Biohaven Pharmaceutical Holding has established an expanded access program (EAP) with sublingual BHV-0223, an investigational drug candidate, for patients with amyotrophic lateral sclerosis (ALS). Through this program, physicians may be able to obtain BHV-0223 for their eligible patients with ALS at no cost. BHV-0223 is a sublingual and lower dose formulation of riluzole which employs the 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 shown to prolong survival. Biohaven anticipates submitting a New Drug Application (NDA) for BHV-0223 to the FDA in the third quarter of 2018. Details regarding this EAP, including eligibility criteria, are available at www.earlyaccesscare.com/biohaven.
EAPs are regulated by the FDA for the purpose of providing 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.
“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.”
BHV-0223 40 mg is a Zydis sublingual tablet administered twice a day, that provides bioequivalent systemic exposures as Rilutek (riluzole) 50 mg tablets, but with a 20 percent lower total daily drug burden. Its use is expected to be particularly amenable for patients with trouble swallowing (dysphagia) as it is administered under the tongue, where it dissolves in seconds and does not require swallowing or administration with liquids.
Dysphagia is one of the most critical and debilitating symptoms associated with ALS. Approximately one-third of ALS patients present with some degree of dysphagia on diagnosis. As the disease progresses, a majority of patients with ALS will experience dysphagia, and many will experience severe dysphagia, requiring placement of a percutaneous endoscopic gastrostomy (PEG) tube to allow nutrition, fluids and/or medications to be administered directly into the stomach, bypassing the mouth and esophagus.
Even ALS patients with mild or moderate dysphagia who have not yet lost the ability to swallow have difficulty managing small oral boluses such as tablets through the swallowing process. Biohaven’s sublingual formulation obviates the need for swallowing tablets or additional liquids to wash down the tablets.
Vlad Coric M.D., CEO of Biohaven said, “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. 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.”
Biohaven worked in conjunction with Catalent U.K. Swindon Zydis Limited, a subsidiary of Catalent, Inc. to develop the new BHV-0223 ZYDIS formulation of riluzole. Biohaven has also entered into an exclusive agreement with Catalent for the use of the Zydis ODT formulation technology and the active pharmaceutical ingredient riluzole.
Rob Berman M.D., CMO of Biohaven added, “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.”
Filed Under: Drug Discovery