On Apr. 27, 2020, Neurocrine Biosciences announced the U.S. Food and Drug Administration (FDA)  had approved once-daily oral ONGENTYS (opicapone) 25 mg and 50 mg capsules as an add-on treatment to levodopa/carbidopa in patients with Parkinson’s disease experiencing ‘off’ episodes. ONGENTYS also increases ‘on’ time without troublesome dyskinesia, the time when the motor symptoms of a patient with Parkinsonﾒs disease are better controlled.

Parkinson’s disease is the second most common neurodegenerative disorder in the United States after Alzheimer’s disease. About one million Americans have Parkinson’s disease and each year, an estimated 50,000 people in the United States are newly diagnosed with this chronic, progressive and debilitating neurodegenerative disorder.

ONGENTYS is an oral, selective catechol-O-methyltransferase (COMT) inhibitor that helps block the COMT enzyme which breaks down levodopa, the gold standard therapy for controlling motor symptoms in patients with Parkinson’s disease. ONGENTYS protects levodopa by reducing its breakdown in the blood, making more levodopa available to reach the brain, prolonging its clinical effects and helping patients achieve motor symptom control.

The FDA approval of ONGENTYS is supported by data from 38 clinical studies, including two multinational Phase III clinical studies (BIPARK-1 and BIPARK-2), with more than 1,000 Parkinson’s disease patients treated with ONGENTYS. In the BIPARK-1 trial, approximately 600 patients with Parkinson’s disease and motor fluctuations received one of three doses of ONGENTYS (5 mg, 25 mg or 50 mg), placebo or 200 mg doses of the COMT inhibitor entacapone for 14 or 15 weeks. In the BIPARK-2 trial, approximately 400 patients received one of two doses of ONGENTYS (25 mg or 50 mg) or placebo for 14 or 15 weeks. Both studies included a one-year open-label extension. Data from both trials showed that ONGENTYS 50 mg significantly reduced “off” time from baseline to week 14 or 15 compared to placebo. “On” time without troublesome dyskinesia also increased from baseline to week 14 or 15 compared to placebo.

Pooled safety data from the BIPARK-1 and BIPARK-2 studies indicated that the most common adverse reactions across all patients treated with ONGENTYS (incidence at least 4% and greater than placebo) were dyskinesia, constipation, blood creatine kinase increase, hypotension/syncope, and weight decrease.

In June 2016, BIAL – Portela & CA, S.A. (BIAL) received approval from the European Commission for ONGENTYS as an adjunct therapy to preparations of levodopa/DOPA decarboxylase inhibitors in adult patients with Parkinson’s disease and end-of-dose motor fluctuations who cannot be stabilized on those combinations. BIAL currently markets ONGENTYS in Germany, United Kingdom, Spain, Portugal and Italy. Neurocrine Biosciences in-licensed opicapone from BIAL in 2017 and has exclusive development and commercialization rights in the U.S. and Canada.

Parkinson’s disease is a chronic, progressive and debilitating neurodegenerative disorder that affects approximately one million people in the United States and six million people worldwide. Parkinson’s disease is caused by low dopamine levels produced in the brain. Dopamine helps transmit signals between the areas of the brain that control all purposeful movements, including talking, walking and writing. As Parkinson’s disease progresses, dopamine production steadily decreases, resulting in increased problems with motor symptoms including slowed movement (bradykinesia), tremor, rigidity, impaired posture and balance, and difficulty with speech and writing.