This Week at FDA: Becerra asks for new authorities; FDA issues guidances, reports

Welcome to another installment of This Week at FDA, your weekly source for updates – big and small – on FDA, drug and medical device regulation, and what we’re reading from around the web. It’s been a busy week for the agency as Xavier Becerra, secretary of the US Department of Health and Human Services, defended his department’s proposed FY2024 budget in the Senate, the FDA released several guidances and reports, and announced upcoming meetings.

During a Senate Finance Committee hearing, Becerra argued for additional authorities to track medical product shortages that Food and Drug Administration officials have requested as part of the Biden Administration’s proposed budget.

The hearing came after a Senate Homeland Security and Governmental Affairs Committee report that shortages of critical medicines continue to rise.

“Neither the federal government nor industry has end-to-end visibility of the pharmaceutical supply chain—from the key starting materials, APIs, finished dosage and various other manufacturers that are ‘upstream’—to the ‘downstream’ suppliers, which include purchasers and providers,” the report states. “This lack of transparency limits the federal government’s ability to proactively identify and address drug shortages.”

In other news, FDA says it thinks certain medical device shortages are nearing an end. It predicts supply of multitarget respiratory specimen nucleic acid tests, micropipettes, microbiological specimen collection devices and other products used in response to the COVID-19 pandemic will return to normal in the first half of the year.

This week the FDA also published a number of guidances detailing how medical device and diagnostics manufacturers can transition their COVID-19-related products to traditionally marketed products. In particular, the agency released guidances for products that currently fall under its enforcement policies and those that are on the market under an emergency use authorization (EUA).

FDA also published a draft guidance for sponsors of cancer drugs and biologics who are designing trials for drugs that they hope will get accelerated approval. The agency notes that while single-arm studies are commonly used to support accelerated approval, it prefers randomized controlled trials.

FDA updated a 2005 final guidance on pharmacogenomic data submissions by publishing a draft guidance on the same topic. The agency says the updated draft guidance clarifies the contexts in which pharmacogenomic study findings and data must be included in submissions related to investigational new drug applications (INDs), new drug applications (NDAs) and biologics license applications (BLAs) based on its regulations.

Peter Marks, director of the Center for Biologics Evaluation and Research (CBER) said this week the agency needs to start using the controversial accelerated approval pathway to advance gene therapies for rare diseases. "I think the issue here is we can’t be so careful about our approvals under accelerated approval that we prevent potentially lifesaving therapies from getting to market in a timely manner,” he told attendees at the annual meeting of the Muscular Dystrophy Association.

Drugs & Biologics

The Center for Drug Evaluation and Research's (CDER) Office of Compliance published its annual report for fiscal year 2022. It lists the actions the agency took against manufacturers that failed to comply with its regulations and alleged criminals who marketed drugs illegally. In total, the agency says it has issued 101 warning letters and obtained two consent decrees of permanent injunctions, and classified 343 recalls associated with more than 1,500 products.

FDA announced the rates for over-the-counter (OTC) monograph drug user fees for fiscal year 2023 which includes information about facility fee calculations, fee due dates, fee payment procedures and other fees. The agency reminds stakeholders ghat OTC monograph drug facility fees for FY 2023 are due 1 June 2023.

In collaboration with the University of Maryland, FDA plans to host a virtual public workshop entitled “Creating a Roadmap to Quantitative Systems Pharmacology-Informed Rare Disease Drug Development” on 11 May 2023. The event is meant to engage stakeholders to discuss using quantitative systems pharmacology (QSP) to develop rare disease drugs.

FDA announced it is hosting its annual Generic Drugs Forum (GDF) starting on 12 April. It is a two-day virtual event that the agency says will allow stakeholders to hear from FDA subject matter experts and provide information to product applicants by offering "practical advice and taking a deep dive into the Abbreviated New Drug Application (ANDA) assessment process."

A panel of external FDA advisors gave a thumbs down to Biogen's drug tofersen to treat amyotrophic lateral sclerosis (ALS). They said the drug failed to meet its primary and secondary endpoints, and there were some significant safety concerns such as joint and muscle pain. Before the advisory committee met FDA briefing documents said that the safety issues should not preclude it from accelerated approval.

Weeks after Sarepta Therapeutics said FDA told it the agency won't need to convene an advisory committee to discuss its experimental gene therapy called SRP-9001 to treat Duchenne muscular dystrophy, the agency announced it will do exactly that. The company says the meeting date will likely be announced in the next few weeks as the agency needs to give a decision by 29 May but the change of heart from the agency caught analysts and patient advocates by surprise.

Abbvie says FDA has rejected its ABBV-951 investigational 24-hour continuous foscarbidopa/foslevodopa pump intended to treat motor fluctuations in adults with advanced Parkinson disease (PD) in a complete response letter (CRL) where it asked for more information. The company plans to file another new drug application (NDA) and if approved, it would be the first subcutaneous delivery of carbidopa/levodopa, which is considered the standard of care for patients with the disease.

Medtech

This week FDA said it has updated its CDRH Portal to allow multiple users to view the status of 510(k) applications, the most popular medical device pathway. Official 510(k) correspondents can designate multiple delegates who can now view the status of the submissions and its associated milestones according to the agency.

FDA in recent months has published a number of safety communications, reports and guidances regarding elevated risk for a rare form of squamous cell carcinoma from breast implants. The Breast Implant Safety Alliance, however, says regulators have been slow to warn the public of the danger which they've been drumbeating for years.

FDA issued a safety communication for users of joint replacement devices manufactured by Exactech between 2004 and August 2021 and their health care providers. The agency notes the devices that include knees, ankle, and hip devices were recalled because the packaging was defective and could lead to corrosion and other defects.

This Week at FDA: Becerra asks for new authorities; FDA issues guidances, reports

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