FDA advisory panel votes against approval of DMD drugAccording to the National Institutes of Health, around 50,000 individuals in the country are diagnosed with Parkinson’s disease every year; approximately one million people are currently suffering from the neurological condition that at present has no cure.

Last week the U.S. Food and Drug Administration (FDA) announced its approval of a drug for the treatment of Parkinson’s disease in a press release. Xadago (safinamide) is intended for patients that meet two conditions: they must already be taking levodopa/carbidopa, and must be experiencing what is known as “off” episodes (a period when medications are working to ameliorate symptoms like gait issues or tremors). The approval for the drug has been granted to Newron Pharmaceuticals.

“A relentless disease without a cure”

Although Parkinson’s is usually seen in patients over the age of 60, it does less frequently strike individuals much younger. Dopamine is a chemical in the brain that allows for the smooth transmission of messages from one area of the brain to another, resulting in normal bodily movement. But in Parkinson’s, brain cells responsible for the production of dopamine undergo impairment or die off. Often the disease begins gradually, although some patients suffer a rapid onset.

Eric Bastings, M.D., is deputy director of the Division of Neurology Products at the FDA’s Center for Drug Evaluation and Research. “Parkinson’s is a relentless disease without a cure,” he said. “We are committed to helping make additional treatments for Parkinson’s disease available to patients.”

Clinical trials included more than 1,000 patients

Prior to Xadago’s FDA approval, the drug underwent a clinical trial involving more than 600 patients, all of them on the medication levodopa and all of them in an “off” episode. As juxtaposed with those trial participants who received placebo tablets during the study, the patients given Xadago tablets reported more “on” time (a period where symptoms are alleviated and where the patients are not suffering from dyskinesia—involuntary movement). Additionally, those patients received scores on assessments of motor function that were higher than the same assessment prior to taking Xadago.

Another clinical trial that occurred before the FDA gave the medication the green light involved over 500 participants that were also being treated with levodopa and yet experiencing “off” periods. That trial commenced with similar results: more “on” time than “off” for those patients receiving Xadago (versus placebo recipients), as well as improved motor function after taking the drug than before.

Contraindications and side effects

As with many prescription drugs, there is cautionary advice that the FDA wants patients to be aware of. Individuals with liver problems should not take Xadago, according to FDA the press release. Also, people taking monoamine oxidase inhibitors (MAOI) should not take Xadago (the two drugs could cause a spike in blood pressure). Xadago should also not be taken while the patient is treating a cough with medicines containing dextromethorphan. An extremely dangerous (possibly fatal) reaction known as “serotonin syndrome” can occur in individuals who take the drug while taking opioids, some anti-depressants, cyclobenzaprine, or St. John’s wort.

And, as is also not unusual in the world of prescription medications, the potential for adverse reactions exists, such as nausea, insomnia, and uncontrolled, involuntary movements. Less commonly, some patients experienced hallucinations, psychotic behaviors, pathology of the retina, difficulty controlling impulses, fevers and confusion upon withdrawal, elevated blood pressure, and falling asleep during the day.

 

This blog post is provided for educational purposes only and is not offered as, and should not be relied on as, legal advice. Any individual or entity reading this information should consult an attorney for their particular situation. For more information/questions regarding any legal matters, please email info@nelsonhardiman.com or call 310.203.2800.