This Week: FDA staff raise alarm over CNVP, no COVID-19 vaccine boxed warning, and more

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. This week, FDA staffers raised concerns about the potential politicization of the commissioner’s new drug voucher program, FDA said it doesn’t plan on adding a boxed warning to COVID-19 vaccines, and the agency released details about proposals for the next generic drug user fee program.

FDA staffers have told STAT News that the Commissioner's National Voucher Program (CNVP) is being used as a political tool by the Trump administration. While agency product reviews have historically been left to career scientists to avoid the appearance of political interference, agency staff members have anonymously raised concerns that political appointees steered vouchers to companies that are party to the administration's drug-pricing agreements.

Later in the week, FDA announced that it had awarded two CNVP pilot program vouchers to Merck for its drug enlicitide decanoate, an oral PCSK9 inhibitor for lowering low-density lipoprotein (LDL) cholesterol; and sacituzumab tirumotecan, a trophoblast cell-surface antigen 2 (TROP2)-directed antibody-drug conjugate to treat cancer. The agency stated that the drugs have the potential to increase access to treatment and reduce healthcare costs significantly.

Despite recent news reports to the contrary, FDA Commissioner Marty Makary told Bloomberg News that the agency has no plan to require a boxed warning on COVID-19 vaccines following the administration’s concerns that regulators previously downplayed the risks of myocarditis.

"The safety and epidemiology center within the FDA did recommend that," said Makary. "It was a recommendation formally put out, but some of our scientists and leadership, like Dr. Vinay Prasad, have said it may be different today than it was in the first year of COVID when the shot came out.”

CNBC reported that the FDA approved travel for 31 staffers to attend the fall conference of the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) in Singapore during the final hours of the recent government shutdown at a cost of about $250,000, or about $8,000 per person.

FDA finalized several guidances this week, including a much-anticipated medical device guidance that states that the agency will accept real-world evidence (RWE) without requiring identifiable patient-level data in medical device submissions. Instead, reviewers will consider the strength of submitted RWE on an application-by-application basis.

A guidance about FDA’s Bioresearch Monitoring (BIMO) inspections program also was finalized. The agency noted that it fulfills a requirement under the 2022 Food and Drug Omnibus Reform Act (FDORA) to enlighten manufacturers about its BIMO processes and practices.

FDA published a couple of guidances on clinical trial enrollment. It issued a final guidance on practices that can generally improve drug and biologic clinical trial enrollment, taking into consideration both demographic and non-demographic characteristics of the trial cohort.

Regulators also published a draft guidance on how to study sex differences in clinical trials. The guidance includes recommendations on how to increase enrollment of female participants in clinical trials and non-interventional studies, analyzing and interpreting sex-specific data, and including sex-specific information in marketing submissions.

Drugs & Biologics
The standard operating policy and procedure (SOPP) used by regulators to conduct meetings with drug and biologics sponsors was updated by the FDA to clarify its single discipline clarification process. This is the 16th iteration of the SOPP manual.

FDA also published an updated SOPP detailing how it decides to issue a refuse to file (RTF) determination. The 12th iteration of the SOPP updates Appendix B, which includes discipline filing checklists for biologics license applications (BLA), new drug applications (NDA), and efficacy supplements.

Milestone Pharmaceuticals' heart drug Cardamyst (etripamil) nasal spray was approved by FDA to treat episodes of paroxysmal supraventricular tachycardia (PSVT). The company stated that its drug was the first and only self-administered nasal spray approved for the treatment of PSVT.

Daiichi Sankyo's cancer drug Enhertu (fam-trastuzumab deruxtecan-nxki) in combination with pertuzumab was approved as the first-line treatment of adults with unresectable or metastatic HER2-positive (HER2 IHC 3+ or ISH+) breast cancer based on an FDA-approved test. The agency stated that it had simultaneously approved the PATHWAY anti-HER-2/neu (4B5) Rabbit Monoclonal Primary Antibody and the HER2 Dual ISH DNA Probe Cocktail as companion diagnostic devices for use with the drug combination treatment.

FDA also approved pharmaand's cancer drug Rubraca (rucaparib) to treat patients with a deleterious BRCA mutation (BRCAm) (germline and/or somatic)-associated metastatic castration-resistant prostate cancer (mCRPC) previously treated with an androgen receptor-directed therapy diagnosed with an FDA-approved companion diagnostic. The agency noted that the drug was granted accelerated approval in 2020 for a similar indication.

FDA announced it has issued a priority review voucher to Stealth BioTherapeutics for its injection Forzinity (elamipretide) to treat children with Barth Syndrome. An enlarged and weakened heart, muscle weakness, recurrent infections, and short stature are characteristic of the condition, and the drug may help improve muscle strength in patients.

FDA published the meeting minutes from its November and December meetings with industry groups, including the Pharma & Biopharma Outsourcing Association (PBOA), the Association for Accessible Medicines (AAM), and the Bulk Pharmaceuticals Task Force (BPTF), as they work on reauthorizing the Generic Drug User Fee Amendments (GDUFA IV).

In November, FDA proposed to extend goal dates after potential Official Action Indicated (pOAI) alerts, changes to facility inspections and classification, and allow certain companies to request an early surveillance inspection.

The following month, industry proposed changes to the abbreviated new drug application (ANDA) review timelines that it said would create more predictable timelines. It also proposed changes to the complex generics program and controlled correspondence program.

Medtech

Electrical tongue nerve stimulators used to treat motor deficits have been down-classified to Class II devices with special controls. The decision comes after Helius Medical asked FDA for a de novo classification of its Portable Neuromodulation Stimulator (PoNS).

This Week: FDA staff raise alarm over CNVP, no COVID-19 vaccine boxed warning, and more

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