BHV-0223 Expanded Access Program Initiated for ALS

Biohaven Pharmaceutical has established an expanded access program (EAP) with sublingual BHV-0223 for patients with amyotrophic lateral sclerosis (ALS).

EAPs are regulated by the U.S. Food and Drug Administration (FDA) to provide access to drugs in development, prior to regulatory approval for marketing, to eligible patients with serious or life-threatening diseases or conditions for which there are insufficient pharmacologic alternatives. Through this program, physicians will be permitted to acquire the investigational drug candidate for their eligible patients with ALS at no cost.

BHV-0223 is a sublingual and lower dose formulation of riluzole, which engages Zydis orally dissolving tablet technology and does not require swallowing tablets or additional fluids. The active ingredient riluzole is the only approved drug therapy for ALS that has exhibited the ability to prolong survival.

The BHV-0223 40 mg tablet is to be administered twice a day, providing the bioequivalent systemic exposures as Rilutek (riluzole) 50 mh tablets, but with a 20% lower total daily drug burden.

"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,”said Vlad Coric M.D., CEO of Biohaven in a press release. “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, the only drug for ALS known to impact survival. We are happy to help address this pressing need within the FDA's EAP framework."

The drug is anticipated to be exceptionally amenable for patients with dysphagia, or trouble swallowing, as it is administered under the tongue, where it dissolves in seconds and does not require swallowing or administration with liquids. Dysphagia is regarded as one of the most critical and debilitating symptoms associated with ALS, as an estimated one-third of individuals with ALS present with some degree of it at diagnosis.

"It is great for the ALS community that Biohaven is taking advantage of the FDA's expanded access program," said Calaneet Balas, president and CEO of The ALS Association. "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."

In December 2016, Biohaven was granted Orphan Drug designation for BHV-0223 in ALS, and the company anticipates submitting a New Drug Application (NDA) for the therapy to the FDA in the third quarter of 2018.

Further details regarding this EAP, including eligibility criteria, are available at www.earlyaccesscare.com/biohaven.

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