Connecticut-based Biohaven Pharmaceutical has received a significant regulatory boost, with the US FDA granting orphan drug designation for its investigational drug verdiperstat for treating multiple system atrophy (MSA). This rare and debilitating neurodegenerative disease has long been a target for pharmaceutical companies seeking breakthroughs in treatment, and verdiperstat’s designation underscores its potential as a game-changer in the space.

Verdiperstat, previously known as BHV-3241, is an oral, brain-penetrant MPO (myeloperoxidase) inhibitor that works by targeting the MPO enzyme, a key contributor to oxidative stress and inflammation in the brain. The FDA’s designation comes alongside a similar recognition from the European Commission, following a recommendation from the European Medicines Agency’s Committee for Orphan Medicinal Products, further reinforcing verdiperstat’s potential in the global fight against MSA.

Verdiperstat: A promising new option for MSA patients

Irfan Qureshi, Executive Director at Biohaven and lead for the drug’s development, expressed enthusiasm about the FDA’s decision, noting the company’s commitment to advancing therapies for high-unmet medical needs. He highlighted that verdiperstat could be the first effective treatment for MSA, a disease currently with no proven therapies. “We remain on track to start our global Phase 3 clinical trial later this year,” Qureshi remarked.

The path to regulatory recognition for verdiperstat has been marked by encouraging clinical trial data. In Phase 1 trials, the drug demonstrated safety and tolerability at doses of up to 900mg twice daily. Phase 2a trials, which focused on MSA patients, revealed positive outcomes, with patients receiving verdiperstat showing a smaller worsening in their Unified MSA Rating Scale scores compared to placebo-treated patients. Specifically, those on the 300mg twice-daily dose worsened by 3.7 points, while those on the 600mg dose worsened by just 2.6 points.

These numerical improvements suggest verdiperstat’s potential as a neuroprotective agent for MSA patients. Biohaven Pharmaceutical also reported benefits in other measures, including the Composite Autonomic Symptom Score and MSA-Quality of Life scale, supporting the drug’s broader therapeutic effects. Additionally, the drug’s ability to reduce MPO activity—a biomarker of its engagement with the target—further bolsters its case as a promising treatment.

Verdiperstat: Pioneering the path for MSA treatment

The drug’s overall safety and tolerability were confirmed in a trial involving nearly 250 patients, reinforcing its potential for long-term use. Marianne Frost, Head of Regulatory Affairs at Biohaven, pointed out that the orphan drug designations from both the FDA and the European Medicines Agency emphasize the urgent need for treatment options in MSA. She added that these recognitions support Biohaven’s continued efforts to develop verdiperstat as a first-in-class, oral, MPO inhibitor for this devastating disease.

Biohaven is on track to launch a Phase 3 global trial later this year, marking the next critical step in the clinical development of verdiperstat as a potential breakthrough for MSA patients. With the support of the orphan drug designations and promising early trial results, verdiperstat could soon offer hope to those living with MSA, a disease that currently has no effective treatment.

  Biohaven Pharmaceutical, clinical trial, European Medicines Agency, MPO inhibitor, MSA, Multiple System Atrophy, neurodegenerative disease, Orphan Drug Designation, US FDA, verdiperstat