— Agency customers question micro-dystrophin as a surrogate endpoint
by Judy George, Deputy Managing Editor, MedPage Today
May 10, 2023
Scientific research studies to date “do not offer unambiguous proof” for SRP-9001 (delandistrogene moxeparvovec), an investigational gene treatment for ambulatory Duchenne muscular dystrophy clients, FDA customers stated in rundown files launched ahead of an advisory committee conference.
The company likewise raised security issues about the possibility of administering an inefficient gene treatment.
Duchenne muscular dystrophy is defined by an anomaly that results in an absence of dystrophin and muscle loss. It impacts about one in 3,300 young boys and has no recognized remedy.
SRP-9001 is developed to provide a gene that codes for a reduced type of dystrophin, called micro-dystrophin, to assist protect muscle. To get approved for sped up approval, Sarepta Therapeutics proposed expression of micro-dystrophin– an unique, crafted protein– as a surrogate endpoint.
On Friday, the FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee will fulfill to talk about whether micro-dystrophin levels 12 weeks after SRP-9001 treatment can work as a surrogate endpoint that the gene treatment is fairly most likely to anticipate medical advantage.
Corticosteroids are the main pharmacologic treatment for Duchenne. In addition, 4 exon-skipping drugs– consisting of 3 from Sarepta– have actually gotten sped up FDA approval for clients with particular DMD anomalies; their scientific advantage has actually not yet been validated.
The objective of SRP-9001