Clinuvel Pharmaceuticals Ltd (ASX:CUV) has achieved a major milestone in the U.S. after the FDA granted a Fast Track designation for the company's SCENESSE treatment of erythropoietic protoporphyria (EPP).
SCENESSE prevents phototoxicity in adults with EPP, a condition in which light exposure elicits a deep burning reaction in the patients’ skin.
SCENESSE, delivered via a subcutaneous dissolving implant the size of a grain of rice, shields against UV radiation and sunlight by increasing the levels of melanin in the skin.
The Fast Track designation enables Clinuvel to file a New Drug Application (NDA) on a rolling basis for U.S. regulatory assessment.
A pre‐NDA meeting will be scheduled shortly with the FDA to discuss the timing of the first filing of the scientific dossier, which will also be eligible for an abbreviated review time of about six months.
The progress for Clinuvel in the U.S. follows the commercial launch of SCENESSE in Europe last month.
SCENESSE has been accepted as a specialty hospital product by the Dutch authorities and holds the distinction of being the first ever approved standard of care for EPP.
This enables reimbursement of SCENESSE for all adult EPP patients in the Netherlands. Insurers in Germany and Austria have also facilitated access to SCENESSE for EPP patients.
An estimated 45 million patients worldwide suffer from light-sensitive skin disorders that make them potential candidates for SCENESSE.
Clinuvel is well funded with $14.2 million in cash as at 31 March 2016.
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