Biotech

Washington, D.C. – Dr. Pratik Prasad, formerly the Chief Medical and Scientific Officer at the FDA’s Center for Biologics Evaluation and Research (CBER), departed his position on February 29, 2024, following heightened scrutiny surrounding Sarepta Therapeutics‘ gene therapy for Duchenne muscular dystrophy and a targeted online campaign. The campaign, led by conservative activist Laura Loomer, focused on Dr. Prasad’s publicly stated political leanings.

Dr. Prasad’s departure comes at a critical juncture for Sarepta’s Elevidys (delandistrogene moxeparvovec-rokl), a gene therapy approved in June 2023 for ambulatory patients aged 4-5 with Duchenne. The approval was conditional, requiring Sarepta to conduct a confirmatory trial to demonstrate clinical benefit. The FDA’s initial review of Elevidys was marked by internal disagreements, with some agency scientists raising concerns about the limited evidence supporting the drug’s efficacy.Documents released through Freedom of Information Act requests revealed a split within CBER regarding the strength of the data presented by Sarepta.

Loomer’s campaign, which began in late February, amplified Dr. Prasad’s past social media posts identifying as a liberal, alleging bias in his oversight of Sarepta’s product. She specifically highlighted a 2017 Twitter post where he described himself as “fiercely liberal.” The campaign gained traction on platforms like X (formerly Twitter) and Telegram, prompting calls for Dr. Prasad’s removal. Loomer has over 850,000 followers on X.

Duchenne muscular dystrophy is a rare genetic disorder primarily affecting males, caused by mutations in the dystrophin gene. It leads to progressive muscle weakness and degeneration,typically beginning in early childhood. Approximately 1 in 3,500-5,000 male births are affected. currently,treatment options are limited,focusing on managing symptoms and slowing disease progression. Gene therapy offers a potential curative approach by delivering a functional copy of the dystrophin gene.

The circumstances surrounding Dr. Prasad’s exit raise questions about the influence of external political pressure on the FDA’s regulatory processes.Prior to joining the FDA in January 2023, Dr. Prasad was a professor of medicine at the University of California, San Diego, and a practicing cardiologist specializing in heart failure and transplantation at UC San diego Health. He also served as a medical officer at the National Heart, Lung, and Blood Institute (NHLBI) from 2016 to 2022. The FDA has not publicly commented on the reasons for Dr. Prasad’s departure, citing personnel privacy.

Dr. Prasad’s responsibilities at CBER included overseeing the review and approval of biological products, including gene therapies, vaccines, and blood products. His departure leaves a significant gap in the agency’s leadership at a time when gene therapy is rapidly evolving and attracting increasing investment.