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The new approval is based on the result of 38 clinical trials involving more than 1000 patients.
Robert A. Hauser, MD
The US Food and Drug Administration (FDA) has approved opicapone (Ongentys), a once-daily, oral, selective catechol-O-methyltransferase (COMT) inhibitor for the adjunctive treatment of OFF episodes in patients with Parkinson disease.
The approval, awarded to Neurocrine Biosciences, is supported by 38 clinical studies in more than 1000 patients, follows marketing approvals previously granted in Germany, the UK, Spain, Portugal, and Italy.
Opicapone, which will be available in 25 mg and 50 mg capsules for add-on treatment, works by inhibiting the COMT enzyme, which breaks down levodopa in the blood, in turn prolonging the clinical effects of the gold-standard Parkinson treatment.
The clinical trial program included 2 phase 3 trials, BIPARK-1 and BIPARK-2, in which the primary end point was change from baseline in absolute time in the OFF state as assessed by patient diaries.
The double-blind, placebo- and active-controlled BIPARK-1 assessed safety and efficacy of opicapone in approximately 600 patients with Parkinson disease compared with placebo and the active comparator drug entacapone.
Participants were randomly assigned to 1 of 3 doses of the study drug (5 mg, 25 mg, or 50 mg), placebo, or the active comparator for 14 or 15 weeks.
Results of that trial revealed a placebo-adjusted OFF time reduction of 60.8 minutes in the 50-mg group, a 51% greater reduction than what was observed in the active compartor group (40.3 minutes).
“The FDA approval of Ongentys represents an important new treatment option for people with Parkinson disease,” Robert A. Hauser, MD, professor of neurology and director, University of South Florida Parkinson’s Disease and Movement Disorders Center, said in a statement. “As Parkinson disease progresses, first-line treatments such as levodopa begin to lose effectiveness and the beneficial effects of levodopa begin to wear off more quickly, causing more frequent and often debilitating motor fluctuations in patients. Clinical studies have shown that adding once-daily Ongentys to levodopa therapy significantly reduced OFF time, leading to better and more consistent motor symptom control.”
In the open-label extension study, patients who switched from placebo or entacapone experienced a 65 minute and 39 minute decrease in mean OFF time. Notably, ON time without dyskinesia increased by 43 minutes and 46 minutes, respectively.
In BIPARK-2, investigators assessed adjunctive opicapone versus placebo in 427 patients with Parkinson disease and motor fluctuations. That study demonstrated a significant 54.3-minute reduction in OFF time versus placebo in the 50-mg group, with daily off time reduced by 126.3 minutes over 1-year follow-up.
Safety data from both trials indicated the most frequently reported adverse events were dyskinesia, constipation, an increase in blood creatine kinase, hypotension/syncope, and a decrease in weight.
“Due to the progressive nature of Parkinson disease, those living with the condition often struggle to control their motor fluctuations, affecting a wide range of functions, including speech, balance and movement, which adversely impact many aspects of life,” said John L. Lehr, president and chief executive officer of the Parkinson’s Foundation, in a statement. “The Parkinson disease community is encouraged by the FDA approval of a new add-on treatment option to help patients further control symptoms, enabling them to better cope with this progressive disease.”
Neurocrine indicated that the treatment will be made available to patients in the US later this year.