Biohaven: FDA Accepts Filing for ALS Treatment

– If approved, BHV-0223 would become the only formulation of riluzole that does not require swallowing tablets or liquids, offering an important delivery alternative for the standard-of-care treatment of ALS –
– Riluzole is the only FDA-approved treatment for ALS shown to extend tracheostomy-free survival-   

Biohaven Pharmaceutical Holding Company Ltd. (NYSE: BHVN) today announced that its 505(b)(2) new drug application (NDA) for BHV-0223, an investigational sublingual form of riluzole for the treatment of people living with amyotrophic lateral sclerosis (ALS), has been accepted for review by the U.S. Food and Drug Administration (FDA). Biohaven previously announced that the FDA granted orphan drug designation for BHV-0223 in ALS.  BHV-0223 is a novel, lower dose formulation of riluzole designed to be placed under the tongue where it dissolves in seconds and is absorbed sublingually. The active ingredient, riluzole, is the only FDA-approved treatment to extend tracheostomy-free survival in people living with ALS.

“We are extremely proud to announce the FDA acceptance for review of our 505(b)(2) NDA submission of BHV-0223, as it represents Biohaven’s first NDA. If approved, we believe BHV-0223 would provide an important advancement in treatment for people living with ALS, including the large number of patients who have difficulty swallowing tablets or liquids,” said Robert Berman, M.D., Chief Medical Officer of Biohaven. “We are committed to designing new treatment options for life-altering neurological diseases and believe that our sublingual, orally-dissolving tablet formulation of riluzole can provide a meaningful alternative for people living with ALS.”

ALS is a progressive neurodegenerative motor neuron disease affecting nerve cells in the brain and the spinal cord in which patients experience muscle weakness, trouble swallowing and muscle atrophy that ultimately progresses to paralysis, impaired breathing and death. More than 80% of people living with ALS experience dysphagia, which is difficulty swallowing, during their disease course.

The 505(b)(2) NDA submission is supported by data from multiple studies and clinical trials evaluating the bioequivalence of BHV-0223 to riluzole oral tablets (Rilutek®), as well as the safety and tolerability of BHV-0223. Key findings from the clinical program showed that sublingual administration of BHV-0223 (40 mg) achieved similar blood exposures to orally ingested Rilutek (50 mg). In these studies and trials, BHV-0223 was generally well tolerated. Patients also reported the sublingual formulation easy to use in clinical trials in which BHV-0223 was administered to patients with ALS and dysphagia.

https://www.biospace.com/article/releases/biohaven-announces-fda-acceptance-of-505-b-2-nda-filing-for-bhv-0223-a-novel-sublingual-delivery-form-of-riluzole-for-the-treatment-of-amyotrophic-lateral-sclerosis-als-/