http://www.raredr.com/news/primary-data-dichlorphenamide
Strongbridge to Present Primary Data of Periodic Paralysis Drug at AAN Meeting

Mathew Shanley

Strongbridge Biopharma plc announced this morning that it will be presenting new clinical analyses for dichlorphenamide (Keyevis) at the 70th American Academy of Neurology (AAN) Meeting in April.

Dichlorphenamide is the first and only treatment intended for hyperkalemic, hypokalemic, and related variants of primary periodic paralysis to have been approved by the U.S. Food and Drug Administration (FDA). It was granted approval for the group of rare genetic disorders in August 2015.

Dichlorphenamide is a carbonic anhydrase inhibitor that assists in the reducing of of blood-potassium levels, thereby normalizing them. In an oral presentation titled “Efficacy of Dichlorphenamide in Primary Periodic Paralysis: Pooled-Data Analysis of Two Phase 3 Clinical Trials,” Perry B. Shieh, M.D., Ph.D., lead investigator and associate professor of the neuromuscular program at the University of California at Los Angeles, David Geffen School of Medicine will offer a pooled analysis of efficacy data collected from two different Phase 3 placebo-controlled trials, both of which assessed dichlorphenamide in 138 patients with primary periodic paralysis.

The trials measured changes in weekly attack rates from baseline. The results are consistent with previous clinical evidence for the drug, supporting its efficacy across disease variants.

Additionally, in a poster presentation titled “Efficacy and Safety of Dichlorphenamide in Adolescent Patients with Primary Periodic Paralysis,” Emma Ciafaloni, M.D., lead investigator and professor of neurology and pediatrics at the University of Rochester Medical Center, will unveil new findings from a post-hoc analysis of a Phase 3, placebo-controlled, cross-over trial comparing the effects of dichlorphenamide in adolescent and adult patients with primary periodic paralysis.

Results of the study exhibit similar improvements in short-term attack rates and side effects in both patient populations.

 “These new analyses help to validate the overall clinical profile of Keyevis in a variety of patient populations and disease variants,” said Fredric Cohen, M.D., chief medical officer of Strongbridge Biopharma in a press release. “Primary periodic paralysis is a rare genetic neuromuscular condition with significant unmet need and we are encouraged by the consistency of positive data for Keyevis and the potential value it can bring to the underserved periodic paralysis community.”

Forms of periodic paralyses are separated by criteria, including underlying genetic mutations and changes in blood-potassium during attack. While hypokalemic and hyperkalemic are the two most common types of periodic paralyses, the National Institute of Health (NIH) estimates that the disorder affects less than 5,000 people in the United States.

The 70th AAN Annual Meeting will be held from April 21-27 in Los Angeles, California.

For more on data pertinent to the rare disease community being presented at upcoming meetings, follow Rare Disease Report on Facebook and Twitter.
 
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