Latest news with #DuchenneMuscularDystrophy
India Today
a day ago
- Health
- India Today
What forced Vinay Prasad to exit top job at US FDA in just 3 months
The US Food and Drug Administration's (FDA) top vaccine official, Vinay Prasad, stepped down in less than three months after his appointment, following intense scrutiny over his regulatory decisions and political attacks from conservative activists. His stringent oversight of biopharmaceutical company Sarepta Therapeutics' gene therapy for Duchenne Muscular Dystrophy (DMD), a severe genetic disorder, sparked backlash from patient groups and investors, while his approach also raised concerns among biotech firms about innovation, clashing with FDA priorities, according to reports in US-based media an oncologist who was a fierce critic of the US COVID-19 vaccine and mask mandates, was appointed in May by FDA Commissioner Marty Makary. Together, they promised to speed regulatory action and pilot new review pathways, news agency Reuters he faced mounting scrutiny over his decision to pause shipments of Sarepta's Elevidys, a gene therapy for DMD, after three patient deaths raised safety concerns, Reuters reported. The FDA, on July 18, asked Sarepta to stop all shipments of the approved DMD therapy, citing safety FDA's decision to halt shipments sparked outrage from patient groups and biotech investors, who argued the therapy's benefits for a fatal disease were July 28, the FDA reversed course, allowing shipments to resume for ambulatory regulatory approach and the absence of clearly defined standards prompted concern within parts of the biotech industry, with some suggesting it could impact investment and innovation – raising questions about alignment with Marty Makary's priorities, according to an op-ed in the Wall Street Journal (WSJ).The agency's lack of clear, predictable standards under Prasad was creating enormous uncertainty for the industry, especially small biotech companies, and threatened to chill investment, according to the inflexible position on the design of clinical trials also risked slowing and raising costs for drug development. This is the opposite of what Marty Makary says he wants to do, the WSJ op-ed Republican Senator Rick Santorum, who has connections to Sarepta Therapeutics, reportedly contacted senior White House officials to express concerns over Prasad's attempts to restrict the use of Elevidys, Daily Beast reported, citing The has maintained that its treatment was not responsible for the reported deaths. Prior to joining the FDA, Dr Prasad had voiced criticism of the company, and following his appointment, Sarepta's stock dropped by nearly 25%, according to people familiar with the situation told the Associated Press (AP) that Prasad was ousted following several recent joined the FDA in May after years as an academic researcher at the University of California, San Francisco, where he frequently criticised the FDA's approach to drug approvals and Covid-19 vaccines, AP PRASAD RESIGNS TO AVOID DISTRACTIONA Department of Health and Human Services spokesperson told CNN on July 29 that Vinay Prasad resigned to avoid being a "distraction" to the agency's work under the Trump administration, choosing to return to California to spend more time with his 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," the spokesperson told the Prasad was also given the role of FDA chief medical and scientific several health officials later appointed under the Trump administration, Prasad had previously been outspoken in his criticism of the government's handling of the Covid-19 pandemic and its vaccine departure came amid fresh pressure from the White House for him to resign, according to a person familiar with the matter who was granted anonymity to describe the internal dynamics, and followed days of criticism from Laura Loomer, a right-wing activist with extraordinary access to President Donald Trump, CNN July 20, Loomer, a close Trump supporter, published a blog post accusing Prasad of being a "progressive leftist saboteur" and claiming he was actively working against the mission of the former administration officials and advisers, from campaign strategists to public health voices, faced public targeting by Loomer before being sidelined or dismissed by who called herself an investigative journalist, has built a reputation for aggressive rhetoric, confrontational tactics, and a social media presence that thrives on Loomer has no formal role in Donald Trump's campaign or political machinery, her influence seems to travel in parallel with the MAGA ecosystem. Several former administration officials and advisers – ranging from campaign strategists to public health voices – have found themselves publicly targeted by Loomer shortly before being sidelined or dismissed by Trump or his inner days before his resignation, the FDA Commissioner publicly defended Prasad, telling Politico that he is "an impeccable scientist" and "one of the greatest scientific minds of our generation."Prasad also drew criticism from former officials and vaccine experts after internal memos from May revealed that he overrode FDA scientists on recommendations for two new versions of COVID-19 vaccines, CNN reported.- EndsMust Watch
Wall Street Journal
2 days ago
- Health
- Wall Street Journal
WSJ Opinion: The FDA's Gene-Therapy Reversal and the Exit of Vinay Prasad
A top official at the Food and Drug Administration steps down after the agency changes course on a treatment for Duchenne muscular dystrophy. What does this say about the Trump Administration's approach toward drug innovation?
CNBC
4 days ago
- Health
- CNBC
Sarepta resumes shipping of gene therapy Elevidys to patients who can walk
Sarepta Therapeutics said on Monday it will resume shipping of its gene therapy Elevidys to patients with a rare muscular disorder who can walk, after the FDA recommended the removal of a voluntary hold that was placed on the therapy. However, the use of the gene therapy remains on hold for Duchenne Muscular Dystrophy patients who cannot walk, the FDA said, adding that it is continuing to work with the company while investigating the death of two patients. The FDA's recommendation for ambulatory patients — those who can walk — followed a probe that showed the death of an eight-year-old boy in Brazil, was unrelated to the gene therapy, the agency said. Roche, which has partnered with Sarepta for commercialization outside the U.S., had previously said the patient's death was not related to the therapy, according to the reporting physician's assessment. Sarepta is also facing intense scrutiny following the death of two non-ambulatory teenage boys associated with Elevidys, as well as a 51-year-old man who had received its experimental gene therapy SRP-9004.
Medscape
25-07-2025
- Health
- Medscape
EMA Says No to Duchenne Gene Therapy Elevidys
At its July 2025 meeting, the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use recommended not granting marketing authorization for the Duchenne muscular dystrophy (DMD) gene therapy treatment delandistrogene moxeparvovec (Elevidys, Sarepta Therapeutics). Patients with DMD lack normal dystrophin, found primarily in skeletal and cardiac muscle cells, such that their muscles become progressively weaker and eventually stop working. Elevidys is an adeno-associated virus vector-based gene therapy product for ambulatory patients aged 4 years and older with a confirmed mutation in the DMD gene. It contains the active substance delandistrogene moxeparvovec, made of a virus containing genetic material, to produce a truncated version of dystrophin and thereby slow down disease progression. It is given as a single infusion as a one-time treatment designed to treat the underlying cause of DMD. In making their decision, the EMA said that a study had failed to show that Elevidys had an effect on movement abilities after 12 months. No Significant Improvement The study involved 125 children aged between 4 and 7 years with DMD who were able to walk and who received one infusion of either delandistrogene moxeparvovec or placebo. The main measure of effectiveness was an effect on movement abilities over 12 months, assessed using the North Star Ambulatory Assessment (NSAA). The scale ranges from 0 to 34, with higher scores indicating better movement abilities. Improvements in NSAA scores were observed in patients who received delandistrogene moxeparvovec and placebo. The difference in the change in scores between the two groups was 0.65, which was not statistically significant. In addition, although many patients treated with delandistrogene moxeparvovec were shown to produce a shorter form of the dystrophin protein, the levels of dystrophin could not be linked to an improvement in movement abilities, the EMA said. Acute Liver Failure Deaths In March this year, a 16-year-old boy died from acute liver failure after receiving treatment with Elevidys in December the previous year. In the wake of this — and at the request of the EMA — in April this year, the company temporarily halted clinical studies of the treatment in the EU. Patients who had previously been treated with Elevidys in a clinical trial continued to be monitored. In June, a second patient, aged 15, who had been treated with the drug also died from acute liver failure. Earlier this month, the FDA asked the company to include a "black-box" warning for the risk of acute liver injury and liver failure in patients with DMD who can walk. The EMA said that the company that had applied for a marketing authorization may ask for re-examination of the opinion within 15 days of receiving the agency's opinion. Rob Hicks is a retired National Health Service doctor. A well-known TV and radio broadcaster, he has written several books and has regularly contributed to national newspapers, magazines, and online publications. He is based in the United Kingdom.
Yahoo
23-07-2025
- Business
- Yahoo
Sarepta stock faces multiple downgrades and questions of solvency amid Elevidys pause
Sarepta Therapeutics' (SRPT) stock has been hammered in the past week after the company was forced to pause shipments of its top-selling Duchenne muscular dystrophy (DMD) therapy Elevidys — throwing the future of the company into question. Elevidys netted $821 million of the company's $1.78 billion in 2024 revenue and contributed to more than 50% of the company's $513 million in second quarter revenue this year. It's the company's top-selling product. It's why seven analysts have downgraded the stock since Friday, including Bank of America's Tazeen Ahmad on Wednesday. Ahmad downgraded the stock to Underperform and revised the firm's price target to $10 from $20. The stock is currently trading at $13.90 per share and closed down more than 2% on Wednesday. Sarepta "has requested a meeting with FDA and might have more visibility on length of the pause once FDA responds to their submission for a revised label," Ahmad wrote in a note to clients. "In short, we think this could impact the Elevidys brand further, limiting uptake following highly focused media attention as well as a renewed chance of Elevidys being removed from the US market." Leerink Partners downgraded the stock last week from Outperform to Market Perform. Analyst Joseph Schwartz wrote in a note to clients on Monday that how long the pause lasts is important for future modeling. "This decision is also roughly aligned with our published SRPT model, which assumes a significant slowing of sales in the third quarter, zero sales in the fourth quarter, and a resumption of shipments in early 2026," Schwartz wrote. "We lack visibility into how long this pause may last, or what the intermediate scenarios are from here (e.g., a narrower approval)." The stock now has five Buy, 17 Hold, and four Sell ratings. Domino effect The technology to deliver the therapy in Elevidys is at the center of the issue facing the company. A 51-year-old, late-stage patient in a phase 1 trial of a different muscular dystrophy therapy, known only by its trial name, SRP-9004, died from liver toxicity last month, but Sarepta failed to disclose the death — even as it announced it was discontinuing the trial as part of a restructuring last week. That began the domino effect of downgrades and the stock sell-off as analysts questioned why the company had kept quiet. On a call with investors Friday, CEO Doug Ingram defended his reasoning for not mentioning it. "We did not discuss this matter in our call on Wednesday because it was neither material nor central to the topics at hand on Wednesday," Ingram said. In a note following the call, Leerink's Schwartz wrote, "Is This Material?!? All Credibility Lost." It's the third death linked to a gene therapy from the company this year and a reason why the FDA previously required the company to add new warning labels to Elevidys, which is part of the pause in shipments currently. But now, the company's future is in flux as its revenue stream has been paused for an undetermined amount of time. Jefferies analyst Andrew Tsai, one of the few who has maintained a Buy rating, said the company could defend the therapy as sicker and older patients have been more negatively impacted, while younger patients who are still able to move independently have benefited. "Prior to the third death, SRPT's internal stress tests suggested the DMD franchise could 'floor' at $1.4B annually into 2027, including $500M+ for Elevidys in ambulatory," Tsai wrote in a note to clients Sunday. "For Elevidys, one can point to how the deaths have occurred only in older patients." "More cost reductions is possible too," Tsai noted of how Sarepta could end up handling the hit. Anjalee Khemlani is the senior health reporter at Yahoo Finance, covering all things pharma, insurance, provider services, digital health, PBMs, and health policy and politics. That includes GLP-1s, of course. Follow Anjalee as AnjKhem on social media platforms X, LinkedIn, and Bluesky @AnjKhem. Click here for in-depth analysis of the latest health industry news and events impacting stock prices
