This is the Company's third orphan drug designation request granted by the FDA.
ALS is a progressive neurodegenerative motor neuron disease that affects nerve cells in the brain and the spinal cord. The disease belongs to a group of disorders known as motor neuron diseases, which are characterized by the gradual degeneration and death of motor neurons.
ALS affects up to 20,000 individuals in the United States and typically presents in patients with painless muscle weakness, trouble swallowing and muscle atrophy that ultimately progresses to paralysis and death. Since the FDA's approval of riluzole in 1995, there have not been further clinical improvements or advances in ALS drug therapeutics over the last two decades. Several therapies are currently in clinical trials.
BHV-0223 is a sublingually absorbed and oral dissolving tablet (ODT) formulation of riluzole. BHV-0223's novel formulation is designed to address some of the shortcomings associated with the solid oral dosage form of riluzole that ALS patients have difficulty swallowing.
Because BHV-0223 is designed to be systemically absorbed through the oral mucosa, rather than through the gastrointestinal system, the Company believes that it can eliminate the negative food effect associated with riluzole, bypass first-pass metabolism and deliver effective doses of the drug at lower concentrations, while also allowing sublingual absorption in patients who experience difficulty swallowing and eliminating the need for three hour fasting twice daily.
Biohaven CMO Robert Berman, M.D. said: “Patients with ALS develop a wide range of disabilities with the vast majority developing significant difficulty swallowing. Eventually, most muscles under voluntary control are affected, and individuals lose their strength and the ability to move their arms, legs, and body. As a sublingually administered and orally dissolving tablet form of riluzole, we believe that BHV-0233 may offer important advantages to ALS patients."
Biohaven CEO Vlad Coric, M.D. said: “Receiving the orphan drug designation request approval for BHV-0223 in the treatment of ALS advances our global development strategy and one of our primary goals of providing therapies for patients suffering from neurologic disorders with high unmet need.
If approved, this unique formulation will provide another therapeutic option to patients living with this devastating disease."
About BHV-0223
BHV-0223 is a novel formulation of a glutamate-modulating agent that utilizes the Zydis ODT fast-dissolve technology under an exclusive worldwide agreement with Catalent. Agents that modulate glutamate neurotransmission may have therapeutic potential in multiple disease states involving glutamate dysfunction, including ALS, ataxia, Alzheimer's disease, Rett syndrome, dementia, dystonia, tinnitus, anxiety disorders, and affective disorders like major depressive disorder.
Biohaven is pursuing the use of glutamate-modulating agents across several therapeutic indications. The Company intends to pursue regulatory approval of BHV-0223 for ALS in the United States under Section 505(b)(2) of the U.S. Federal Food, Drug and Cosmetic Act.
The FDA cleared the Company's investigational new drug application (IND) for BHV-0223 in August 2015, and Biohaven has completed a pharmacokinetic study with this drug candidate in humans and is planning to launch a pivotal bioequivalence study in 2017.