FDA Grants Cytokinetics Treatment Orphan Status
SOUTH SAN FRANCISCO, Calif. (AP) — Biotechnology company Cytokinetics Inc. said Wednesday regulators have granted "orphan drug" status to its potential Lou Gehrig's disease treatment.
The company said it plans to start a mid-stage clinical trial in the first half of this year to test the drug's effectiveness in patients with the disease.
Lou Gehrig's disease is a fatal neurological disorder that steals the body's ability to move, speak and ultimately to breathe. Also known as amyotrophic lateral sclerosis or ALS, it currently has no cure or treatment that halts or reverses the condition.
Cytokinetics said the disease affects between 20,000 and 30,000 people in the United States. The average life expectancy for a patient is about three to five years.
The Food and Drug Administration gives orphan drug status to drugs aimed at rare conditions or conditions that have a lack of treatments on the market. Incentives that come with it include seven years of market exclusivity following FDA approval, assistance in clinical trial design, a reduction in user fees, and tax credits.
A product can be designated an orphan drug if it is meant to treat an illness than affects fewer than 200,000 people.