Investigational Inhaled L-Dopa for Treatment of OFF Periods in Parkinson’s Disease

CVT-301 is a self-administered, inhaled formulation of levodopa (L-dopa). It is being studied for the treatment of symptoms of OFF periods in people with Parkinson’s disease taking an oral carbidopa / levodopa regimen. OFF periods are characterized by the re-emergence of Parkinson’s symptoms. We plan to file a New Drug Application with the U.S. Food and Drug Administration in the second quarter of 2017.

In February 2017, Acorda announced results from a Phase 3 clinical trial of 339 participants who received CVT-301 or placebo to assess the safety and efficacy of CVT-301 to improve motor function in people with Parkinson’s disease experiencing OFF periods. . In addition, two long-term safety studies evaluated use of CVT-301 for up to 12 months. You can find details on these studies here.

Approximately one million people in the U.S. and 1.2 million Europeans are diagnosed with Parkinson’s disease (PD); OFF periods are experienced by approximately 350,000 in the U.S. and 420,000 in Europe.

Parkinson’s is a progressive neurodegenerative disorder resulting from the gradual loss of certain neurons responsible for producing dopamine. It causes a range of symptoms including impaired movement, muscle stiffness and tremors. As PD progresses, people with Parkinson’s experience OFF periods, which are characterized by the re-emergence of PD symptoms. This re-emergence can occur even when an individual’s treatment regimen has been optimized. OFF periods can be very disruptive to the lives of people with Parkinson’s, their families and caregivers. OFF periods can increase in frequency and severity during the course of the disease.

CVT-301 utilizes Acorda’s investigational ARCUS® platform for inhaled therapeutics. CVT-301 is designed to deliver a precise dose of a dry powder formulation of L-dopa to the lung. Oral medication can be associated with variable onset of action, as the medicine is absorbed through the gastrointestinal (digestive) tract before reaching the brain. Inhaled treatments enter the body through the lungs and then reach the brain, bypassing the digestive system.

Early CVT-301 clinical studies were funded in part by grants from The Michael J. Fox Foundation for Parkinson’s Research.