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.
Researchers at the University of Minnesota and collaborating institutions have developed a promising stem cell-based therapy for the treatment of muscular dystrophies. The team has successfully created a novel myogenic progenitor cell product called Myopaxon, derived from human-induced pluripotent stem cells (iPSCs).
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.
Carving out $400 million in annual cost savings is going over well with Sarepta Therapeutics Inc.’s investors, as the company’s stock got a 19.6% boost on July 17. Its big seller, the gene therapy Elevidys (delandistrogene moxeparvovec), continued its revenue numbers decline, so Sarepta chopped operating expenses by letting about 500 employees go in a 36% cutback and pared its development path.
Capricor Therapeutics Inc. received a complete response letter (CRL) from the U.S. FDA on the BLA for deramiocel to treat cardiomyopathy in Duchenne muscular dystrophy patients. The company’s CEO said the letter was unexpected.
In yet another fail for the Duchenne muscular dystrophy (DMD) field, Taiho Pharmaceutical Co. Ltd.’s pizuglanstat (TAS-205) did not meet the primary endpoint in a phase III trial. The phase III Reach-DMD trial, a randomized, placebo-controlled, double-blind and open-label, extension study of pizuglanstat in patients with DMD showed no significant difference in the mean change from baseline to 52 weeks in the primary endpoint of time to rise from the floor in the ambulatory cohort of the study.
Edgewise Therapeutics Inc. CEO Kevin Koch speculated that “perhaps a different environment at the FDA” from four months ago led to reviewers’ caution on sevasemten, his firm’s fast skeletal myelin inhibitor for Becker muscular dystrophy and Duchenne muscular dystrophies.
The realignment within the U.S. FDA continued with reports of the removal of two high level executives. When asked by BioWorld if the Center for Biologics Evaluation and Research’s (CBER) Office of Therapeutic Products director and deputy director had been forced out and if so, why, an HHS spokesperson responded on background with a single sentence: “Center directors deserve to be supported by managers that are aligned with aggressive goals to expeditiously advance therapeutics for rare diseases using the gold standard of science.”
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.
Regenxbio Inc.’s gene therapy in treating Duchenne muscular dystrophy (DMD) produced positive initial phase I/II results from its first five patients. However, the company’s stock (NASDAQ:RGNX) shuddered on June 5 as shares closed at $8.36 each, a drop of 17% on the day.
