Neurocrine Gets FDA Nod for Once-Daily “Add-On” Parkinson’s Pill

Xconomy San Diego — 

Many patients with Parkinson’s disease take a combination medication called levodopa/carbidopa to control their symptoms, but over time, experience “off” episodes in which the tremor, slowed movement, and difficulty walking that characterize the disorder occur.

Now the FDA has approved a Neurocrine Biosciences (NASDAQ: NBIX) drug intended to be taken along with levodopa/carbidopa to extend the therapy’s “on” time for patients.

About 50,000 people in the United States are diagnosed with Parkinson’s disease yearly. After Alzheimer’s disease, the condition is the second most common neurodegenerative disorder in the country.

The Neurocrine drug, opicapone (Ongentys), is part of a class of medications known as catechol-O-methyltransferase (COMT) inhibitors that are used to prolong the effects of levodopa by blocking the COMT enzyme to prevent it from breaking down that drug. The San Diego-based biotech designed the pill to increase “on” time for patients between levodopa doses without causing dyskinesia, or involuntary movements other than those typical of Parkinson’s—a side effect of long-term use of the longtime therapy for the disease.

The once-daily pill was evaluated in two Phase 3 clinical studies that enrolled more than 1,000 patients with Parkinson’s disease. In both trials a 50 mg dose reduced “off” time compared to a placebo, according to the company. The most common side effects reported by those taking opicapone were dyskinesia, constipation, increase in blood creatine kinase, low blood pressure causing syncope, or fainting, and weight loss. The drug’s label recommends a 25 mg dose for patients with “moderate” hepatic impairment, or liver damage; those with severe impairment shouldn’t take it, the label advises.

Opicapone is the only COMT inhibitor approved for once-daily use. In 2017 Neurocrine licensed the rights to develop and commercialize the drug in the US and Canada from Bial, a Portuguese pharma company. At the time, Bial, which retains rights elsewhere, had received approval from European regulators to market the drug there to some Parkinson’s patients.

Neurocrine relies on Bial for its supply of opicapone, and earlier this month announced that it would delay its US launch until “later this year” given its concerns over Bial’s ability to adequately supply it with commercial inventory amid the ongoing pandemic.

The company’s stock price reached $101.67 per share Monday, up about 4 percent compared to Friday’s close. SVB Leerink analyst Marc Goodman, in a research note, attributed the “somewhat muted” response by investors to an expectation of approval, plus the delayed launch. Goodman estimated peak sales of opicapone to range from $200 million to $250 million.

It’s the second FDA nod for Neurocrine, whose first drug, valbenazine (Ingrezza), was OK’d in 2017 for patients with tardive dyskinesia, involuntary movements associated with the long-term use of drugs designed to treat mental health conditions. Product sales last year totaled nearly $753 million.

Another drug Neurocrine discovered, elagolix (Orilissa), was approved in April 2018 as a treatment for moderate to severe endometriosis pain. The biotech licensed the compound in 2010 to AbbVie (NYSE: ABBV), which brought it to commercialization.