Ractigen’s ALS Drug Candidate Gains Orphan Drug Designation

The FDA has granted Orphan Drug status to Ractigen Therapeutics’ RAG-17, an investigational treatment of patients with amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease.

RAG-17 is a candidate small interfering RNA (siRNA) therapy designed to reduce expression of the SOD1 enzyme in ALS patients who carry certain gene mutations.

Several preclinical studies have shown RAG-17’s potential as a treatment for this patient population, showing significant potency, the company said.

The FDA’s Orphan Drug designation is for drugs and biologics intended to treat a rare disease or condition that affects fewer than 200,000 patients in the U.S. The designation gives companies incentives such as seven-year marketing exclusivity from the date of FDA approval and a waiver of user fees.