Biohaven Pharmaceuticals Announces FDA Has Accepted Its 505(b)(2) New Drug Application to Review BHV-0223 for Treatment of ALS

On Nov. 26, Biohaven Pharmaceuticals announced that the Food and Drug Administration (FDA) has accepted its 505(b)(2) New Drug Application (NDA) to review BHV-0223, a sublingual (placed under the tongue) formulation of riluzole for the treatment of patients living with amyotrophic lateral sclerosis (ALS). In submitting its NDA using the 505(b)(2) pathway, Biohaven was able to utilize a more streamlined development and approval process to evaluate BHV-0223 given that riluzole is already approved by the FDA.

If approved, BHV-0223 would be the first formulation of riluzole to be approved by the FDA that does not require swallowing. Riluzole is currently the only FDA-approved treatment for ALS that is shown to extend tracheostomy-free survival.

The tablet, placed under the tongue, would dissolve within seconds and be absorbed into the bloodstream, serving as an important delivery alternative for patients who have difficulty swallowing or who no longer are able to swallow. Many people with ALS develop weakness in the muscles of the face and throat that can cause problems with swallowing, chewing, drinking and speaking. This weakness can lead to choking and aspiration, as well as difficulties in taking medications in tablet form. While some people may crush pills to continue to take medications, it is not known whether crushing the pills alters effectiveness.

The 505(b)(2) NDA application contained data from multiple trials of BHV-0223 that showed a bioequivalence with riluzole oral tablets, meaning that BHV-0223 produced the same effect and was used by the body in a similar way as riluzole. In addition, BHV-0223 has shown acceptable safety and tolerability profiles.

To read Biohaven’s press release, click here.