BEDFORD, Mass., and Suzhou, China, January 23, 2024 — Frontera Therapeutics announces that its pioneering AAV gene therapy product, FT-002, has received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA) for the treatment of Inherited Retinal Dystrophies (IRD) caused by RPGR gene variants.
About Orphan Drug Designation
Orphan Drug Designation, granted by the FDA Office of Orphan Product Development (OOPD), recognizes FT-002 as an eligible drug for the prevention, treatment, and diagnosis of rare diseases. This designation brings several benefits, including tax credits for clinical trial fees (up to 25%) conducted in the United States, waivers for BLA/NDA application fees, and a 7-year market exclusivity period post drug approval.
FT-002 is a groundbreaking recombinant adeno-associated virus (AAV) gene therapy developed by Frontera to address IRD caused by RPGR gene mutations. With no effective treatments available for this disease globally, FT-002 represents a significant advancement. Administered as an intraocular injection of rAAV, the therapy carries the target gene, enabling retinal cells to express active functional proteins, thereby repairing damaged retinal cell structure and function.
The safety, tolerability, and preliminary efficacy of FT-002 is under investigation in an ongoing investigator initiated clinical study (NCT05874310) in patients with RPGR gene mutation associated X-linked Retinitis Pigmentosa (XLRP). Enrollment has been complete for this study with 18 subjects enrolled from two investigational sites and received one time FT-002 administration in a dose ascending manner. FT-002 is well tolerated in all patients, with no dose-limiting toxicity observed. Improved retinal sensitivity and visual function were seen in the medium and high dose group assessed by microperimetry, full-field stimulus threshold (FST) or mobility test up to the most recent visit (Day 84-168).
Cleared by CDE on November 6, 2023, FT-002 has progressed to Phase I/II clinical studies.