The Expanded Access Program for BHN-0223 is thought to be the first EAP approved by the FDA in ALS which aims to offer broad access to an experimental medication outside of a traditional clinical trial.
Biohaven Pharmaceuticals announced the initiation of an Expanded Access Program (EAP) in ALS for their orally dissolving tablet of riluzole. This program is thought to be the first EAP approved by the FDA in ALS which aims to offer broad access to an experimental medication outside of a traditional clinical trial to upwards of 250 people with ALS, while the company prepares to submit an application for full marketing approval to the FDA later this year.
BHV-0223 is a tablet version of riluzole that dissolves almost instantly after being placed under the tongue (sublingual). While riluzole was approved as a treatment more than 25 years ago, there may be people with ALS who have not been able to access it, due to their inability to swallow pills, or their concerns about liver toxicity. This new formulation of riluzole attempts to address both of those issues.
Since BHV-0223 is a tablet that dissolves almost immediately when placed under the tongue, a person with ALS who is not taking riluzole because they cannot swallow pills will have an option to try. And, for those who have been advised by their physician against taking riluzole due to worries regarding how it may impact their liver function, BHV-0223 may be an option as smaller doses are required for the same bioavailability.
Following those early stage clinical trials, all conducted with individuals who did not have ALS, the company received guidance from the FDA that additional large-scale trials would not be required prior to submitting an application. A small, open-label clinical trial in ~20 people with ALS is currently enrolling, and at the same time, the company is offering people with ALS access through its EAP.
The EAP is being managed by a third party called Open Access Care. Only a physician can request a patient be included in the EAP. To do so the doctor must email or call Open Access Care using information found here.
BHV-0223 was the #2 item discussed by ALS TDI in its January webinar entitled The Top 10 Things to Watch in ALS Research in 2018, which can be viewed here.
Note: This article was updated to include a link and other information from the EAP protocol as it appeared on clinicaltrials.gov at the time of its publication there on May 28, 2018.