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.
Sarepta Therapeutics Inc. made known a third gene therapy death, this time with SRP-9004 for limb-girdle muscular dystrophy. The patient was a late-stage, non-ambulatory 51-year-old man participating in the phase I Discovery trial, who expired about a month ago of acute liver failure, as did the two previous subjects who passed away after they were treated with Elevidys (delandistrogene moxeparvovec), Cambridge, Mass.-based Sarepta’s gene product for Duchenne muscular dystrophy.
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.
A total of 33 states, plus the District of Columbia and Puerto Rico, agreed to participate in the U.S. CMS’ voluntary, outcomes-based program aimed at helping state Medicaid programs cover high-priced cell and gene therapies, starting with therapies for sickle cell disease.
Under a new licensing deal announced July 8, JCR Pharmaceuticals Co. Ltd. granted Alexion Pharmaceuticals Inc. rights to its adeno-associated virus (AAV) capsids for use in up to five of Alexion’s genomic medicines programs.
Manufacturing issues are the latest problem for Ultragenyx Pharmaceutical Inc. to solve after last week’s disappointment in a phase III study to treat brittle bones. The U.S. FDA gave the company a complete response letter (CRL) regarding the BLA for its gene therapy to treat Sanfilippo syndrome type A, saying it needs more details and improvements made about CMC after having finished manufacturing facility inspections.
Under a new licensing deal announced July 8, JCR Pharmaceuticals Co. Ltd. granted Alexion Pharmaceuticals Inc. rights to its adeno-associated virus (AAV) capsids for use in up to five of Alexion’s genomic medicines programs.
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.
Using informed consent to do what Congress couldn’t, the U.S. FDA is flexing its regulatory authority to halt clinical trials that involve sending cells from American patients to China or other adversarial nations for genetic engineering and subsequent infusion back into the patient.
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