fda grants clinuvel orphan drug designation

The US Office for Orphan Products Development (OOPD) issues designation for the use of photoprotective afamelanotide (CUV1647) in the treatment of erythropoietic porphyrias.
Clinuvel Pharmaceuticals Limited (ASX: CUV; XETRA-DAX: UR9; ADR: CLVLY) is pleased to announce that its photoprotective drug afamelanotide (CUV1647) has been granted orphan-drug designation (ODD) by the US Food and Drug Administration (FDA) for the treatment of erythropoietic porphyrias.
Pursuant to the Federal Food, Drug, and Cosmetic Act the FDA has awarded Clinuvel the right to utilize afamelanotide in the management of erythropoietic porphyrias, metabolic disorders causing phototoxicity affecting less than 200,000 patients in the United States.
The FDA’s orphan-drug designation is reserved for new drugs or therapies being developed to treat rare diseases or conditions that affect smaller populations in the United States. The orphan-drug designation allows for an accelerated review process by the FDA, seven-year market exclusivity in the United States upon obtaining marketing authorization, tax benefits, and exemption from user fees.
Clinuvel is developing its photoprotective drug afamelanotide as a prophylactic treatment for a range of UV-related skin disorders as well as cancer related treatments. Trials are underway in connection with five indications including erythropoietic protoporphyria.
Clinuvel’s CEO, Dr Philippe Wolgen said:
“US FDA orphan-drug designation marks a significant moment in our Company. It is the first time we have obtained regulatory recognition in the US and it follows earlier acknowledgement in Europe when ODD designation was received from the European Medicines Agency (EMEA) and Swissmedic.”
Clinuvel’s CSO, Dr Hank Agersborg said:
“Having regulatory recognition is a vital step towards our goal to make afamelanotide available as a prescriptive drug for severely affected patients, but ultimately, ongoing safety and clinical results will determine our regulatory success in developing afamelanotide”
“The next major step in the regulatory process will be for afamelanotide to obtain US FDA Investigational New Drug (IND) status allowing Clinuvel to conduct trials in the United States. Advanced trials are currently progressing in Europe and Australia in relation to Erythropoietic Protoporphyria (EPP).”