1. What’s happening
As the head of the #FDA’s vaccines & related biologics products division, Vinay Prasad, prepares to exit, the turnover could be a net positive for the biotech industry, especially if the regulatory pathway for rare-disease drugs becomes less fraught under Prasad’s replacement.
2. What’s the big deal
Prasad, the head of the Food and Drug Administration’s Center for Biologics Evaluation and Research (CBER), will be departing at the end of next month, FDA Commissioner Marty Makary told The Wall Street Journal on March 6.
- Prasad blocked a number of rare-disease drugs from receiving accelerated approval, a program in which regulators green-light a treatment based on early signals versus accumulating years of real-world outcomes, even as Makary expressed a desire to expand the program.
- While a precise number of rejections under his watch isn’t available, firms saying they received unexpected CRLs (complete response letters) for experimental rare-disease therapies include Pierre Fabre Pharma and Regenxbio.
- UniQure: Last year, the FDA denied accelerated approval for its Huntington’s gene therapy, AMT-130, following what management said had been positive guidance from the agency. UniQure subsequently reported that, not only had it been told its early studies were insufficient, but that Prasad wanted a new, placebo-controlled trial.
- The latter, which would entail subjects in the control group undergo a 10-hour fake brain surgery, understandably raised eyebrows among the Huntington’s disease community.
- The UniQure saga reached a head on March 5, when a senior FDA official anonymously criticized the company and its AMT-130 application in a call with reporters.
- The appointment of CBER’s future leader is sure to have a bearing on emerging biotech companies, particularly those in the pre-commercial phase and gearing up for a first product launch, says Beghou Partner Vishal Singal.
- “Next Generation Therapy companies would look at Prasad’s exit as clearing the way for a more lenient approach to approvals for rare disease companies,” he notes, namely Makary’s recent promises around accelerating development of such therapies.
- That lenience “would require them to be agile in their support for approvals, such as innovative statistical methods, flexibility in their ways of working with the FDA, potential infrastructure ecosystem, and resources to support launch at scale across all stakeholders – patient, HCP and payer,” Singal adds.
- While his brief but tumultuous tenure appears to be coming to an end, it’s anybody’s guess whether Prasad's successor will fulfill Makary’s calls for “regulatory flexibility.” Indeed, his coming departure is no guarantee that drama won’t remain under the leadership of Makary and his boss, HHS Secretary Robert F. Kennedy.
- But for the biotech sector, which has been on the road to recovery the past year, this changing of the guard offers hope for a return to much-needed stability.
3. What’s at stake
As Prasad and his boss gave mixed signals, these companies have endured a roller-coaster ride. One has become the poster child…
4. What it means for commercial execs
It’s highly unusual for a regulator to comment directly about a specific company. Together with the string of CRLs, the official’s words only deepened the agency’s rift with industry.
5. What’s next
Prasad, who also stirred controversy over his regulation of Moderna’s application for a novel flu vaccine and Sarepta’s gene therapy for Duchenne muscular dystrophy, had already left FDA once and returned last year.
- While his brief but tumultuous tenure appears to be coming to an end, it’s anybody’s guess whether Prasad's successor will fulfill Makary’s calls for “regulatory flexibility.” Indeed, his coming departure is no guarantee that drama won’t remain under the leadership of Makary and his boss, HHS Secretary Robert F. Kennedy.
- But for the biotech sector, which has been on the road to recovery the past year, this changing of the guard offers hope for a return to much-needed stability.