Gene Therapy for Limb-Girdle Muscular Dystrophy Type 2i/R9 Receives Fast Track Designation 

Gene therapy (LION-101; AskBio, Research Triangle Park, NC) for the rare type 2I/R9 limb-girdle muscular dystrophy (LGMD), in phase 1/2 clinical trials, has received fast-track designation from the Food and Drug Administration (FDA). The gene therapy, a copy of the fukutin-related protein (FKRP) gene, is being developed as a 1-time intravenous infusion of a novel recombinant adeno-associated virus (rAAV) based vector. Mutations in FKRP are known to cause LGMD type 2I/R9.

Participants in the trial will be adolescents and adults with genetically confirmed LGMD 2I/9R. Initial dosing is expected to begin in the first half of 2022. LGMD 2I/R9 is a congenital muscular dystrophy with late childhood onset of symptoms that progressively limit mobility. Most individuals with LGMD 2I/R9 need to use a wheelchair within 23 to 26 years of onset.

Fast-track designation is designed to expedite review of new therapies intended to treat serious conditions with unmet medical needs. Fast-track treatments that reach the approval stage are eligible for the accelerated approval and priority review pathway. There are no disease-modifying treatments for LGMD 2I/9R available to date. 

"The FDA Fast Track Designation for LION-101 is an important step for the development of this program and is a clear recognition of the profound burden faced by LGMD2i/R9 patients," said Sheila Mikhail, Co-Founder & CEO, AskBio. "We look forward to initiating clinical trials with this novel therapy, and we hope to bring a new therapeutic option to patients and families in the LGMD2I/R9 community who live with this devastating disease."