data suggest that vaccine effectiveness against COVID-19 wanes after the second dose of the vaccine in all authorized populations.

The FDA has determined that the known and potential benefits of a single booster dose of the Pfizer-BioNTech COVID-19 Vaccine for children 5 through 11 years of age at least five months after completing a primary series outweigh its known and potential risks and that a booster dose can help provide continued protection against COVID-19 in this and older age groups.”

Data Supporting Effectiveness

The EUA for a single booster dose of the Pfizer-BioNTech COVID-19 Vaccine for children 5 through 11 years of age is based on FDA’s analysis of immune response data in a subset of children from the ongoing randomized placebo-controlled trial that supported the October 2021 authorization of the Pfizer-BioNTech COVID-19 Vaccine primary series in this age group.

Antibody responses were evaluated in 67 study participants who received a booster dose 7 to 9 months after completing a two-dose primary series of the Pfizer-BioNTech COVID-19 Vaccine.

The antibody level against the SARS-CoV-2 virus one month after the booster dose was increased compared to before the booster dose.

FDA Evaluation of Safety

The safety of a single booster dose of the Pfizer-BioNTech COVID-19 Vaccine in this age group was assessed in approximately 400 children who received a booster dose at least five months (range 5 to 9 months) after completing a two-dose primary series.

The most commonly reported side effects were pain, redness and swelling at the injection site, as well as fatigue, headache, muscle or joint pain and chills and fever.

The FDA did not hold a meeting of its Vaccines and Related Biological Products Advisory Committee on today’s action, as the agency previously convened the committee for extensive discussions regarding the use of booster doses of COVID-19 vaccines and, after review of Pfizer’s EUA request, the FDA concluded that the request did not raise questions that would benefit from additional discussion by committee members.

The FDA will make available on its website relevant documents regarding today’s authorization. 

The amendment to the EUA was granted to Pfizer Inc.”

https://www.fda.gov/news-events/press-announcements/coronavirus-covid-19-update-fda-expands-eligibility-pfizer-biontech-covid-19-vaccine-booster-dose

AstraZeneca signs licence agreement with RQ Biotechnology for monoclonal antibodies against COVID-19

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AstraZeneca signs licence agreement with RQ Biotechnology for monoclonal antibodies against COVID-19

May 17, 2022: “AstraZeneca has entered into a licence agreement with RQ Biotechnology Ltd (RQ Bio) for a portfolio of early-stage monoclonal antibodies (mAbs) targeted against SARS-CoV-2, the virus that causes COVID-19.

Under the agreement, AstraZeneca has acquired an exclusive worldwide licence to develop, manufacture and commercialise mAbs against SARS-CoV-2.

Iskra Reic, Executive Vice President, Vaccines & Immune Therapies, AstraZeneca, said: “The COVID-19 pandemic has changed the landscape for immune therapies, including the use of monoclonal antibodies to protect vulnerable patients who can’t respond adequately to vaccination alone.

Scientific innovation is rapidly accelerating, and this agreement reflects our continued commitment to the discovery and development of new medicines to help prevent and treat infectious disease, including COVID-19.”

RQ Bio is a UK-based biotechnology company focused on developing treatments and preventative therapies based on potent broad-spectrum mAbs to address areas of unmet need in vulnerable patient populations.”

https://www.astrazeneca.com/content/astraz/media-centre/press-releases/2022/astrazeneca-signs-licence-agreement-with-rq-biotechnology-for-monoclonal-antibodies-against-covid-19.html

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