BHV-0223 is a new and sublingual formulation of riluzole, meaning it is placed under the tongue to dissolve in the mouth. The drug is being developed by Biohaven Pharmaceuticals for the treatment of amyotrophic lateral sclerosis (ALS).

How BHV-0223 works

BHV-0223 is a sublingual formulation of the glutamate-modulating agent riluzole. Glutamate-modulating agents are thought to have neuroprotective properties in situations that involve glutamate dysfunction, such as ALS, and other neurological conditions. (Glutamate is a key molecule that transmits information from one nerve cell to another and plays a crucial role in normal brain function).

This new formulation of riluzole intends to address some of the problems associated with using the therapy, such as swallowing difficulties. It is designed to be absorbed by the oral mucosa (lining of the mouth) rather than the gastrointestinal system, so researchers believe that BHV-0223 will also eliminate the negative effects associated with pill-form riluzole, such as having to be taken on an empty stomach and avoiding caffeine-containing products.

Next steps for BHV-0223

The drug was granted orphan drug designation by the U.S. Food and Drug Administration (FDA) in 2016. The FDA designation is given to drugs and biologic products intended to treat, diagnose or prevent rare diseases or disorders, meaning those that affect fewer than 200,000 people in the United States. It also gives a drug developer benefits and incentives, including a period of product exclusivity.

Biohaven Pharmaceuticals completed a pharmacokinetic study with BHV-0223  in humans and expects to start a pivotal bioequivalence study in 2017, comparing the sublingual to oral riluzole, to support a new drug application (NDA), seeking approval of BHV-0223 as an ALS treatment. (Riluzole, approved by the FDA in 1995, remains the only approved ALS treatment.) According to the company, the new formulation will provide another therapeutic option to patients living with ALS.

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