UPDATE: On November 26, 2018, Biohaven announced that the FDA accepted its new drug application (NDA) to review BHV-0223.
Biohaven Pharmaceuticals, Inc. initiated an expanded access program (EAP) of BHV-0223 drug, a sublingual, lower dose formulation of Rilutek® (riluzole). The drug uses a Zydis® orally dissolving tablet technology, which does not require swallowing or additional fluids. Riluzole is the first drug approved by the U.S. Food and Drug Administration to treat ALS. It prolongs life approximately three months.
“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.”
Through the expanded access program, physicians will be able to obtain BHV-0223 for eligible people with ALS at no cost. Biohaven is committed to a fair and impartial evaluation of each request for access to the program. No one is guaranteed entry, so physicians must apply on each person’s behalf. Read more information about the Biohaven EAP and how to apply here.
By definition, expanded access programs, which are regulated by the FDA, provide access to drugs in development, prior to regulatory approval for marketing, to eligible people with life-threatening diseases, like ALS, for which there are limited drug alternatives. For example, EAPs allow people to receive investigational medicines following a clinical trial they participated in before the drug is FDA approved. EAPs are also helpful for people who did not meet the criteria for the clinical trial or if a clinical trial is not currently available.
BHV-0223 is designed with people in mind who have problems swallowing, called dysphagia. Many people with ALS either present with dysphagia upon diagnosis or experience dysphagia as the disease progresses.
BHV-0223 is a 40 mg drug taken twice a day that is an alternative formulation of riluzole. Differences between BHV-0223 and riluzole include the following:
- It provides a bioequivalent systemic exposure as Rilutek® 50mg tablets, but with a 20% lower total daily drug burden.
- It is a sublingual dissolvable tablet, meaning it is taken under the tongue, versus a pill.
- Due to its dissolvable action, it does not require swallowing or need to be taken with liquid.