New Parkinson's drug, 2 others approved

FDA approved safinamide (Xadago, Newron Pharmaceuticals) tablets as an add-on treatment for patients with Parkinson’s disease.

Xadago is for patients who are currently taking levodopa/carbidopa and experiencing “off” episodes, or times when a patient’s medications are not working well, which causes an increase in Parkinson’s symptoms, such as tremor and difficulty walking.

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

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An estimated 50,000 Americans are diagnosed with Parkinson’s disease annually according to the National Institutes of Health, and about 1 million Americans have the condition.

The efficacy of Xadago in treating Parkinson’s disease was shown in a clinical trial of 645 participants who were also taking levodopa and were experiencing “off” time. Those receiving Xadago experienced more beneficial “on” time, when Parkinson’s symptoms are reduced, without troublesome uncontrolled involuntary movement (dyskinesia), compared to those receiving a placebo.

The increase in “on” time was accompanied by a reduction in “off” time and better scores on a measure of motor function assessed during “on” time than before treatment.

In another clinical trial of 549 participants, the participants adding Xadago to their levodopa treatment had more “on” time without troublesome uncontrolled involuntary movement compared to those taking a placebo, and also had better scores on a measure of motor function assessed during “on” time than before treatment.

FDA also recently approved pembrolizumab (Keytruda, Merck) for adult and pediatric patients with refractory classical Hodgkin lymphoma (cHL), or those who have relapsed after 3 or more prior lines of therapy.

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Under FDA’s accelerated approval regulations, this indication is approved based on tumor response rate and durability of response.

The approval is based on data in 210 patients from the KEYNOTE-087 trial, which demonstrated an overall response rate (ORR) with Keytruda (200 mg every 3 weeks) of 69%, along with a complete remission rate (CRR) of 22% and a partial remission rate (PRR) of 47%.

The median follow-up time was 9.4 months. Among the 145 responding patients, the median duration of response was 11.1 months.

“Today’s approval-the first for Keytruda in a hematologic malignancy-reinforces the hope that immunotherapy will prove useful in a wide variety of cancers,” said Roger M. Perlmutter, MD, PhD, president of Merck Research Laboratories, in a statement from the company.

Meanwhile, FDA also granted breakthrough therapy designation status to rituximab (Rituxan, Genentech) for pemphigus vulgaris, a rare, serious and life-threatening condition characterized by progressive painful blistering of the skin and mucous membranes.

Genentech is currently enrolling a phase 3 study in pemphigus vulgaris (PEMPHIX, NCT02383589), a disease for which there are limited treatment options.

“People with pemphigus vulgaris need more options and we look forward to working with the FDA to make Rituxan available to patients with this potentially deadly disease,” said Sandra Horning, MD, chief medical officer and head of Global Product Development, in a Genentech statement. “We are committed to developing therapies that target B cells in areas of unmet need across a range of immune and malignant diseases, including new antibody constructs that enhance efficacy, safety or both.”

Breakthrough therapy designation status was granted based on data from a Roche-supported randomized trial conducted in France, which evaluated Rituxan plus oral corticosteroid (CS) treatment compared to CS as a first-line treatment in patients with moderate to severe pemphigus.

Results of the study, published in The Lancet, show that Rituxan may provide substantial improvement in pemphigus vulgaris remission rates and successful tapering and/or cessation of CS therapy.

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