Biohaven Pharmaceuticals has developed a novel sublingual formulation of riluzole, BHV-0223, for the treatment of ALS. BHV-0223 (sublingual) dissolves under the tongue in seconds and effectively delivers riluzole into the bloodstream through sublingual absorption. Clinical studies demonstrate that BHV-0223 (sublingual) requires a lower dose of riluzole (40mg) than the tablet (50mg) to achieve the same blood levels.1
BHV-0223 is still an investigational drug. FDA approval and commercial availability are anticipated in mid- to late 2019. The company has opened an Expanded Access Program (EAP) in an effort to bring this investigational treatment option to People with ALS (PALS) today. [www.ClinicalTrials.gov (NCT03537807)]
Riluzole is the only FDA-approved treatment demonstrated to extend time to tracheostomy and survival in ALS.[Rilutek USPI] Reports of tolerability issues with swallowing riluzole may result in premature discontinuation or altering its delivery (e.g. crushing and taking with food or chewing), reducing the potential benefit of riluzole.2
BHV-0223 sublingual formulation of 40mg bypasses gastrointestinal absorption, avoids initial high concentration first pass liver metabolism and provides an ease of administration (no swallowing or liquids required) for patients with dysphagia.
Value of BHV-0223 Sublingual Early Access Program
The FDA works with pharmaceutical companies to allow patients access to investigational drugs outside of the clinical trial setting before approval through an early access program.
The BHV-0223 (sublingual) EAP offers an opportunity for PALS to try a sublingual formulation of riluzole at no cost to the patient or institution. It will allow an opportunity to collect serial clinical data from a broader ALS patient population, to evaluate patients' views on ease of administration, tolerability, safety and clinical benefits of BHV-0223. [www.ClinicalTrials.gov (NCT03537807)]
The protocol has been reviewed by the FDA and approved by an IRB. Click here for additional information about the BHV-0223 protocol.
If you are a physician and are interested in enrolling patients in the BHV-0223 Expanded Access Protocol for ALS, please contact Early Access Care at:
Phone: 1-(203) 441-7938 (option 6).
A brief webinar, hosted by the ALS Association, provides an overview of the Expanded Access Program. Dr. Richard Bedlack MD, PhD, Director of Duke ALS Clinic, Duke University provides insights into the Expanded Access Program. The webinar is available here.
1 Qureshi et al. A Phase 1 Study to Evaluate Bioequivalence Between BHV-0223 40mg Zydis Sublingual Formulation and Riluzole 50mg Oral Tablet in Healthy Volunteers. Poster presented at American Academy of Neuromuscular and Electrodiagnostic Medicine (AANEM) Clinical Conference; 2018 Oct 9-12, Washington D.C. [Poster]
2Introna et al. Adherence to ALS treatments. Neuropsychiatric Disease and Treatment 2018;14:193-203.