Verdiperstat, a Potential Treatment for Multiple System Atrophy, Granted Orphan Drug Status

The Food and Drug Administration has granted orphan drug status to verdiperstat (BHV-3241; Biohaven Pharmaceutical, New Haven, CT). Verdiperstat is a novel brain-penetrant myeloperoxidase (MPO) inhibitor being studied as a potential first-in-class treatment for multiple system atrophy (MSA). Verdiperstat has also received orphan drug designation for the treatment of MSA from the European Commission.

In a phase 2a clinical trial, patients with MSA treated with 300 mg or 600 mg verdiperstat twice daily for 12 weeks had less worsening (300 mg: 3.7 ± 1.2 points, n = 17; 600 mg: 2.6  ± 1.4, n = 18)) on the Unified MSA Rating scale than those given placebo (4.6 ± 1.1 points; n = 17). 

Benefits of treatment were also seen on the Composite Autonomic Symptom Score and MSA-Quality of Life scale. In phase 1 and 2 trials, verdiperstat has been generally safe and well tolerated in approximately 250 patients. A phase 3 trial was approved by the FDA in late January and is expected to begin later this year.

It is thought that verdiperstat has a neuroprotective effect by reducing reduce oxidative stress and brain inflammation through the inhibition of MPO. In the phase 2a trial, persons treated with verdiperstat had significantly decreased plasma activity of MPO. 

Irfan Qureshi, MD, Biohaven Executive Director and development lead for verdiperstat, commented, "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."

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