BlueRock Therapeutics (Bayer) lemiretprocel awarded FDA and EMA orphan designations
- FDA granted orphan drug designations for retinitis pigmentosa and cone-rod dystrophy.
- EMA granted orphan drug designations for syndromic and non‑syndromic inherited retinal disorders of the rod‑dominant phenotype and non‑syndromic cone‑dominant IRDs.
- Lemiretprocel (OpCT-001) is an investigational iPSC‑derived cell therapy in a Phase 1/2a CLARICO study targeting primary photoreceptor diseases.
- The therapy aims to replace lost retinal photoreceptor cells; it is investigational and its efficacy and safety have not been established.
Regulatory designations
Lemiretprocel (OpCT-001) has received orphan drug designations from both the U.S. FDA and the EU EMA. The FDA designations cover retinitis pigmentosa and cone‑rod dystrophy, while the EMA designations cover syndromic and non‑syndromic inherited retinal disorders of the rod‑dominant phenotype and non‑syndromic cone‑dominant IRDs.
Candidate and clinical programme
Lemiretprocel is an investigational induced pluripotent stem cell (iPSC)‑derived cell therapy being tested in a Phase 1/2a clinical study named CLARICO. It targets primary photoreceptor diseases, a subgroup of inherited retinal disorders that include retinitis pigmentosa, Usher syndrome and cone‑rod dystrophy, and aims to restore vision by replacing lost photoreceptor cells in the retina.
Status and safety
The therapy has not been approved by any regulatory authority; its efficacy and safety have not been established or fully evaluated. Lemiretprocel is the International Nonproprietary Name (INN) acknowledged by the World Health Organization for OpCT‑001.
Source: Bayer