EL-PFDD Meeting For Six Subtypes of LGMD

---

On September 23, 2022, a coalition of limb-girdle muscular dystrophy (LGMD) organizations held an Externally-Led Patient Focused Drug Development (EL-PFDD) meeting for six LGMD subtypes: 2C (R5), 2D (R3), 2E (R4), 2F (R6), 2A (R1), and 2i (R9). The LGMD Coalition includes the following organizations: Coalition to Cure Calpain 3, CureLGMD2i, Kurt+Peter Foundation, LGMD2D Foundation, McColl-Lockwood Laboratory for Muscular Dystrophy Research, and the Speak Foundation. 

The goal of this meeting was to provide the U.S. Food and Drug Administration (FDA), product developers, clinicians, industry, and academic researchers an opportunity to hear perspectives from individuals with LGMD on the health effects, daily impacts, treatment goals, and decision factors considered when seeking out or selecting a treatment. This meeting was  conducted as a parallel effort to FDA’s EL-PFDD initiative, a commitment under the fifth authorization of the Prescription Drug User Fee Act (PDUFA V) to more systematically gather patients’ perspectives on their conditions and available therapies to treat their conditions.

Please note that this initial endeavor represented Phase 1 and included six LGMD subtypes.  We anticipate additional LGMD subtypes will pursue EL-PFDD meetings in the future.