Clinical TrialsThe Phase 3 SELVA trial design reflects regulatory flexibility for ultra-rare conditions with no FDA-approved therapies, supported by Breakthrough Therapy, Fast Track, and Orphan Drug designations.
Market PotentialThe commercial opportunity for QTORIN in microcystic lymphatic malformations is compelling, with an estimated 20,000–30,000 addressable U.S. patients and no current standard-of-care.
Therapeutic DevelopmentPalvella Therapeutics is developing therapies for rare genetic skin diseases with significant unmet needs, which highlights a strong market potential.