CLINSQUARE: CLINICAL RESEARCH COURSES
Wednesday, February 5, 2025
CLINSQUARE: Clinical Rsearch Courses by LIFEPRONOW
Home Blog Page 2

FDA Facilitates Broader Adoption of Vaporized Hydrogen Peroxide for Medical Device Sterilization

admin
-
0
FDA Facilitates Broader Adoption of Vaporized Hydrogen Peroxide for Medical Device Sterilization

January 08, 2024: “The U.S. Food and Drug Administration is announcing that it considers vaporized hydrogen peroxide (VHP) to be an established method of sterilization for medical devices, recognizing VHP’s long history of safety and effectiveness.

The FDA has revised the final guidance, Submission and Review of Sterility Information in Premarket Notification (510(k)) Submissions for Devices Labeled as Sterile, to list VHP as an example of an Established Category A method of sterilization.

This update will facilitate broader adoption of VHP as a sterilization method for the medical device industry, is part of the agency’s multi-pronged approach to reducing the use of ethylene oxide (EtO) where possible and further supports the agency’s efforts to advance medical device supply chain resiliency. 

Effective sterilization processes are necessary for certain devices to be safe because sterilization inactivates or kills potentially harmful microorganisms.

In addition to effectively inactivating or killing potentially harmful microorganisms, sterilization processes must not damage devices.

For many devices marketed as sterile, a premarket submission must contain information sufficient to show the sterilization process is effective and consistent with internationally accepted consensus standard(s) that the FDA has recognized. 

EtO is the most commonly used sterilization method for medical devices in the U.S., with more than 20 billion devices sold in the U.S. every year sterilized with EtO, accounting for approximately 50% of devices that require sterilization. Since 2019, the FDA has promoted the development of alternatives to EtO and has implemented a number of programs and initiatives to support innovation in medical device sterilization.

This includes developing Sterilization Master File Pilot programs to support certain changes to sterilization processes, launching innovation challenges to encourage new strategies to reduce EtO emissions and the development of new sterilization methods or technologies and proactively engaging with industry to help advance innovative alternatives to EtO. 

“The FDA’s commitment is to protect public health, a critical mission in today’s complex medical device ecosystem,” said Suzanne Schwartz, M.D., M.B.A., director of the Office of Strategic Partnerships and Technology Innovation in the FDA’s Center for Devices and Radiological Health.

“Vaporized hydrogen peroxide’s addition as an established sterilization method helps us build a more resilient supply chain for sterilized devices that can help prevent medical device shortages.

As innovations in sterilization advance, the FDA will continue to seek additional modalities that deliver safe and effective sterilization methods that best protect public health.”

Methods with a long history of safe and effective use on medical devices are considered Established Category A sterilization methods and include moist heat, dry heat, EtO and radiation.

With the recent FDA recognition of the ISO standard 22441:2022, the FDA is adding VHP to Established Category A, which the agency expects will strengthen industry’s capacity to adopt alternative sterilization processes that pose less potential risk to the environment and communities in which they operate. 

The FDA remains committed to reducing adverse impacts to the environment and public health and to developing solutions that avert potential shortages of devices that the American public relies upon.”

https://www.fda.gov/news-events/press-announcements/fda-facilitates-broader-adoption-vaporized-hydrogen-peroxide-medical-device-sterilization

GSK enters exclusive license agreement with Hansoh for HS-20093

admin
-
0
GSK enters exclusive license agreement with Hansoh for HS-20093

December 20, 2023: “GSK plc and Hansoh Pharma, a Chinese biopharmaceutical company committed to discovering and developing life-changing medicines to help patients conquer serious diseases and disorders” announced that they have entered into an exclusive license agreement for HS-20093, a B7-H3 targeted antibody-drug conjugate (ADC) utilising a clinically validated topoisomerase inhibitor (TOPOi) payload.

Under the agreement, GSK will obtain exclusive worldwide rights (excluding China’s mainland, Hong Kong, Macau, and Taiwan) to progress clinical development and commercialisation of HS-20093.

Hesham Abdullah, SVP, Global Head Oncology, R&D, GSK, said: “B7-H3 is highly expressed in a broad range of solid tumours where there remains a significant need for novel treatment options.

We look forward to progressing this potential new treatment across several indications and in future potential combination approaches with our established portfolio.”

This agreement provides GSK with a second clinical-stage ADC that complements GSK’s existing capabilities and strengths in developing medicines to address unmet medical needs in various solid tumours.

HS-20093 is currently being investigated in ongoing phase I and II trials in China.

Data from the ARTEMIS-001 phase I trial (NCT05276609), for HS-20093 in advanced solid tumours, was presented at the 2023 American Society of Clinical Oncology (ASCO) annual meeting in which initial clinical activity was observed in small cell lung cancer, non-small cell lung cancer and sarcoma with multiple confirmed responses and a manageable safety profile.

Eliza Sun, Executive Director of Board, Hansoh Pharma, said: “HS-20093 is a novel B7-H3 targeting antibody-drug conjugate showing encouraging early clinical signals in lung cancer.

We are excited to enter this new license agreement with GSK, our existing licensee on HS-20089, furthering Hansoh’s goal of bringing a potentially transformative treatment option to cancer patients globally.”

GSK plans to begin phase I trials for HS-20093 outside of China in 2024.

In October 2023, GSK and Hansoh entered into an agreement for HS-20089, a B7-H4 targeted ADC currently in phase II clinical trials in China. HS-20089 has best-in-class potential in ovarian and endometrial cancer with opportunities in other solid tumours.

Federal Court Enters Consent Decree Against Pharmasol for Distributing Adulterated Drugs

admin
-
0
Federal Court Enters Consent Decree Against Pharmasol for Distributing Adulterated Drugs

December 14, 2023: “The U.S. District Court for the District of Massachusetts has entered a consent decree of permanent injunction ordering Pharmasol Corporation, a Massachusetts-based company, and President Marc L. Badia to stop distributing drugs until the company complies with the Federal Food, Drug, and Cosmetic (FD&C) Act and other requirements listed in the consent decree.

According to the complaint, which was filed along with the consent decree by the U.S. Department of Justice, Pharmasol and Badia unlawfully distributed adulterated drugs, meaning they do not comply with manufacturing quality requirements within the U.S. marketplace. 

“When drug manufacturers violate the law and disregard safety standards, they put consumers at significant risk.

In this case, the defendant’s company distributed adulterated, poor-quality drugs without regard for patients and consumers,” said Jill Furman, director of the Office of Compliance in the FDA’s Center for Drug Evaluation and Research.

“The FDA plays an important role in protecting consumers, and we will continue to work with our law enforcement partners to pursue and bring into compliance those who do not prioritize the health and safety of the American public.” 

Pharmasol manufactures and distributes over-the-counter drugs, as well as human and animal prescription drugs such as topical corticosteroids and inhalant anesthetics. Pharmasol is under contract with multiple pharmaceutical companies. 

According to the complaint, the defendants violated federal law under the FD&C Act by introducing drugs into interstate commerce that fail to comply with current good manufacturing practice requirements; therefore, these drugs are adulterated. 

The most recent inspection of the company’s facilities in 2022 found the majority of the inspectional observations repeated those found in past FDA inspections and detailed in a 2019 warning letter. Violations mentioned in the complaint include failure to: 

  • fully investigate errors and ensure that the responsibilities and procedures applicable to the quality control unit are in writing and fully followed, including reporting drug defects to customers; 
  • follow written procedures that describe the handling of written and oral complaints regarding a drug product; and, 
  • adequately clean and maintain equipment. 

In the 2019 warning letter, the agency cited customer complaints of product leakage out of the container and the company’s failure to investigate. Despite repeated warnings, the company and Badia continued to violate the law. 

The consent decree prohibits Pharmasol and Badia from directly or indirectly manufacturing, preparing, processing, packing, repacking, receiving, labeling, holding and/or distributing any drug, at or from their facilities, unless and until defendants meet certain requirements to ensure that Pharmasol operates in compliance with the FD&C Act, the FDA’s regulations, and the decree, and defendants receive written notice from the FDA that they appear to be in compliance with these requirements.”

https://www.fda.gov/news-events/press-announcements/federal-court-enters-consent-decree-against-pharmasol-distributing-adulterated-drugs

Navigating the Job Interview: How to Answer the Question, “Why Are You Changing Jobs?”

-
0
Navigating the Job Interview: How to Answer the Question, “Why Are You Changing Jobs?”

Job interviews can be nerve-wracking, but one question that you can almost guarantee will come up is, “Why are you changing jobs?” While it may seem like a straightforward query, crafting a thoughtful and convincing response can significantly impact the outcome of your interview. In this blog post, we’ll explore how to answer this common interview question effectively.

1. Emphasize Career Growth and Learning Opportunities

One of the most positive reasons for changing jobs is to seek new challenges and opportunities for professional development. When an interviewer asks about your reasons for changing jobs, highlighting your desire for growth can be a compelling response.

Here’s a sample answer:

“I’m seeking a new opportunity that will allow me to further develop my skills and take on more challenging responsibilities. While my current job has provided a strong foundation, I believe that a change will expose me to a wider range of experiences and help me grow both personally and professionally. I’m excited about the prospect of learning new things and contributing my expertise to a new team.”

This response not only demonstrates your commitment to self-improvement but also shows that you are proactive in shaping your career.

2. Address Issues Respectfully and Positively

Sometimes, the reasons for changing jobs are less about seeking opportunities and more about addressing challenges or issues in your current position. Whether it’s a toxic work environment, lack of growth prospects, or a company’s values not aligning with yours, it’s crucial to address these concerns in a way that reflects positively on you.

For example:

“I’ve had a great experience with my current employer, but I’ve reached a point where I believe my skills and values could be better utilized elsewhere. I’m looking for a work environment that aligns more closely with my personal values and offers a stronger platform for my professional growth. I believe this change is a natural progression in my career, and I’m excited about the possibilities it presents.”

This response acknowledges the positive aspects of your current job while emphasizing your desire for a better fit elsewhere.

3. Showcase Alignment with the Company’s Values and Goals

Employers appreciate candidates who have thoroughly researched their organization and can demonstrate alignment with the company’s mission, values, and goals. When you’re asked why you’re changing jobs, take the opportunity to show that you’re not just looking for any job but one that aligns with your aspirations and the company’s objectives.

Here’s how you can do it:

“I’ve been impressed by [Company’s Name]’s commitment to innovation and its dedication to making a positive impact in the industry. I’m excited about the opportunity to contribute my skills and experiences to a team that shares my values and vision. This role seems like the perfect fit for my career goals, and I believe it’s a step forward in the right direction for both of us.”

This response illustrates that you’ve done your homework and genuinely believe in the company’s mission, making you a more attractive candidate in the eyes of the interviewer.

In conclusion, the question, “Why are you changing jobs?” is an opportunity to showcase your professionalism, commitment to growth, and alignment with the organization’s values. Crafting a well-thought-out response that focuses on these aspects can help you make a strong impression during your job interview and increase your chances of landing the position you desire.

Bayer extends Partnership with Peking University to foster Pharmaceutical Innovation in China

admin
-
0
Bayer extends Partnership with Peking University to foster Pharmaceutical Innovation in China

July 10, 2023: “Bayer and Peking University (PKU) will collaborate to foster the translation of basic pharmaceutical research into drug discovery and development, while accelerating scientific research on cutting-edge technologies across the pharmaceutical value chain.

The collaboration will focus on selected key areas of interests, such as oncology, cardiorenal, immunology, as well as cell and gene therapy.

Under the agreement, Bayer will provide funding and support for joint research projects, as well as to scientists at PKU in recognition of their research excellence in life sciences and drug innovation.

This new agreement builds on a strategic academic partnership between Bayer and PKU under which a joint research center at Peking University has been founded to advance the translation of drug discovery and research.

“We look forward to a new chapter of the partnership between Bayer and Peking University moving forward,” said Dr. Friedemann Janus, Acting Head of Business Development & Licensing and Open Innovation, Bayer Pharmaceuticals.

“This partnership reflects Bayer’s consistent commitment to China as we see more exciting and disruptive innovation coming from China. We will continue to reinforce our collaboration with Peking University and to explore open innovation models.

These could translate scientific breakthroughs into innovative treatments, thus making a meaningful impact to lives of patients.”

“Peking University has been actively integrating internal and external resources and undertaking the important task of scientific and technological innovation through discipline construction and personnel training.

I look forward to working together with Bayer to promote the development of life sciences and medical technology, to achieve a win-win situation for both academia and industry, and to ultimately benefit patients in China and around the world,” said Hong Wu, Chair Professor of School of Life Sciences in Peking University and Senior Investigator of Peking-Tsinghua Center for Life Sciences.

Academic collaborations have been an integral part of Bayer’s long-standing commitment to fostering drug innovation originated in China and the translation of drug discovery and research. Bayer established strategic collaborations with Peking University and Tsinghua University respectively.

Bayer and the two prestigious universities are carrying out joint research projects, including discovery and structural analysis of novel targets, pathogenesis and drug mechanism of action research, drug screening, efficacy evaluation as well as exploration on new modalities, new synthetic methods, and innovative formulations.

About Bayer’s Collaborate to Cure Hub China
Bayer’s Collaborate to Cure Hub China was established in 2022.

The hub’s team is fully dedicated to developing regional business development strategy and driving key partnering initiatives and deal making, as well as managing strategic alliances covering both commercial and academic collaborations for Bayer Pharmaceuticals in China.

As an integral part of Bayer’s partnering network worldwide, the team is committed to strengthening connection with the China innovation ecosystem and collaborations with local stakeholders and partners, including biopharma, biotech companies and start-ups, as well as universities and research institutions.

Its experts focus on scouting local innovation across the entire value chain of drug innovation, exploring partnering opportunities and facilitating dealmaking.”

https://www.bayer.com/media/en-us/bayer-extends-partnership-with-peking-university-to-foster-pharmaceutical-innovation-in-china/

FDA Approves First Nonprescription Daily Oral Contraceptive

admin
-
0
FDA Approves First Nonprescription Daily Oral Contraceptive

July 13, 2023: “The U.S. Food and Drug Administration approved Opill (norgestrel) tablet for nonprescription use to prevent pregnancy— the first daily oral contraceptive approved for use in the U.S. without a prescription.

Approval of this progestin-only oral contraceptive pill provides an option for consumers to purchase oral contraceptive medicine without a prescription at drug stores, convenience stores and grocery stores, as well as online.  

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

Other approved formulations and dosages of other oral contraceptives will remain available by prescription only. 

“Today’s approval marks the first time a nonprescription daily oral contraceptive will be an available option for millions of people in the United States,” said Patrizia Cavazzoni, M.D., director of the FDA’s Center for Drug Evaluation and Research. “When used as directed, daily oral contraception is safe and is expected to be more effective than currently available nonprescription contraceptive methods in preventing unintended pregnancy.”

Nonprescription availability of Opill may reduce barriers to access by allowing individuals to obtain an oral contraceptive without the need to first see a health care provider. Almost half of the 6.1 million pregnancies in the U.S. each year are unintended. Unintended pregnancies have been linked to negative maternal and perinatal outcomes, including reduced likelihood of receiving early prenatal care and increased risk of preterm delivery, with associated adverse neonatal, developmental and child health outcomes. Availability of nonprescription Opill may help reduce the number of unintended pregnancies and their potential negative impacts.

The contraceptive efficacy of norgestrel was established with the original approval for prescription use in 1973. HRA Pharma applied to switch norgestrel from a prescription to an over-the-counter product. For approval of a product for use in the nonprescription setting, the FDA requires that the applicant demonstrate that the product can be used by consumers safely and effectively, relying only on the nonprescription drug labeling without any assistance from a health care professional. Studies showed that consumer understanding of information on the Opill Drug Facts label was high overall and that a high proportion of consumers understood the label instructions, supporting their ability to properly use the drug when it is available as an over-the-counter product. When properly used, Opill is safe and effective.

Opill should be taken at the same time every day; adherence to daily use at the same time of day is important for the effectiveness of Opill. Using medications that interact with Opill can result in decreased efficacy of Opill or the other medication, or both, potentially resulting in unintended pregnancy. 

The most common side effects of Opill include irregular bleeding, headaches, dizziness, nausea, increased appetite, abdominal pain, cramps or bloating. 

Opill should not be used by those who have or have ever had breast cancer. Consumers who have any other form of cancer should ask a doctor before use. Opill also should not be used together with another hormonal birth control product such as another oral contraceptive tablet, a vaginal ring, a contraceptive patch, a contraceptive implant, a contraceptive injection or an IUD (intra-uterine device).

Use of Opill may be associated with changes in vaginal bleeding patterns, such as irregular spotting and prolonged bleeding. Consumers should inform a health care provider if they develop repeated vaginal bleeding after sex, or prolonged episodes of bleeding or amenorrhea (absence of menstrual period). Individuals who miss two periods (or have missed a single period and have missed doses of Opill) or suspect they may be pregnant should take a pregnancy test. Consumers should discontinue Opill if pregnancy is confirmed. 

Opill is not for use as emergency contraception and does not prevent pregnancy after unprotected sex.

Oral contraceptives do not protect against transmission of HIV, AIDS and other sexually transmitted diseases such as chlamydia, genital herpes, genital warts, gonorrhea, hepatitis B and syphilis. Condoms should be used to prevent sexually transmitted diseases.

The FDA granted the approval to Laboratoire HRA Pharma, recently acquired by Perrigo Company plc.”

https://www.fda.gov/news-events/press-announcements/fda-approves-first-nonprescription-daily-oral-contraceptive

Enhertu approved in China for patients with HER2-low metastatic breast cancer

admin
-
0
Enhertu approved in China for patients with HER2-low metastatic breast cancer

July 12, 2023: “AstraZeneca and Daiichi Sankyo’s Enhertu (trastuzumab deruxtecan) has been approved in China as a monotherapy for the treatment of adult patients with unresectable or metastatic HER2-low (IHC 1+ or IHC 2+/ISH-) breast cancer who have received a prior systemic therapy in the metastatic setting or developed disease recurrence during or within six months of completing adjuvant chemotherapy.

Enhertu is a specifically engineered HER2-directed antibody drug conjugate (ADC) being jointly developed and commercialised by AstraZeneca and Daiichi Sankyo.

The approval by China’s National Medical Products Administration (NMPA) is based on the results of the DESTINY-Breast04 Phase III trial, first presented at the American Society of Clinical Oncology 2022 Annual Meeting and published in The New England Journal of Medicine.

It follows the approval granted by China’s NMPA for Enhertu in patients with previously treated unresectable or metastatic HER2-positive breast cancer in February 2023.

In China, breast cancer is the most common cancer in women, with more than 415,000 patients diagnosed in 2020.2 There were nearly 120,000 breast cancer deaths in China in 2020, representing around 18% of global breast cancer deaths.

Approximately half of all breast cancers are considered HER2-low.

Binghe Xu, MD, Director of the National Clinical Research Center for New Anticancer Drugs, Tenured Professor and Former Director, Department of Medical Oncology, Cancer Hospital Chinese Academy of Medical Sciences and Peking Union Medical College, said: “Historically, breast cancer tumours with low levels of HER2 expression have been classified as HER2-negative and have not been eligible for treatment with HER2-directed therapies.

With this approval in China, based on the results of the DESTINY-Breast04 trial, clinicians will now be able to identify and potentially treat a distinct patient population based on HER2-low status.”

Dave Fredrickson, Executive Vice President, Oncology Business Unit, AstraZeneca, said: “Patients with HR-positive or HR-negative, HER2-low metastatic breast cancer previously had few effective treatment options beyond chemotherapy.

The results from the DESTINY-Breast04 trial show Enhertu provides a significant improvement in outcomes compared to chemotherapy for patients whose tumours are determined to be HER2-low via routine testing.

This approval is an important advance in the way breast cancer is classified and treated in China and supports our vision to bring Enhertu to more patients worldwide.”

Kiminori Nagao, Head of the Asia, South & Central America (ASCA) Business Unit, Daiichi Sankyo, said: “This approval of Enhertu for patients with HER2-low metastatic breast cancer, which comes shortly after the approval of Enhertu in patients with HER2-positive disease, marks the first time patients with HER2-low tumours will have the opportunity to be treated with a HER2-directed therapy. 

Enhertu now has the potential to become a new standard of care treatment option in China for a broad range of patients with HER2-expressing metastatic breast cancer.”

In DESTINY-Breast04, Enhertu reduced the risk of disease progression or death by 50% versus physician’s choice of chemotherapy (median progression-free survival [PFS] 9.9 vs. 5.1 months; hazard ratio [HR] of 0.50; 95% confidence interval [CI] 0.40-0.63; p<0.0001) in all randomised patients with HER2-low metastatic breast cancer (either hormone receptor (HR)-positive or HR-negative disease).

A 36% reduction in the risk of death (HR of 0.64; 95% CI 0.49-0.84; p=0.001) also was seen with Enhertu compared to chemotherapy with a median overall survival (OS) of 23.4 months in patients treated with Enhertu versus 16.8 months in those treated with chemotherapy.

The safety profile observed in patients treated with Enhertu in the DESTINY-Breast04 trial was consistent with that seen in other trials of Enhertu in breast cancer with no new safety signals identified.”

https://www.astrazeneca.com/media-centre/press-releases/2023/enhertu-approved-in-china-as-the-first-her2-directed-therapy-for-patients-with-her2-low-metastatic-breast-cancer.html

FDA Approves First Drug to Treat Agitation Symptoms Associated with Dementia due to Alzheimer’s Disease

admin
-
0
FDA Approves First Drug to Treat Agitation Symptoms Associated with Dementia due to Alzheimer’s Disease

May 11, 2023: “The U.S. FDA is announcing the supplemental approval of Rexulti (brexpiprazole) oral tablets for the treatment of agitation associated with dementia due to Alzheimer’s disease.

This is the first FDA-approved treatment option for this indication.

“Agitation is one of the most common and challenging aspects of care among patients with dementia due to Alzheimer’s disease.

‘Agitation’ can include symptoms ranging from pacing or restlessness to verbal and physical aggression,” said Tiffany Farchione, M.D., director of the Division of Psychiatry in the FDA’s Center for Drug Evaluation and Research.

“These symptoms are leading causes of assisted living or nursing home placement and have been associated with accelerated disease progression.”

Dementia is a serious and debilitating neurological condition characterized by progressive decline in one or more cognitive domains in the brain.

Dementia can seriously impair an individual’s ability to function independently. Many people living with dementia require permanent at-home or residential care. 

Alzheimer’s disease is the most common cause of dementia. Alzheimer’s disease is an irreversible, progressive brain disorder affecting more than 6.5 million Americans.

Patients with dementia often have behavioral and psychological disturbances. Agitation is among the most persistent, complex, stressful, and costly aspects of care among patients with behavioral and psychological symptoms of dementia.

The effectiveness of Rexulti for the treatment of agitation associated with dementia due to Alzheimer’s disease was determined through two 12-week, randomized, double-blind, placebo-controlled, fixed-dose studies.

In these studies, patients were required to have a probable diagnosis of Alzheimer’s dementia; have a score between 5 to 22 on the Mini-Mental State Examination, a test that detects whether a person is experiencing cognitive impairment; and exhibit the type, frequency, and severity of agitation behaviors that require medication.

Trial participants ranged between 51 to 90 years of age. 

In the first study patients received 1 or 2 milligrams (mg) of Rexulti; in the second study patients received 2 or 3 mg of Rexulti.

The primary efficacy endpoint in these two studies was the change from baseline in the Cohen-Mansfield Agitation Inventory total (CMAI) score at week 12. CMAI is a survey tool that uses input from caregivers to rate the frequency of certain agitative behaviors in dementia patients on a scale from 1 to 7.

In both studies, patients who received 2 mg or 3 mg of Rexulti showed statistically significant and clinically meaningful improvements in total CMAI scores compared to patients in the placebo group at week 12.

The recommended starting dosage for the treatment of agitation associated with dementia due to Alzheimer’s disease is 0.5 mg taken once daily on days 1 to 7. Patients should increase the dosage on days 8 through 14 to 1 mg once daily, and on day 15 to 2 mg once daily. The recommended target dose is 2 mg once daily.

The dosage can be increased to the maximum recommended daily dosage of 3 mg once daily after at least 14 days, based on clinical response and tolerability.

The most common side effects among patients with agitation associated with dementia due to Alzheimer’s disease include headache, dizziness, urinary tract infection, nasopharyngitis, and sleep disturbances (both somnolence and insomnia).

Rexulti will retain the Boxed Warning for medications in this class that elderly patients with dementia-related psychosis treated with antipsychotic drugs are at an increased risk of death.

The FDA granted this application Fast Track designation. 

Fast Track is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need.

The purpose is to get important new drugs to the patient earlier.

The supplemental approval of Rexulti was granted to Otsuka Pharmaceutical Company Ltd., and Lundbeck Inc.”

https://www.fda.gov/news-events/press-announcements/fda-approves-first-drug-treat-agitation-symptoms-associated-dementia-due-alzheimers-disease

FDA Approves Novel Drug to Treat Moderate to Severe Hot Flashes Caused by Menopause

admin
-
0
FDA Approves Novel Drug to Treat Moderate to Severe Hot Flashes Caused by Menopause

May 12, 2023: “The U.S. FDA approved Veozah (fezolinetant), an oral medication for the treatment of moderate to severe vasomotor symptoms, or hot flashes, caused by menopause.

Veozah is the first neurokinin 3 (NK3) receptor antagonist approved by the FDA to treat moderate to severe hot flashes from menopause.

It works by binding to and blocking the activities of the NK3 receptor, which plays a role in the brain’s regulation of body temperature.

“Hot flashes as a result of menopause can be a serious physical burden on women and impact their quality of life,” said Janet Maynard, M.D., M.H.S., director of the Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine, in the FDA’s Center for Drug Evaluation and Research.

“The introduction of a new molecule to treat moderate to severe menopausal hot flashes will provide an additional safe and effective treatment option for women.”

Menopause is a normal, natural change in a woman’s life when her period stops, usually occurring between ages 45 and 55. Menopause is often referred to as “the change of life” or “the change.” During menopause a woman’s body slowly produces less of the hormones estrogen and progesterone.

A woman has reached menopause when she has not had a menstrual period for 12 consecutive months. Hot flashes occur in around 80% of menopausal women and can include periods of sweating, flushing and chills lasting for several minutes.

Some women who experience hot flashes and have a history of vaginal bleeding, stroke, heart attack, blood clots or liver disease, cannot take hormone therapies. Veozah is not a hormone.

It targets the neural activity which causes hot flashes during menopause. 

Patients taking Veozah should take one 45 milligram pill orally, once a day, with or without food. The pill should be taken at the same time each day.

If a dose is missed, or not taken at the regular time, patients should take it as soon as possible and return to their regular schedule the following day.

The effectiveness of Veozah to treat moderate to severe hot flashes was demonstrated in each of the first 12-week, randomized, placebo-controlled, double-blind portions of two phase 3 clinical trials.

In both trials, after the first 12 weeks, the women on placebo were then re-randomized to Veozah for a 40-week extension study to evaluate safety. Each trial ran a total of 52 weeks. The average age of the trial participants was 54 years old.

The prescribing information for Veozah includes a warning for elevated hepatic transaminase, or liver injury. Before using Veozah, patients should have blood work done to test for liver damage.

While on Veozah, routine bloodwork should be performed every three months for the first nine months of using the medication.

Patients experiencing symptoms related to liver damage—such as nausea, vomiting, or yellowing of the skin and eyes—should contact a physician.

Veozah cannot be used with CYP1A2 inhibitors. Patients with known cirrhosis, severe renal damage or end-stage renal disease should not take Veozah. 

The most common side effects of Veozah include abdominal pain, diarrhea, insomnia, back pain, hot flush and elevated hepatic transaminases. 

The FDA granted the Veozah application Priority Review designation.

The approval of Veozah was granted to Astellas Pharma US, Inc.”

https://www.fda.gov/news-events/press-announcements/fda-approves-novel-drug-treat-moderate-severe-hot-flashes-caused-menopause

Farxiga extended in US to reduce risk of cardiovascular death and hospitalisation for heart failure to a broader range of patients

admin
-
0
Farxiga extended in US to reduce risk of cardiovascular death and hospitalisation for heart failure to a broader range of patients

May 09, 2023: “AstraZeneca’s Farxiga (dapagliflozin) has been approved in the US to reduce the risk of cardiovascular (CV) death, hospitalisation for heart failure (hHF) and urgent heart failure (HF) visits in adults with HF.

The approval by the Food and Drug Administration (FDA) was based on positive results from the DELIVER Phase III trial.1 Farxiga was previously approved in the US for adults with HF with reduced ejection fraction (HFrEF).

Ruud Dobber, Executive Vice-President, BioPharmaceuticals Business Unit, AstraZeneca, said: “Approximately half of heart failure patients die within five years of diagnosis, highlighting an urgent unmet need for well-tolerated treatment options that can bring life-saving benefits and reduce the risk of cardiovascular death.

The approval of Farxiga in the US not only reinforces AstraZeneca’s commitment to reducing the burden of this complex and life-threatening disease, but will help patients across the full spectrum of heart failure lead healthier lives.”

HF is a chronic, long-term condition that worsens over time and affects almost seven million people in the US.3 It is also the leading cause of hospitalisation for those over 65 years and represents a significant clinical and economic burden.

Approximately half of all HF patients have HF with mildly reduced ejection fraction (HFmrEF) or HF with preserved ejection fraction (HFpEF) and these patients are not only at greater risk of death and hospitalisations but experience an especially high burden of symptoms and physical limitations, and a poor quality of life.

The data from the DELIVER Phase III trial, published in The New England Journal of Medicine, showed Farxiga reached a statistically significant and clinically meaningful early reduction in the primary composite endpoint of CV death or worsening HF in patients with HF with HFmrEF or HFpE.F1 The results from a pre-specified, pooled analysis of the DAPA-HF and DELIVER Phase III trials, published in Nature Medicine, showed that the treatment effect of Farxiga on the composite endpoint of cardiovascular death, hospitalisation for heart failure or urgent heart failure was consistent across the left ventricular ejection fraction (LVEF)  range and established Farxiga as the first sodium-glucose cotransporter 2 (SGLT2) inhibitor to demonstrate a mortality benefit.

Farxiga is approved for the treatment of patients with type-2 diabetes (T2D), HFrEF and chronic kidney disease (CKD) in more than 100 countries around the world including the US, the European Union (EU), China and Japan.

It has most recently received regulatory approvals in the EU, Great Britain, Japan and Turkey to extend the HF indication to include patients across the full LVEF range.”

https://www.astrazeneca.com/media-centre/press-releases/2023/farxiga-extended-in-the-us-for-heart-failure.html

BMS Receives EC Approval for CAR T Cell Therapy Breyanzi for Relapsed or Refractory Large B-cell Lymphoma After One Prior Therapy

admin
-
0
BMS Receives EC Approval for CAR T Cell Therapy Breyanzi for Relapsed or Refractory Large B-cell Lymphoma After One Prior Therapy

May 05, 2023: “Bristol Myers Squibb announced that the European Commission (EC) has granted approval for Breyanzi (lisocabtagene maraleucel; liso-cel), a CD19-directed chimeric antigen receptor (CAR) T cell therapy, for the treatment of adult patients with diffuse large B-cell lymphoma (DLBCL), high grade B-cell lymphoma (HGBCL), primary mediastinal large B-cell lymphoma (PMBCL) and follicular lymphoma grade 3B (FL3B), who relapsed within 12 months from completion of, or are refractory to, first-line chemoimmunotherapy.

This approval covers all European Union (EU) member states.*

The approval is based on results from the pivotal Phase 3 TRANSFORM trial in which Breyanzi demonstrated statistically significant and clinically meaningful improvements in the study’s primary endpoint of event-free survival (EFS), and key secondary endpoints of complete responses (CR) and progression-free survival (PFS) compared to standard therapy (consisting of salvage immunochemotherapy followed by high-dose chemotherapy and hematopoietic stem cell transplant [HSCT]), along with a manageable and well-established safety profile.

“With Breyanzi, people in Europe living with relapsed or refractory DLBCL now have a differentiated CAR T cell therapy option earlier in the treatment paradigm that provides long-term clinical benefit,” said Anne Kerber, senior vice president, head of Cell Therapy Development, Bristol Myers Squibb.

“This marks the approval of our third indication in Europe for our CAR T cell therapy portfolio, underscoring our continued drive to deliver the promise of cell therapy with curative potential for more patients.”

In DLBCL, the most common form of non-Hodgkin lymphoma, up to 40% of patients have disease that is refractory to or relapses following initial therapy.

The standard therapy for these patients consists of intensive salvage immunochemotherapy followed by high-dose chemotherapy and HSCT for those whose disease responds to the salvage therapy and are eligible for transplant.

However, only an estimated 25% of patients are considered eligible for transplant and experience long-term clinical benefit, leaving a continued unmet need for second-line treatment options with curative potential.

“Based on results of the TRANSFORM trial, Breyanzi provides significantly improved outcomes compared to the standard of care that has been in place for decades, along with a well-established safety profile, demonstrating the benefit of using a CAR T cell therapy earlier for patients with relapsed or refractory DLBCL,” said Bertram Glass, M.D., TRANSFORM trial investigator and Chief Physician of the Department of Hematology and Stem Cell Transplantation, Helios Klinikum, Berlin, Germany.

“This approval represents a significant milestone for patients with continued progress toward transforming second-line treatment practice to provide a personalized treatment option that offers the potential for durable remission.”

*Centralized Marketing Authorization does not include approval in Great Britain (England, Scotland and Wales).

TRANSFORM Clinical Trial Results

In the TRANSFORM study, Breyanzi more than quadrupled median EFS compared to standard therapy (10.1 months vs. 2.3 months [HR: 0.349; 95% CI (0.229-0.530) p<0.0001]) at the time of prespecified interim analysis with a median follow-up of 6.2 months.

Results of the primary analysis, with a median follow-up of 17.5 months were consistent with the interim analysis, with median EFS not reached for Breyanzi (95% CI: 9.5-NR) vs. 2.4 months for standard therapy (95% CI: 2.2-4.9).

With Breyanzi, the majority (73.9%) of patients achieved a CR compared to less than half (43.5%) of those who were treated with standard therapy.

Median PFS was not reached (95% CI: 12.6-NR) with Breyanzi vs. 6.2 months (95% CI: 4.3-8.6) with standard therapy (HR: 0.400; 95% CI: 0.261-0.615; p<0.0001).

The safety profile of Breyanzi is well-established, and in the TRANSFORM study, occurrences of cytokine release syndrome (CRS) and neurologic events were generally low-grade, and mostly resolved quickly with standard protocols and without the use of prophylactic steroids. Any-grade CRS was reported in less than half of patients (48.9%), with Grade 3 CRS reported in 1% of patients.

The median time to onset of CRS was five days (range: 1 to 63) and median duration of CRS was four days (range: 1 to 16). Any-grade neurologic events were reported in 10.9% of patients treated with Breyanzi, with Grade 3 neurologic events reported in 4.3% of patients.

The median time to onset of neurologic events was 11 days (range: 7 to 17 days). The median duration of neurologic toxicities was 4.5 days (range: 1 to 30 days).

For full details on the Special Warnings and Precautions for Use and Adverse Reactions (including appropriate management), please refer to the EU Summary of Product Characteristics (SmPC).

About TRANSFORM

TRANSFORM (NCT03575351) is a pivotal, global, randomized, multicenter Phase 3 trial evaluating Breyanzi compared to current standard of care (platinum-based salvage chemotherapy followed by high-dose chemotherapy and autologous hematopietic stem cell transplant [HSCT] in patients responding to salvage chemotherapy) in patients with large B-cell lymphoma that was primary refractory or relapsed within 12 months after CD20-antibody and anthracycline containing first-line therapy.

Patients were randomized to receive Breyanzi or standard of care salvage therapy, including rituximab plus dexamethasone, high-dose cytarabine, and cisplatin (R-DHAP), rituximab plus ifosfamide, carboplatin and etoposide (R-ICE), or rituximab plus gemcitabine, dexamethasone and cisplatin (R-GDP) per the investigators’ choice before proceeding to high-dose chemotherapy (HDCT) and auto-HSCT.

The primary endpoint of the study was event-free survival, defined as time from randomization to death from any cause, progressive disease, failure to achieve complete response or partial response, or start of new antineoplastic therapy due to efficacy concerns, whichever occurs first.

Complete response rate was a key secondary endpoint. Other efficacy endpoints included progression-free survival, overall survival, overall response rate and duration of response.

About Breyanzi

Breyanzi is a CD19-directed CAR T cell therapy with a 4-1BB costimulatory domain, which enhances the expansion and persistence of the CAR T cells. 

Breyanzi is approved by the U.S. Food and Drug Administration (FDA) for the treatment of adult patients with large B-cell lymphoma (LBCL), including diffuse large B-cell lymphoma (DLBCL) not otherwise specified (including DLBCL arising from indolent lymphoma), high-grade B-cell lymphoma, primary mediastinal LBCL, and follicular lymphoma grade 3B who have refractory disease to first-line chemoimmunotherapy or relapse within 12 months of first-line chemoimmunotherapy, or refractory disease to first-line chemoimmunotherapy or relapse after first-line chemoimmunotherapy and are not eligible for hematopoietic stem cell transplant due to comorbidities or age, or relapsed or refractory disease after two or more lines of systemic therapy. 

Breyanzi is not indicated for the treatment of patients with primary central nervous system lymphoma.

Please see the Important Safety Information section below, including Boxed WARNINGS for Breyanzi regarding cytokine release syndrome and neurotoxicity.

Breyanzi is also approved in Japan for the second-line treatment of relapsed or refractory LBCL, and in Japan, Europe, Switzerland and Canada for relapsed or refractory LBCL after two or more lines of systemic therapy.

Bristol Myers Squibb’s clinical development program for Breyanzi includes clinical studies in earlier lines of treatment for patients with relapsed or refractory LBCL and other types of lymphomas and leukemia. For more information, visit clinicaltrials.gov.

Full European Summary of Product Characteristics for Breyanzi is available from the EMA website at www.ema.europa.eu .

U.S. Important Safety Information

BOXED WARNING: CYTOKINE RELEASE SYNDROME and NEUROLOGIC TOXICITIES

  • Cytokine Release Syndrome (CRS), including fatal or life-threatening reactions, occurred in patients receiving BREYANZI. Do not administer BREYANZI to patients with active infection or inflammatory disorders. Treat severe or life-threatening CRS with tocilizumab with or without corticosteroids.
  • Neurologic toxicities, including fatal or life-threatening reactions, occurred in patients receiving BREYANZI, including concurrently with CRS, after CRS resolution or in the absence of CRS. Monitor for neurologic events after treatment with BREYANZI. Provide supportive care and/or corticosteroids as needed.
  • BREYANZI is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the BREYANZI REMS.

Cytokine Release Syndrome (CRS)

CRS, including fatal or life-threatening reactions, occurred following treatment with BREYANZI. CRS occurred in 46% (122/268) of patients receiving BREYANZI, including ≥ Grade 3 (Lee grading system) CRS in 4% (11/268) of patients. One patient had fatal CRS and 2 had ongoing CRS at time of death. The median time to onset was 5 days (range: 1 to 15 days).

CRS resolved in 119 of 122 patients (98%) with a median duration of 5 days (range: 1 to 17 days). Median duration of CRS was 5 days (range 1 to 30 days) in all patients, including those who died or had CRS ongoing at time of death.

Among patients with CRS, the most common manifestations of CRS include fever (93%), hypotension (49%), tachycardia (39%), chills (28%), and hypoxia (21%). 

Serious events that may be associated with CRS include cardiac arrhythmias (including atrial fibrillation and ventricular tachycardia), cardiac arrest, cardiac failure, diffuse alveolar damage, renal insufficiency, capillary leak syndrome, hypotension, hypoxia, and hemophagocytic lymphohistiocytosis/macrophage activation syndrome (HLH/MAS).

Ensure that 2 doses of tocilizumab are available prior to infusion of BREYANZI. Sixty-one of 268 (23%) patients received tocilizumab and/or a corticosteroid for CRS after infusion of BREYANZI.

Twenty-seven (10%) patients received tocilizumab only, 25 (9%) received tocilizumab and a corticosteroid, and 9 (3%) received corticosteroids only.

Neurologic Toxicities

Neurologic toxicities that were fatal or life-threatening, occurred following treatment with BREYANZI.

CAR T cell-associated neurologic toxicities occurred in 35% (95/268) of patients receiving BREYANZI, including ≥ Grade 3 in 12% (31/268) of patients.

Three patients had fatal neurologic toxicity and 7 had ongoing neurologic toxicity at time of death.

The median time to onset of the first event was 8 days (range: 1 to 46 days). The onset of all neurologic events occurred within the first 8 weeks following BREYANZI infusion.

Neurologic toxicities resolved in 81 of 95 patients (85%) with a median duration of 12 days (range: 1 to 87 days).

Three of four patients with ongoing neurologic toxicity at data cutoff had tremor and one subject had encephalopathy.

Median duration of neurologic toxicity was 15 days (range: 1 to 785 days) in all patients, including those with ongoing neurologic events at the time of death or at data cutoff.

Seventy-eight (78) of 95 (82%) patients with neurologic toxicity experienced CRS. Neurologic toxicity overlapped with CRS in 57 patients.

The onset of neurologic toxicity was after onset of CRS in 30 patients, before CRS onset in 13 patients, same day as CRS onset in 7 patients, and same day as CRS resolution in 7 patients. Neurologic toxicity resolved in three patients before the onset of CRS. Eighteen patients experienced neurologic toxicity after resolution of CRS.

The most common neurologic toxicities included encephalopathy (24%), tremor (14%), aphasia (9%), delirium (7%), headache (7%), dizziness (6%), and ataxia (6%).

Serious events including cerebral edema and seizures occurred with BREYANZI. Fatal and serious cases of leukoencephalopathy, some attributable to fludarabine, have occurred in patients treated with BREYANZI.

CRS and Neurologic Toxicities Monitoring

Monitor patients daily at a certified healthcare facility during the first week following infusion, for signs and symptoms of CRS and neurologic toxicities.

Monitor patients for signs and symptoms of CRS and neurologic toxicities for at least 4 weeks after infusion; evaluate and treat promptly. Counsel patients to seek immediate medical attention should signs or symptoms of CRS or neurologic toxicity occur at any time.

At the first sign of CRS, institute treatment with supportive care, tocilizumab or tocilizumab and corticosteroids as indicated.”

https://news.bms.com/news/corporate-financial/2023/Bristol-Myers-Squibb-Receives-European-Commission-Approval-for-CAR-T-Cell-Therapy-Breyanzi-lisocabtagene-maraleucel-for-Relapsed-or-Refractory-Large-B-cell-Lymphoma-After-One-Prior-Therapy/default.aspx

Koselugo approved in China for paediatric patients with neurofibromatosis type 1 and plexiform neurofibromas

admin
-
0
Koselugo approved in China for paediatric patients with neurofibromatosis type 1 and plexiform neurofibromas

May 8, 2023: “Koselugo (selumetinib) has been approved in China for the treatment of symptomatic, inoperable plexiform neurofibromas (PN) in paediatric patients with neurofibromatosis type 1 (NF1) aged three years and above.

The approval by the National Medical Products Administration (NMPA) in China was based on positive results from the SPRINT Stratum 1 trial sponsored by the National Institutes of Health’s National Cancer Institute (NCI) Cancer Therapy Evaluation Program (CTEP).

The trial showed Koselugo, an oral treatment option, reduced the size of inoperable tumours in children.

NF1 is a rare, progressive genetic condition affecting one in 3,000 individuals worldwide, most commonly diagnosed in children under the age of 10.

In 30-50% of patients, tumours develop on the nerve sheaths (plexiform neurofibromas) and cause clinical issues such as disfigurement, motor dysfunction, pain, airway dysfunction, visual impairment and bladder or bowel dysfunction.

Li Qingfeng, Vice President of Shanghai Ninth People’s Hospital, JiaoTong University School of Medicine, Chairman of Department of Plastic and Reconstructive Surgery, said: “Children living with NF1 PN may face physical challenges and significant disruption to their daily lives, and early intervention is essential as tumour growth is often progressive and rapid, especially in the first decade of life.

The approval of Koselugo in China has the potential to change the treatment trajectory of this debilitating genetic condition, providing a new option for addressing inoperable tumours by targeting the underlying cause of tumour growth through MEK inhibition.”

Marc Dunoyer, Chief Executive Officer, Alexion, said: “Koselugo offers hope for children whose quality of life and overall well-being is impacted by the growth of painful, debilitating tumours throughout the body.

We are proud to bring forward a new, innovative treatment option to the NF1 community in China, delivering on Alexion’s commitment to reach more people living with rare diseases in China, and transform the lives of patients and caregivers across the globe.”

Results from the SPRINT Stratum 1 Phase II trial were published in The New England Journal of Medicine and showed that Koselugo demonstrated an objective response rate (ORR) of 66% (33 of 50 patients, confirmed partial responses) in paediatric patients with PNs in NF1 when treated with Koselugo as twice-daily oral monotherapy.

ORR is defined as the percentage of patients with confirmed complete (disappearance of PNs) or partial response (at least 20% reduction in tumour volume).

The most common adverse reactions in the SPRINT trial were vomiting, blood creatine phosphokinase increase, diarrhoea and nausea.

In addition to China, Koselugo is also approved in the US, EU, Japan and several other countries for the treatment of paediatric patients with NF1 and symptomatic, inoperable PNs.

Notes

NF1
NF1 is a debilitating genetic condition that is caused by a spontaneous or inherited mutation in the NF1 gene.

NF1 is associated with a variety of symptoms, including soft lumps on and under the skin (cutaneous neurofibromas) and skin pigmentation (so-called ‘café au lait’ spots) and, in 30-50% of patients, tumours develop on the nerve sheaths (plexiform neurofibromas).

These plexiform neurofibromas (PNs) can cause clinical issues such as disfigurement, motor dysfunction, pain, airway dysfunction, visual impairment and bladder or bowel dysfunction.

PNs begin during early childhood, with varying degrees of severity, and can reduce life expectancy by up to 15 years.

SPRINT
The SPRINT Stratum 1 Phase II trial was designed to evaluate the objective response rate and impact on patient-reported and functional outcomes in paediatric patients with NF1-related inoperable PNs treated with Koselugo (selumetinib) monotherapy.

This trial sponsored by NCI CTEP was conducted under a Cooperative Research and Development Agreement between NCI and AstraZeneca with additional support from Neurofibromatosis Therapeutic Acceleration Program (NTAP).

Koselugo
Koselugo (selumetinib) is the first and only approved therapy by China’s NMPA for the treatment of symptomatic, inoperable PNs in paediatric patients with NF1 aged three years and above.

Koselugo blocks specific enzymes (MEK1 and MEK2), which are involved in stimulating cells to grow.

In NF1, these enzymes are overactive, causing tumour cells to grow in an unregulated way. By blocking these enzymes, Koselugo slows down the growth of tumour cells.

Koselugo is approved for use in the US, EU, Japan and China and has received Orphan Drug Designation in the US, EU, Russia, Switzerland, South Korea, Taiwan, Japan and Australia, and health authorities worldwide are reviewing regulatory submissions.

AstraZeneca and MSD Strategic Collaboration
In July 2017, AstraZeneca and Merck & Co., Inc., Rahway, NJ, US, known as MSD outside the US and Canada, announced a global strategic collaboration to co-develop and co-commercialise Lynparza and Koselugo (selumetinib), a mitogen-activated protein kinase (MEK) inhibitor, for multiple cancer types.

Working together, the companies will develop Lynparza and Koselugo in combination with other potential new medicines and as monotherapies.

Independently, the companies will develop Lynparza and Koselugo in combination with their respective PD-L1 and PD-1 medicines.

Alexion
Alexion, AstraZeneca Rare Disease, is the group within AstraZeneca focused on rare diseases, created following the 2021 acquisition of Alexion Pharmaceuticals, Inc.

As a leader in rare diseases for more than 30 years, Alexion is focused on serving patients and families affected by rare diseases and devastating conditions through the discovery, development and commercialisation of life-changing medicines.

Alexion focuses its research efforts on novel molecules and targets in the complement cascade and its development efforts on haematology, nephrology, neurology, metabolic disorders, cardiology and ophthalmology.

Headquartered in Boston, Massachusetts, Alexion has offices around the globe and serves patients in more than 50 countries.”

https://www.astrazeneca.com/media-centre/press-releases/2023/koselugo-approved-in-china-for-paediatric-patients-with-neurofibromatosis-type-1-and-plexiform-neurofibromas.html

123...156Page 2 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