FDA Grants Dyskinesia Treatment Orphan Drug Designation

The FDA Office of Orphan Drug Products (OOPD) has designated a 7-year orphan drug exclusivity for amantadine (GOCOVRI) extended-release capsules.

Gocovri, the only FDA-approved therapy for dyskinesia in Parkinson’s disease (PD) patients receiving levodopa-based therapy — with or without concomitant dopaminergic medications — was originally approved on August 24. Its exclusive orphan drug status will run through August 24, 2024.

The Adamas Pharmaceuticals drug is a once-daily bedtime 274 mg amantadine, functioning by delivering consistent and high levels of amantadine in the morning and throughout the day.

It serves as a resolve to the residual strength of levodopa therapy for PD patients, who are at significant risk of suffering from dyskinesia while on the common therapy. About 1 million US residents are inflicted with PD, and 15 to 20% suffer from dyskinesia, according to Adamas.

A study published 2 months before GOCOVRI’s FDA approval reported that oral amantadine was capable of reducing levodopa-induced dyskinesia (LID) and the total time PD medication fails to control motor symptoms in patients.

Aparna Wagle Shukla, MD, an editorial author reviewing the study, wrote that amantadine was the sole oral medication “to show a definite robust antidyskinetic effect” on studied patients.

Gocovri was also tested to treat sleep disorders in PD patients, who generally suffer from such symptoms to their overall detriment.

Aleksander Videnovic, MD, associate professor at Massachusetts General Hospital, told MD Magazine that PD patients who are able to sleep well experience less symptoms of their disease the following day.

“Perhaps, even responsiveness to the medications they take for the symptoms is better after a good night of sleep,” Videnovic said.

Adamas’ breakthrough therapy will have a long monopoly on treating a consistently difficult symptom in PD patients. Involuntary movements induced by dyskinesia are a burden on patients’ quality of life, Chris O’Brien, MD, chief medical officer at Neurocrine Sciences, told MD Magazine.

"They affect the mouth, the tongue, the jaw, the limbs, the trunk,” O’Brien said. “They add to the stigma of mental illness. This is really hard for patients because often the result is social isolation."

A press release regarding the designation was made available.

This article was originally published by MD Magazine.