Bidridistrogene xeboparvovec is under clinical development by Sarepta Therapeutics and currently in Phase III for Limb-Girdle Muscular Dystrophy. According to GlobalData, Phase III drugs for Limb-Girdle Muscular Dystrophy does not have sufficient historical data to build an indication benchmark PTSR for Phase III. GlobalData uses proprietary data and analytics to create drugs-specific PTSR and LoA in the Bidridistrogene xeboparvovec LoA Report. Buy the report here.
GlobalData tracks drug-specific phase transition and likelihood of approval scores, in addition to indication benchmarks based off 18 years of historical drug development data. Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval.
Bidridistrogene xeboparvovec overview
bidridistrogene xeboparvovec (SRP-9003) is under development for the treatment of limb-girdle muscular dystrophy type 2E (LGMD2E). It is administered by intravenous and intramuscular routes. The drug candidate comprises of self-complementary recombinant adeno-associated virus serotype rhesus 74 (AAVrh.74) encoding human beta-sarcoglycan (SGCB) gene. It acts by targeting SGCB gene.
It was also under development for the treatment of limb-girdle muscular dystrophy type 2E (LGMD2E).
Sarepta Therapeutics overview
Sarepta Therapeutics (Sarepta) discovers and develops unique RNA-targeted medicines to treat rare diseases. The company develops its pipeline products using its multi-platform Precision Genetic Medicine Engine in gene therapy, RNA, and gene editing. The company’s platform is based on its pioneering work with phosphorodiamidate morpholino oligomer (PMO) chemistries. Its commercial products include Exondys 51, Vyondys 53, Elevidys and Amondys 45 indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene. Its pipeline product includes SRP-5051, SRP-9003 and SRP-9004 indicated for the treatment of DMD, limb-girdle muscular dystrophies (LGDMs) and other neuromuscular and central nervous system disorders. Sarepta is headquartered in Cambridge, Massachusetts, the US.
For a complete picture of Bidridistrogene xeboparvovec’s drug-specific PTSR and LoA scores, buy the report here.
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GlobalData’s Likelihood of Approval analytics tool dynamically assesses and predicts how likely a drug will move to the next stage in clinical development (PTSR), as well as how likely the drug will be approved (LoA). This is based on a combination of machine learning and a proprietary algorithm to process data points from various databases found on GlobalData’s Pharmaceutical Intelligence Center.
