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

How BHV-0223 works

Riluzole is a glutamate-modulating agent. Its exact mechanism of action is unknown, but it is believed to block the release of glutamate from nerve cells. Glutamate is a neurotransmitter, a key molecule that transmits information from one nerve cell to another and plays a crucial role in normal brain function.

One of the causes of nerve damage in ALS is believed to be glutamate dysfunction where toxic levels of glutamate are released and damage the surrounding nerve cells. Glutamate-modulating agents are thought to have neuroprotective properties in diseases such as ALS, as they may prevent glutamate from reaching damaging levels.

This new formulation of riluzole intends to address some of the problems associated with using the therapy, such as swallowing difficulties, by using Zydis ODT (orally dissolving tablet) technology developed by Catalent. BV-0223 is designed to be placed under the tongue, where it will quickly dissolve and be absorbed. Because it is absorbed by the oral mucosa (the lining of the mouth) rather than by the gastrointestinal system, researchers believe BHV-0223 also will prevent the negative effects associated with pill-form riluzole, such as having to take it on an empty stomach and avoiding caffeinated products.

BHV-0223 in clinical trials

Biohaven Pharmaceuticals successfully completed a Phase 1 study to test the pharmacokinetics, safety, and tolerability of BHV-0223 in 10 patients, with promising results. Biohaven expects to start a pivotal bioequivalence study in 2017 comparing the sublingual formulation of riluzole to the oral tablet. If the results are positive, the study will be used to support a new drug application (NDA) seeking U.S. Food and Drug Administration (FDA) approval for BHV-0223 as an ALS treatment. According to the company, the new formulation will provide another therapeutic option to patients living with ALS.

Next steps for BHV-0223

The FDA granted orphan drug designation to BHV-0223 in 2016. This designation is given to therapies and biologic products intended to treat, diagnose, or prevent rare diseases or disorders, or those that affect fewer than 200,000 people in the U.S. It also gives the therapy’s developer benefits and incentives including a period of market exclusivity.

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