Biohaven Receives Orphan Drug Designation for ALS Treatment

Biohaven, the biopharmaceutical company, has received orphan drug designation from the FDA for BHV-0223, its drug candidate designed to treat amyotrophic lateral sclerosis (ALS). The orally dissolving tablet is a formulation of riluzole, specifically designed for individuals who may have difficulty swallowing.

Orphan drug designation indicates that BHV-0223 may potentially be able to treat a rare disease or disorder affecting fewer than 200,000 people in the United States.

BHV-0223 is designed to be absorbed through the oral mucosa rather than the gastrointestinal system. As a result, there is a reduced risk of negative effects due to the mixing of food and riluzole.

"Patients with ALS develop a wide range of disabilities with the vast majority developing significant difficulty swallowing,” said Robert Berman, MD, CMO of Biohaven, in a press release. “Eventually, most muscles under voluntary control are affected, and individuals lose their strength and the ability to move their arms, legs, and body. As a sublingually administered and orally dissolving tablet form of riluzole, we believe that BHV-0233 may offer important advantages to ALS patients."

"Receiving the orphan drug designation request approval for BHV-0223 in the treatment of ALS advances our global development strategy and one of our primary goals of providing therapies for patients suffering from neurologic disorders with high unmet need,” said Vlad Coric, MD, CEO of Biohaven, in a press release.

The most common adverse effects associated with riluzole are nausea, weakness, and vomiting. Less common effects are bladder pain, bloody urine, blurred vision, and flu-like symptoms. The recommended dosage for Riluzole is 50-mg twice per day.