May 31, 2024 - FDA Roundup: May 31, 2024

Today, the U.S. Food and Drug Administration is providing an at-a-glance summary of news from around the agency: 

• Today, the FDA approved mRESVIA (Respiratory Syncytial Virus Vaccine) for the prevention of lower respiratory tract disease (LRTD) caused by respiratory syncytial virus (RSV) in individuals 60 years of age and older. mRESVIA is an mRNA-based vaccine that is manufactured by Moderna ModernaTX, Inc.
• Today, the FDA issued a final rule to implement our new authority, under the Safeguarding Therapeutics Act (Pub. L. 116-304) that was signed into law on January 5, 2021, to administratively destroy human or animal devices valued at $2500 or less (or such higher amount as the Secretary of the Treasury may set by regulation) that have been refused admission into the United States. Under the final regulation, notice of the intended destruction and an opportunity to appear before the Agency and introduce testimony contesting the refusal and/or destruction will be provided to the owner or consignee of the device. Other new authorities to address counterfeit devices were enacted in the Food and Drug Omnibus Reform Act (FDORA) as part of the Consolidated Appropriations Act for 2023 signed into law on December 29, 2022. The FDORA added a prohibited act and enhanced penalties for counterfeit devices to the Federal Food, Drug and Cosmetics Act. 
• On Thursday, the FDA announced participation in three new collaborative communities in the medical device ecosystem: Implantable Brain-Computer Interface Collaborative Community (iBCI-CC), OpenOximetry Collaborative Community, and Smart and Autonomous Medical Systems (SaAMS) Collaborative Community. A collaborative community is a continuing forum in which private- and public-sector members, which can include the FDA, work together on medical device challenges to achieve common objectives and outcomes. They are convened by interested stakeholders and may exist indefinitely, produce deliverables as needed, and tackle challenges with broad impacts. If you have questions or a collaborative community in which you would like the FDA to participate, please email [email protected]. 
• On Thursday, the FDA approved lisocabtagene maraleucel (Breyanzi, Juno Therapeutics, Inc.) for adult patients with relapsed or refractory mantle cell lymphoma (MCL) who have received at least two prior lines of systemic therapy, including a Bruton tyrosine kinase inhibitor (BTKi). The most common nonlaboratory adverse reactions (≥ 20%) were cytokine release syndrome (CRS), fatigue, musculoskeletal pain, encephalopathy, edema, headache, and decreased appetite. The FDA approved lisocabtagene maraleucel with a Risk Evaluation and Mitigation Strategy due to the risk of fatal or life-threatening CRS and neurologic toxicities. Full prescribing information for Breyanzi will be posted here. 
• On Wednesday, the FDA notified selected participants of their acceptance into the Support for Clinical Trials Advancing Rare disease Therapeutics (START) Pilot Program. The pilot, announced in September 2023, is being conducted by the FDA’s Center for Biologics Evaluation and Research and Center for Drug Evaluation and Research to help further accelerate the development of novel drug and biological products that are intended to address an unmet medical need as a treatment for a rare disease. Selected participants will be able to interact with the FDA through rapid, ad-hoc communication mechanisms to provide a mechanism for addressing product-specific development issues, including but not limited to clinical study design, choice of control group, fine-tuning the choice of patient population, leveraging nonclinical information, or product characterization. These enhanced communications are intended to augment currently available formal meeting procedures between the FDA and sponsors relating to the development and review of drug or biological products.
• On Wednesday, the FDA announced the virtual Medical Device Sterilization Town Hall: Sterilization Open Q&A that will be held on June 12, 2024, from 2-3 p.m. ET. During this town hall, we will discuss questions we’ve received in the medical device sterilization mailbox and host a Q&A session. We encourage attendees to submit questions to [email protected] by 4 p.m. ET on Wednesday, June 5, 2024. There will be an opportunity to ask questions live during the town hall. Registration is not required.
• On Wednesday, the FDA granted accelerated approval to selpercatinib (Retevmo, Eli Lilly and Company) for pediatric patients two years of age and older with the following: This is the first FDA approval of a targeted therapy for pediatric patients < 12 years of age with RET alterations. The most common adverse reactions (≥25%) were musculoskeletal pain, diarrhea, headache, nausea, vomiting, coronavirus infection, abdominal pain, fatigue, pyrexia, and hemorrhage. The most common Grade 3 or 4 laboratory abnormalities (≥5%) were decreased calcium, decreased hemoglobin, and decreased neutrophils. Full prescribing information for Retevmo will be posted here.
  1. advanced or metastatic medullary thyroid cancer (MTC) with a RET mutation, as detected by an FDA-approved test, who require systemic therapy;
  2. advanced or metastatic thyroid cancer with a RET gene fusion, as detected by an FDA-approved test, who require systemic therapy and who are radioactive iodine-refractory (if radioactive iodine is appropriate); and
  3. locally advanced or metastatic solid tumors with a RET gene fusion, as detected by an FDA-approved test, that have progressed on or following prior systemic treatment or who have no satisfactory alternative treatment options.
  This is the first FDA approval of a targeted therapy for pediatric patients < 12 years of age with RET alterations. The most common adverse reactions (≥25%) were musculoskeletal pain, diarrhea, headache, nausea, vomiting, coronavirus infection, abdominal pain, fatigue, pyrexia, and hemorrhage. The most common Grade 3 or 4 laboratory abnormalities (≥5%) were decreased calcium, decreased hemoglobin, and decreased neutrophils. Full prescribing information for Retevmo will be posted here.
• On Tuesday, the FDA approved first generics of Entresto (sacubitril and valsartan) tablets, indicated to reduce the risk of cardiovascular death and hospitalization for heart failure in adult patients with chronic heart failure, and for the treatment of symptomatic heart failure with systemic left ventricular systolic dysfunction in pediatric patients aged one year and older. Heart failure happens when the heart cannot pump enough blood and oxygen to support other organs in the body. The Centers for Disease Control and Prevention estimates that about 6.2 million adults in the United States have heart failure. 
• Entresto tablets are a commonly used medication and are identified by the Centers for Medicare & Medicaid Services as one of the costliest drugs under Medicare Part D. Side effects of Entresto tablets may include hypotension, hyperkalemia, cough, dizziness, and renal failure.The FDA continues to approve safe, effective, high-quality generic drugs to increase access to medications for the American public.
• On Tuesday, the FDA announced a co-sponsored public webcast workshop with the Digital Medicine Society, Using Patient-Generated Health Data in Medical Device Development: Case Examples of Implementation Throughout the Total Product Life Cycle that will be held June 26-27, 2024, 11 a.m.-3 p.m. ET. The purpose of this workshop is to illustrate how patient-generated health data (PGHD) can promote medical device innovation throughout the total product life cycle. Patients, industry sponsors, regulators, and researchers will share their perspectives on how using PGHD can help advance remote clinical trial data collection and support clinical outcome assessments. Registration deadline is June 25, 2024, by 4 p.m. ET.

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