Sarepta's controversial gene therapy for a rare muscle condition will remain available in the US with a strict new warning about potentially deadly liver injuries, a regulatory decision that removes the risk it would be pulled from the market https://t.co/srYddSGw9U ...

Core Viewpoint - BioMarin Pharmaceutical plans to divest its gene therapy product, which was once anticipated to be a blockbuster treatment for a rare bleeding disorder [1] Company Summary - The gene therapy in question was previously expected to generate significant revenue as a leading treatment option for a specific rare bleeding disorder [1]

Healthcare & Insurance Industry Focus - A family's insurance company, Blue Cross Blue Shield, is denying coverage for a potentially life-saving gene therapy (Levitus) for one son with Duchenne Muscular Dystrophy (DMD), while covering it for another son with the same diagnosis [1][5] - Levitus, a state-of-the-art gene therapy, costs $3.2 million [4] - Blue Cross Blue Shield declined to discuss the specifics of the case, citing patient privacy, but stated its decisions are guided by nationwide best practices [6] - The FDA is scrutinizing Levitus after two boys died after receiving it, though their DMD was advanced [7] Duchenne Muscular Dystrophy (DMD) - DMD is a genetic disorder that progressively attacks a person's muscles, typically leading to wheelchair use around age 12 and a life expectancy of under 30 years [2] - The gene therapy, Levitus, has shown to slow DMD's progress [4] - DMD causes muscle weakness and fatigue, with scar tissue layering on larger calf muscles [3] - The family emphasizes the urgency of treatment, as the son's condition is deteriorating rapidly [8]

Longtime drug developer Suma Krishnan was 51 when she cofounded Krystal Biotech. Now the company has one gene therapy on the market and more in the works. #ForbesOver50 (Photo: Jamel Toppin for Forbes) https://t.co/eU154GZ8Kg https://t.co/fmDPWfCYnm ...

Company Performance & Regulatory Actions - Shares of Sarepta Therapeutics experienced a pre-market increase of approximately 36% [1] - Sarepta Therapeutics will resume shipping Alvdus, a gene therapy for muscular disorders, to ambulatory patients [1] - The FDA had previously recommended a pause in shipments due to safety concerns following three patient deaths [1] - Sarepta's stock is still down by about 84% this year [1] Safety Concerns & FDA Response - Initial concerns arose regarding deaths in non-ambulatory patients [2] - The FDA's Center for Biologics Evaluation and Research initially advocated for a complete halt to shipments [2] - The FDA later allowed shipments to continue for ambulatory patients due to pushback from patient families [3][4] - A recent patient death was deemed unrelated to the gene therapy by the FDA [1] Industry & Political Context - The Wall Street Journal published an op-ed criticizing the FDA's approach to drug innovation, referring to it as a "surrepta mugging" [5] - Criticism of the FDA's actions extended across the political spectrum [5][6]

Regulatory Approval - FDA 暂缓批准 Sarepta 将其有争议的基因疗法重新投放市场 [1] - FDA 需确认该疗法不会导致更多死亡案例后，才会批准 [1]

There were warnings to the FDA about Sarepta before US regulators asked the company to halt shipments of its gene therapy https://t.co/ZZC0xgoUpS ...

For more on Soft's path ahead, Missouo healthcare strategist Jared Holes joins us here on set. Jared, great to have you with us. Um, the management had a call uh this morning and you were on that call.You talked to investors about the call. They highlighted that the the belief that they think that the ambulatory patient segment being treated with this drug could support profitability. But then there didn't seem to be real um comfort uh in in this event not happening in the in that group as well, right.I mea ...

Safety Concerns & Regulatory Impact - Serepta's gene therapy Elevidys linked to two patient deaths due to liver failure in 15 and 16-year-old boys with Duchenne muscular dystrophy [1][2] - FDA initially approved Elevidys for 4 and 5-year-olds who could still walk, later expanding the label to nearly all Duchenne patients, a decision that proved controversial due to limited evidence of efficacy in older patients [3] - Serepta has halted shipments of Elevidys for more advanced patients while exploring a new safety regimen [4] Financial Performance & Projections - Serepta is suspending its 2025 guidance and will update its full-year revenue outlook when it announces the second quarter results [4] - Prior analyst projections estimated Elevidys sales of $1.5 billion for the year, but these projections are now in serious doubt [4][5] Market Reaction & Stock Performance - Serepta's stock (SPTO) is down approximately 48% today and about 85% over the last year [5] - Safety concerns may cause eligible patients to reconsider treatment decisions [5]