In an about-face, Sarepta Therapeutics Inc. said it would “voluntarily and temporarily” pause all shipments of Duchenne muscular dystrophy gene therapy Elevidys (delandistrogene moxeparvovec) in the U.S. The move comes only a few days after the firm publicly declined a U.S. FDA request to halt shipping of the therapy in the wake of a third patient death, this one linked to a gene therapy using the same adeno-associated virus (AAV) vector as Elevidys.
Sarepta Therapeutics Inc. is declining a U.S. FDA request to voluntarily halt shipping its gene therapy, Elevidys (delandistrogene moxeparvovec), in the U.S. On July 18, Sarepta said had it received “an informal request” from the FDA to stop the shipments following a third patient’s death, tied to the gene therapy SRP-9004, which uses the same vector as Elevidys.
How the U.S. FDA might respond became a serious question for Wall Street as Sarepta Therapeutics Inc. made known a second death due to acute liver failure with gene therapy Elevidys (delandistrogene moxeparvovec), cleared for Duchenne muscular dystrophy (DMD). Shares of Cambridge, Mass.-based Sarepta (NASDAQ:SRPT) closed June 16 at $20.94, down $15.24, or 42%, as Wall Street digested the news.
Wall Street was weighing the gravity of the death from acute liver failure of a patient who was treated for Duchenne muscular dystrophy (DMD) with Sarepta Therapeutics Inc.’s gene therapy, Elevidys (delandistrogene moxeparvovec). Liver injury is a known possible side effect of the product, first approved by the U.S. FDA in June 2023 for DMD, as well as other AAV-mediated gene therapies, and the potential problem is highlighted in Elevidys’ prescribing information.
NLRP3 inflammasome activation, pro-inflammatory cytokine production and pyroptosis are key features of inflammation that contribute to liver fibrosis progression, cirrhosis and end-stage liver failure. Pyroptosis, a lytic form of inflammatory-regulated cell death, is regulated by multiprotein complexes expressed in both parenchymal and nonparenchymal hepatic cells. Researchers from Genfit SA presented preclinical efficacy data on CLM-022, a synthetic pentacyclic triterpenoid derivative designed to target the NLRP3 complex.
Baxter International Inc. has joined forces with Miromatrix Medical Inc. to advance the development of bioengineered organs for patients with acute liver failure (ALF). Under the collaborative research agreement, Baxter will combine its Prismax system with Miromatrix's single-use bioengineered liver, miroliver, to provide external support to patients' native livers as they await transplant or potential regeneration.
