OCU400 receives fourth orphan drug designation from FDA
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The FDA has granted a fourth orphan drug designation for OCU400 to treat PDE6B gene mutation-associated retinal diseases, according to an Ocugen press release.
The most recent designation targets retinitis pigmentosa caused by mutations in the PDE6B gene. RP stemming from this mutation leads to blindness by midlife and is characterized by the progressive loss of photoreceptors.
Ocugen’s modifier gene therapy platform allows ophthalmologists to address multiple diseases with a single therapy. Recently, OCU400 was granted an ODD to treat RHO mutation-associated retinal degeneration.
“I look forward to Ocugen commencing clinical trials for OCU400 next year and the potential of helping patients by restoring retinal integrity and function across a range of genetically diverse inherited retinal diseases including broad-spectrum RP,” Carl D. Regillo, MD, FACS, member of Ocugen’s retina scientific advisory board, said in the release.
Ocugen plans to begin two parallel phase 1/2 clinical trials next year targeting two inherited retinal diseases.
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