PYC Therapeutics (ASX:PYC) has secured a significant regulatory milestone in Europe, with the European Medicines Agency granting Orphan Drug Designation to its investigational therapy VP-001, a candidate targeting the rare blinding condition Retinitis Pigmentosa type 11.
The company said VP-001 has the potential to become the first approved treatment for patients with RP11, a genetic eye disease with no available therapies.
The designation by the EMA is intended to support the development of medicines for rare diseases and brings a series of regulatory and commercial incentives. These include ten years of market exclusivity in Europe upon approval, access to scientific advice from the regulator to guide clinical trial design, and reduced fees during the review process.
The European decision builds on a series of designations already granted to VP-001 in the United States, where the Food and Drug Administration has provided Orphan Drug Designation, Fast Track Status and Rare Pediatric Disease Designation.
PYC, a clinical-stage biotechnology company focused on genetic diseases, is currently progressing VP-001 through a Phase 2 study. The company said the EMA decision highlights both the unmet need in RP11 and the potential for the therapy to deliver meaningful benefits to patients.
The development program is continuing alongside engagement with regulators. PYC recently held a Type D meeting with the FDA to align on requirements for a future New Drug Application and is now awaiting further data from its ongoing Phase 2 trial to refine the design of a registrational study.
An update on long-term follow-up data from the Phase 2 study is expected later this year.