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news.US-based pharmaceutical firm Retrophin has received orphan drug from the US Food and Drug Administration (FDA) for RE-024 to treat pantothenate kinase-associated neurodegeneration (PKAN), a rare and lethal autosomal recessive neurodegenerative disorder.
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PKAN is believed to affect about one to three persons per million worldwide and there are currently no approved treatment available.
The company has started the screening of healthy volunteers for its recently announced Phase I trial evaluating the safety and tolerability of single oral doses of its investigational phosphopantothenate replacement therapy, RE-024.
The orphan status will provide RE-024 with seven years of marketing exclusivity if approved by the FDA for the treatment of PKAN.
Retrophin is focused on developing, acquiring and commercializing drugs to treat serious, catastrophic or rare diseases for which there are currently no viable options for patients.
Approved products of the company include Chenodal, Cholbam and Thiola, while its pipeline includes compounds for several catastrophic diseases, including focal segmental glomerulosclerosis (FSGS), pantothenate kinase-associated neurodegeneration (PKAN), infantile spasms, nephrotic syndrome and others.