Inhaled L-Dopa for Treatment of Parkinson’s Disease Off Episodes

CVT-301 is a potentially transformative therapy in development for people with Parkinson’s disease (PD) to treat off episodes. We began enrolling a Phase 3 study in December 2014. If successful, our goal is to file a New Drug Application with the U.S. Food and Drug Administration by the end of 2016.

There are approximately one million people in the U.S. and 1.2 million Europeans diagnosed with PD. PD is a progressive neurodegenerative disorder resulting from the gradual loss of certain neurons responsible for producing dopamine. This results in the emergence of motor symptoms such as impaired ability to move, tremors and muscle stiffness.

CVT-301 is being developed as a self-administered, inhaled levodopa (L-dopa) therapy for the episodic treatment of off episodes in Parkinson’s disease, and is being studied to provide delivery of a precise dose of L-dopa through the lungs to return people with Parkinson’s to an on state; oral L-dopa is viewed as the gold standard in the treatment of Parkinson’s disease. Off episodes are defined by periods of time when background L-dopa therapy does not adequately control Parkinson’s symptoms. Approximately 50% of people using L-dopa experience off episodes. These episodes can be very disruptive to the lives of people with Parkinson’s disease, their families and caregivers. Off episodes increase in frequency and severity during the course of the disease. Approximately 350,000 people with Parkinson’s disease may be appropriate for treatment with CVT-301.

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