Solid Biosciences wins FDA orphan status for muscle disorder therapy

hapabapa Solid Biosciences ( NASDAQ: SLDB ) traded higher premarket Friday after a federal register indicated that the U.S. FDA had granted Orphan Drug Designation for a treatment developed by the company for Duchenne muscular dystrophy (DMD), a muscle wasting disease. According to the agency, the treatment is identified as “Adeno associated virus serotype SLB101 containing the human microdystrophin gene” in generic terms.

With its orphan drug designation, the FDA aims to offer financial incentives to drug developers targeting rare diseases and conditions. In addition to tax credits for clinical trial costs and a waiver of the user fee for marketing applications, the developers can claim seven years of U.S. marketing exclusivity upon regulatory approval of the drug with the orphan indication.

Solid’s ( SLDB ) pipeline includes Duchenne gene transfer candidates SGT 003 and SGT 001. The former received FDA Fast Track designation for DMD in December. In April 2022, the company announced it had concluded enrollments for its IGNITE DMD Phase 1/2 clinical trial for SGT 001 in DMD. More on Solid Biosciences Solid Biosciences raises $109M through private placement Solid Biosciences receives FDA Fast Track designation for Duchenne gene therapy candidate Seeking Alpha’s Quant Rating on Solid Biosciences Historical earnings data for Solid Biosciences Financial information for Solid Biosciences.