Portage Biotech and Biohaven Pharmaceuticals recently announced they have received positive feedback from the U.S. Food and Drug Administration (FDA) on their Pre-Investigational New Drug Application (PIND) interaction regarding Biohaven’s investigative drug BHV-0223, intended for treatment of amyotrophic lateral sclerosis (ALS).
BHV-0223 is the glutamate modulating compound riluzole, contained in the tablet formulations Zydis ODT fast-dissolve technology. The drug will be produced under an exclusive worldwide agreement with Catalent, which makes Zydis.
Glutamate modulating drugs are of interest for a number of diseases in addition to ALS, such as Alzheimer’s disease, Rett syndrome, dementia, dystonia, tinnitus, anxiety disorders, and affective disorders.
The formal correspondence from the FDA came after an in-person meeting, and detailed clinical, nonclinical, and regulatory issues for the drug. The feedback correspondence also discussed the trial design of the Investigational New Drug (IND) opening clinical trial and a proposed development plan for ALS.
In a press release, Biohaven says the FDA response contained no issues that could delay the planned bioequivalence trial in 2016. The FDA also agreed that Biohaven does not need to provide additional efficacy or toxicology studies before submission of the New Drug Application. The FDA also agreed that for ALS, the 505(b)2 regulatory pathway is acceptable for BHV-0223. The pathway is a faster track than a full application, and is usually reserved for new products with well-studied ingredients.
“We now have a clear regulatory path forward for BHV-0223 in ALS. The responses we received from FDA were aligned with our expectations, and no material issues were raised that would delay the timely initiation of our bioequivalence study,” said Vlad Coric, CEO of Biohaven, in the press release.
“Our clinical program will expand upon our existing data with BHV-0223 and the goal will be to establish bioequivalence to the active pharmaceutical ingredient (riluzole) with lower doses of our new formulation. After we establish bioequivalence in the upcoming trial and demonstrate the advantages of this formulation to patients, we will be in position for a timely NDA submission. We believe that the enhanced formulation, dosage and route of administration of BHV-0223 will benefit patients with this devastating disease,” Coric said.
Declan Doogan, CEO of Portage and chairman of Biohaven, said, “The Biohaven team continues to demonstrate their ability to develop and execute on their strategy for BHV-0223. Biohaven will next take steps to prepare for an anticipated commercialization of the investigational agent upon successful results from the planned bioequivalence study. This will allow Biohaven to progress rapidly into the clinic and then to the market after NDA approval. The obligations are much less than a standard new drug application. We are very pleased with Portage investment in Biohaven, a company with a great team and an exciting product line.”