Latest news with #SareptaTherapeuticsInc.
Mint
30-07-2025
- Health
- Mint
Who is Vinay Prasad? US FDA's top regulator exits agency after controversy over Sarepta gene therapy
Dr. Vinay Prasad, a top regulator at the US Food and Drug Administration, has resigned in less than three months in the job following a controversy over the handling of Sarepta Therapeutics Inc.'s gene therapy. 'Dr. Prasad did not want to be a distraction to the great work of the FDA in the Trump administration and has decided to return to California and spend more time with his family,' a report by CNN quoted a spokesperson for the US Department of Health and Human Services. In May, Prasad, a hematologist and oncologist was appointed head of the FDA's Centre for Biologics Evaluation and Research, granting him authority over vaccines and biological medicines. Subsequently, he was also appointed the FDA's chief medical and scientific officer. Similar to several Trump administration health appointees, Prasad had been a vocal critic of the government's response and vaccine policies during the COVID-19 pandemic. The report, citing people aware of the development, said Prasad resigned amid pressure from the White House. Additionally, Laura Loomer, a right-wing activist known to have close ties with Donald Trump, consistently criticised Prasad. She publicly criticised him for days on her website and social media, calling him a 'progressive leftist saboteur' who was 'undermining President Trump's FDA.' Loomer called out Prasad's previous social media posts and podcast episodes, where she claimed that he supported liberal politicians and expressed 'disdain' for Trump. However, FDA Commissioner Dr. Marty Makary defended Prasad just days ago. In an interview with Politico, Makary said Prasad is an 'impeccable scientist … one of the greatest scientific minds of our generation.' 'We thank him for his service and the many important reforms he was able to achieve in his time at FDA,' the spokesperson for HHS said. Prasad took on his role at the FDA following years of outspoken criticism of certain drug approvals by the agency. Notably, he condemned the approval of Sarepta's Duchenne muscular dystrophy drug, Elevidys, asserting that there was insufficient evidence to demonstrate it effectively slowed or reversed symptoms of this rare and deadly genetic condition. This month, the FDA asked Sarepta to stop shipments of the drug after a reported death of a young patient in Brazil. Just one day before Prasad's departure, the agency unexpectedly reversed its decision and allowed Sarepta to continue shipments for certain patients. Prasad faced criticism from former officials and vaccine experts after May's internal memos showed he overruled FDA scientists on two new Covid-19 vaccine versions. The then-CDER director criticised the broad use of these vaccines; ultimately, the FDA approved them for older and immunocompromised individuals but did not recommend them for younger Americans without underlying health conditions.
Mint
17-07-2025
- Business
- Mint
Sarepta Slashes Workforce After Patient Deaths on Gene Therapy
(Bloomberg) -- Sarepta Therapeutics Inc. is cutting more than one-third of its workforce and will add a black box warning label to its gene therapy for a fatal muscle disorder after two patient deaths raised doubts about the future of the company. The Cambridge, Massachusetts-based biotech company said Wednesday it would eliminate about 500 employees as part of a sweeping restructuring. It's also pausing several drugs in its pipeline. Sarepta said the moves would contribute to an estimated $400 million in annual cost savings. The company agreed to warn doctors and patients about the risk of liver failure from Elevidys, its gene therapy to treat Duchenne muscular dystrophy, at the request of the US Food and Drug Administration. Black box labels are the most serious safety warnings a drug can have, and indicate severe or potentially deadly complications. Sarepta said the job cuts will help it maintain access to a $600 million revolving credit line and create cash flow that could help it repay a convertible note due in 2027. The company also disclosed preliminary second-quarter results showing total net product revenue of $513 million. More than half came from Elevidys. Last month, Sarepta reported that a second teenage boy died of acute liver failure while being treated with its $3.2 million gene therapy. Both fatalities occurred in patients who weren't able to walk because of the muscle-wasting disease. At the time, the company suspended its 2025 revenue guidance, and pledged to take a careful look at its costs going forward. Sarepta also paused a clinical trial and halted shipments of the drug for non-ambulatory patients. Sarepta said it will submit a plan to the FDA for patients who can't walk that includes suppressing their immune systems before administering Elevidys, which may reduce their risk of complications. It will also discuss a path to resuming shipment of the therapy for those patients, typically older boys and young men. The FDA is investigating the Elevidys deaths. The fatalities pose one of the first big tests for Vinay Prasad, the new head of the regulator's gene therapy division. As an academic at the University of California San Francisco, prior to his current role, he was highly critical of the FDA's expedited approval process and its use for Sarepta's gene therapies in particular. In social media posts in March, Prasad questioned Sarepta's treatment, saying it 'seems to be killing kids' with Duchenne and 'destroying their livers.' More stories like this are available on
