Verdiperstat Receives Orphan Drug Designation from FDA for Multiple System Atrophy

Biohaven announced it received orphan drug designation from the U.S. Food and Drug Administration (FDA) for its product candidate verdiperstat (previously BHV-3241), a novel myeloperoxidase (MPO) inhibitor, for the treatment of multiple system atrophy (MSA).

Verdiperstat is a potential first-in-class, oral, brain-penetrant, irreversible inhibitor of MPO, an enzyme that acts as a key driver of pathological oxidative stress and inflammation in the brain. Verdiperstat also has received orphan drug designation for the treatment of MSA from the European Commission upon recommendation from the European Medicines Agency's Committee for Orphan Medicinal Products.

"We are very pleased the FDA granted orphan drug designation for verdiperstat. This highlights Biohaven's commitment to developing innovative therapies in areas of high unmet medical need," Irfan Qureshi, M.D., Biohaven Executive Director and development lead for verdiperstat, said. "We believe verdiperstat has the potential to be the first effective treatment for people living with MSA and we remain on track to start our global Phase 3 clinical trial later this year."

Verdiperstat completed Phase 1 clinical trials at doses up to 900mg twice a day and preliminary results from a Phase 2a trial in patients with MSA showed numerical improvements on the change from baseline Unified MSA Rating Scale. After 12 weeks of treatment, placebo-treated patients worsened by 4.6 points (SE=1.1, n=17) on the Unified MSA Rating Scale, while verdiperstat-treated patients worsened by 3.7 points (SE= 1.2, n=17) at the 300mg twice-daily dose and by 2.6 points (SE=1.4, n=18) at the 600mg twice-daily dose. Corresponding benefits were also observed in other outcome measures, such as the Composite Autonomic Symptom Score and MSA-Quality of Life scale. These clinical findings were consistent with neuroprotective effects of verdiperstat observed in animal models. In the Phase 2a trial, verdiperstat significantly decreased MPO activity in human blood, a biomarker of the drug engaging its target. Verdiperstat has been generally safe and well tolerated in approximately 250 patients.

"The orphan drug designations from both the FDA and European Medicines Agency underscore the high unmet medical need in MSA and support Biohaven's efforts to advance verdiperstat as a potential first-in-class, oral, MPO inhibitor treatment for people with this severe neurological disease," Marianne Frost, Head of Regulatory Affairs at Biohaven, said.

Potential benefits of orphan drug designation include tax credits, waiver of marketing application user fees and 7 years of marketing exclusivity if regulatory approval is ultimately received.

MSA is a rare, rapidly progressive, and fatal neurodegenerative disease that leads to death after an average of 6 to 10 years from disease onset. MSA causes Parkinson's disease-like movement problems (e.g., slow movement, rigid muscles, tremor, and poor balance), cerebellar ataxia, as well as problems with involuntary (autonomic) functions, including blood pressure control, bladder function, and digestion. There is no cure for MSA. Only symptomatic and palliative therapies are available.

Verdiperstat is a potential first-in-class, oral, brain-penetrant, irreversible inhibitor of MPO that Biohaven is developing as a treatment for MSA. Verdiperstat also has the potential to be developed in a number of other disease indications associated with oxidative stress, inflammation, and neurodegeneration. Biohaven licensed verdiperstat from AstraZeneca in September 2018, where it was known as AZD3241.  Verdiperstat has also been known as BHV-3241 at Biohaven.

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