Biohaven secures FDA orphan drug designation for MSA drug verdiperstat

Connecticut-based biopharma company Biohaven Pharmaceutical has secured orphan drug designation from the US FDA for its myeloperoxidase (MPO) inhibitor verdiperstat for the treatment of multiple system atrophy (MSA).

According to Biohaven Pharmaceutical, verdiperstat (formerly known as BHV-3241) is an oral, brain-penetrant, irreversible inhibitor of the MPO enzyme. MPO is a key driver of pathological oxidative stress and inflammation in the brain.

Verdiperstat also has orphan drug designation for the multiple system atrophy treatment from the European Commission following recommendation from the European Medicines Agency’s Committee for Orphan Medicinal Products.

Irfan Qureshi – Executive Director of Biohaven Pharmaceutical and development lead for verdiperstat, said: “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.

“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.”

The investigational MSA drug has undergone phase 1 clinical trials at doses up to 900mg two times a day. Initial results from a phase 2a trial clinical in patients with multiple system atrophy demonstrated numerical improvements on the change from baseline Unified MSA Rating Scale.

Upon 12 weeks of treatment, placebo-treated patients worsened by 4.6 points on the Unified MSA Rating Scale, while patients treated with verdiperstat worsened by 3.7 points at the 300mg twice-daily dose and by 2.6 points at the 600mg two times a day dose.

Corresponding benefits were also seen in other outcome measures including the Composite Autonomic Symptom Score and MSA-Quality of Life scale, said Biohaven Pharmaceutical.

Overall, the clinical findings were on par with neuroprotective effects of verdiperstat as seen in animal models. In the phase 2a clinical trial, the investigational MSA drug lowered MPO activity significantly in human blood – which is a biomarker of the drug engaging its target. The investigational multiple system atrophy drug has been generally safe and well tolerated in nearly 250 patients, said Biohaven Pharmaceutical.

Marianne Frost – Head of Regulatory Affairs at Biohaven Pharmaceutical, said: “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.”

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