Biohaven Establishes Expanded Access Program for Investigational ALS Therapy BHV-0223

Biohaven Pharmaceuticals has established an expanded access program (EAP) for patients with amyotrophic lateral sclerosis (ALS) for its investigational treatment candidate, BHV-0223, the company recently announced.

The United States Food and Drug Administration regulates the expanded access program to help provide access to therapies that are currently in development and have not yet received regulatory approval for marketing. The program was established to help eligible patients with serious or life-threatening conditions get quicker access to as-yet unapproved therapies for which there is an unmet need.

“It is great for the ALS community that Biohaven is taking advantage of the FDA’s expanded access program,” Calaneet Balas, president and CEO of The ALS Association, said in a press release. “We believe this is the first instance of a company offering expanded access for widespread use in ALS. Such programs enable people with ALS and their doctors to consider the use of investigational drugs.”

BHV-0223 contains the active ingredient riluzole, which is the only approved therapy for ALS shown to prolong survival. A 40-mg dose of BHV-0223 provides the same systemic exposure as a 50-mg Rilutek tablet, allowing for a  20 percent lower daily dose.

BHV-0223 was created using Zydis technology, developed by Catalent, which allows for the tablet to be administered under the tongue where it dissolves within seconds. This is particularly useful for a lot of ALS patients who develop dysphagia, or trouble swallowing. Even patients with mild dysphagia can have issues with swallowing medications.

“Recognizing that many ALS patients with dysphagia may be unable to take conventional riluzole tablets for reasons that include difficulty with swallowing, Biohaven is pleased to be able to offer patients living with ALS access to sublingual BHV-0223 via this expanded access program while we prepare to submit our NDA [new drug application],” said Vlad Coric, MD, CEO of Biohaven.

Biohaven expects to submit an NDA for BHV-0223 to the FDA in the third quarter of 2018.

“For those patients with significant dysphagia, waiting for the approval of sublingual BHV-0223 may mean never getting the opportunity to take this drug and receive the benefits of riluzole [Rilutek], the only drug for ALS known to impact survival.  We are happy to help address this pressing need within the FDA’s EAP framework,” Coric said.

Biohaven collaborated with Catalent’s Swindon, U.K., division to develop the new BHV-0223 Zydis formulation of riluzole. Additionally, Biohaven has an exclusive agreement with Catalent for the use of the Zydis technology and the active ingredient riluzole.

“Sublingual BHV-0223 ZYDIS was specifically designed for ALS patients who have difficulty swallowing tablets and to offer a lower dose version of the currently approved active drug ingredient. We understand the urgency of patients’ needs and while the EAP is ongoing, Biohaven will be diligently working toward submitting an NDA in the next quarter,” said Rob Berman, MD, chief medical officer of Biohaven.