Neurocrine Biosciences announced the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for opicapone, a novel, once-daily, oral, selective catechol-O-methyltransferase (COMT) inhibitor as an adjunctive treatment to levodopa/carbidopa in patients with Parkinson's disease experiencing OFF episodes. Parkinson's disease is a chronic, progressive and debilitating neurodegenerative disorder that affects approximately one million people in the U.S. The FDA has set a standard 12-month review process with a Prescription Drug User Fee Act (PDUFA) target action date of April 26, 2020.

"People living with Parkinson's disease often struggle to control their motor fluctuations due to the progressive neurodegenerative effects of the disorder. With opicapone, we aim to prolong the benefits of levodopa by providing a new treatment option to patients with Parkinson's disease in the U.S.," said Eiry W. Roberts, M.D., Chief Medical Officer, Neurocrine Biosciences. "It is our goal to help patients maintain good ON time – the period when their motor symptoms are better controlled – and reduce OFF time – the period when the effects of levodopa have worn off. We look forward to working with the FDA to bring this new treatment option to patients coping with this debilitating disorder."

The NDA for opicapone is supported by data from 38 clinical studies, including two Phase III studies (BIPARK-1 and BIPARK-2), with more than 1,000 Parkinson's disease patients treated with opicapone. BIPARK-1 was a Phase III, randomized, double-blind placebo- and active-controlled study of opicapone as an adjunct to levodopa therapy in which approximately 600 patients with Parkinson's disease and motor fluctuations received once-daily opicapone (5 mg, 25 mg, or 50 mg), placebo, or 200 mg doses of the COMT inhibitor entacapone for 14 to 15 weeks. BIPARK-2 was a Phase III, randomized, double-blind placebo-controlled study of opicapone as an adjunct to levodopa therapy in which approximately 400 patients with Parkinson's disease and motor fluctuations received once-daily opicapone (25 mg or 50 mg) or placebo for 14 to 15 weeks. The primary endpoint in both studies was the change from baseline in absolute time in the OFF state, as assessed by patient diaries.

In June 2016, BIAL – Portela & CA, S.A. (BIAL) received approval from the European Commission for ONGENTYS® (opicapone) as an adjunct therapy to preparations of levodopa/DOPA decarboxylase inhibitors (DDCIs) 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. As a result of the FDA's acceptance of the NDA submission, Neurocrine Biosciences will pay a $10 million milestone payment to BIAL.

Parkinson's disease is a chronic, progressive and debilitating neurodegenerative disorder that affects approximately one million people in the U.S and six million people worldwide. Parkinson's disease is characterized by a loss of dopamine and its function. Dopamine is a chemical "messenger" that is produced in the brain and is involved in the control of movement. As the disease progresses, dopamine production steadily decreases resulting in slowed movement (bradykinesia), tremor, rigidity, impaired posture and balance, and speech and writing difficulty.

There is no present cure for Parkinson's disease and management consists of controlling the motor symptoms primarily through administration of dopaminergic therapies, including levodopa. While levodopa improves patients' motor symptoms, as the disease progresses, the beneficial effects of levodopa begin to wear off more quickly, causing symptoms to worsen as patients experience motor fluctuations throughout the day.

Opicapone, an investigational treatment for Parkinson's disease in the U.S., is a novel, once-daily, selective catechol-O-methyltransferase (COMT) inhibitor. Opicapone works by prolonging the clinical effect of levodopa through decreasing its conversion rate into 3-O-methyldopa to allow for greater availability in the brain. Discovered in the BIAL laboratories, it is designed to provide patients and physicians with a once-daily option as an adjunct treatment to levodopa/carbidopa for Parkinson's disease.

In June 2016, the European Commission approved ONGENTYS® (opicapone) as an adjunct therapy to preparations of levodopa/DOPA decarboxylase inhibitors (DDCIs) in adult patients with Parkinson's disease and end-of-dose motor fluctuations who cannot be stabilized on those combinations. In February 2017, Neurocrine Biosciences entered into an exclusive licensing agreement with BIAL for the development and commercialization of opicapone in the U.S. and Canada. Opicapone is an investigational drug not approved for use in the U.S. or Canada.

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