Ride For Life: FDA grants orphan drug status to ALS treatment

Phytopharm PLC said the US Food and Drug Administration granted "orphan drug designation" to its treatment of amyotrophic lateral sclerosis.

PYM50018 is a patented, orally active, neuroprotective and neuroregenerative compound from the P59 programme. ALS, also known as Lou Gehrig's disease, is the most common motor neurone disease, Phytopharm said.

Orphan drug designation qualifies a product for possible financial incentives, including seven years of marketing exclusivity upon FDA approval, the potential of an expedited approval, and possible tax incentives for a company's investment in U.S. clinical research, Phytopharm noted.

Phytopharm has recently registered the trademark Myogane as a name for this product. In April it announced the successful completion of a phase I clinical study to evaluate the safety, tolerability and pharmacokinetic profile of PYM50018.