Sarepta Therapeutics, Inc.
NEWS
After Sarepta reported the death of a patient who had recently taken the gene therapy Elevidys, patient advocacy group Parent Project Muscular Dystrophy stepped up—as they always do.
Sarepta will update Elevidys’ label after a patient died following treatment; the FDA issues flu vaccine recommendations without advisor input; Trump CDC nominee Dave Weldon pulled at last minute; and FDA decisions expected for Alnylam’s Amvuttra in ATTR-CM and Milestone’s etripamil in tachycardia.
After a patient taking the Duchenne muscular dystrophy gene therapy Elevydis died of liver injury, Sarepta will update the label to reflect the safety signal.
Faced with the encroaching threats of patent expirations and generics, biopharma companies in 2024 invested 33% more in licensing deals, on average, than in 2023 with an eye toward enriching their pipelines with novel and potentially more effective therapies.
While the last decade has brought considerable progress for patients with DMD, substantial unmet need remains. Several companies including Wave, Dyne and Avidity are looking to answer the call with investigational therapies targeting greater efficacy and broader reach.
Biopharma executives were busy Monday, striking high-value deals and providing updates on cancer, obesity and vaccine pipelines.
Sarepta Therapeutics’ Duchenne muscular dystrophy therapy Elevidys handily beat analysts’ expectations in the fourth quarter of 2024, reflecting the biotech’s “world-class” execution, according to BMO Capital Markets analysts.
Other notable greenlights this year include Bristol Myers Squibb’s Cobenfy, the first novel therapeutic for schizophrenia in 35 years, and Madrigal Pharmaceuticals’ Rezdiffra, the first-ever treatment for MASH.
Not exactly known for its dealmaking, Sarepta Therapeutics has thrown down a massive wad of cash to work with Arrowhead Pharmaceuticals on RNAi-based medicines.
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