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

CVT-301 is a self-administered, inhalable 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 refer to 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, pending analysis of clinical trial data.

A Phase 3 clinical trial of 339 participants who received CVT-301 or placebo as an adjunct to their oral carbidopa/levodopa regimen showed a statistically significant improvement in motor function in people with Parkinson’s disease experiencing OFF periods. This was based on the change at Week 12 in Unified Parkinson’s Disease Rating Scale-Part 3 (UPDRS III) score, a validated scale which measures Parkinson’s motor impairment. Additional trials are ongoing. For more information, click 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 slow and 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.