~ Results Demonstrate Long-term Treatment with KEVEYIS is Efficacious and Provides Durable Reduction in Attack Frequency and Severity in Patients with PPP ~
DUBLIN, Ireland and TREVOSE, Pa., May 09, 2019 (GLOBE NEWSWIRE) — Strongbridge Biopharma plc, (Nasdaq: SBBP), a global commercial-stage biopharmaceutical company focused on the development and commercialization of therapies for rare diseases with significant unmet needs, today announced that long-term efficacy results for KEVEYIS® (dichlorphenamide), the first and only U.S. Food and Drug Administration (FDA) approved treatment for hyperkalemic, hypokalemic, and related variants of primary periodic paralysis (PPP), were presented at the 71stAmerican Academy of Neurology (AAN) Annual Meeting held from May 4 – 10, 2019 in Philadelphia, Pennsylvania.
“The data presented at AAN today show that long-term treatment with KEVEYIS in adults is effective in significantly improving both attack frequency and severity in adult patients with primary periodic paralysis, or PPP,” said Fredric Cohen, chief medical officer of Strongbridge Biopharma. “These findings are important because periodic paralysis is a life-long disorder, and thus demands durable treatment. In the ‘HYP/HOP’ study, daily treatment with KEVEYIS durably reduced the average number and severity of attacks of weakness and paralysis that were tracked via patient diaries.”
In a poster presentation entitled, “Long-Term Efficacy of Dichlorphenamide for the Treatment of Primary Periodic Paralysis,” Nicolas E. Johnson, MD, MSCI, FAAN, lead investigator and associate professor, division chief of neuromuscular and vice chair of research in the department of neurology at Virginia Commonwealth University, presented new findings from a post-hoc analysis evaluating the long-term (up to 61 weeks) efficacy of KEVEYIS in the HYP/HOP study of adult patients with PPP. The results show that long-term treatment with KEVEYIS is efficacious and provides durable reduction in attack frequency and severity in patients with PPP.
The HYP/HOP study was a randomized, double-blind, placebo-controlled trial of 63 patients randomized to receive KEVEYIS (n=36) or placebo (n=27) for 9 weeks during a double-blind phase. Patients then entered a 52-week open-label phase: patients in the KEVEYIS group continued to receive the the same dose they were taking at the end of the 9-week double-blind phase, and patients in the placebo group switched to KEVEYIS 50 mg twice daily.i
• Poster P5.4-008 – Long-Term Efficacy of Dichlorphenamide for the Treatment of Primary Periodic Paralysis (PPP); Nicolas E. Johnson; Poster Session P5, Genetic Muscle Disorders II; May 9, 2019 – 11:30 a.m.-6:30 p.m. ET
The poster can be accessed here.
About Strongbridge Biopharma
Strongbridge Biopharma is a global commercial-stage biopharmaceutical company focused on the development and commercialization of therapies for rare diseases with significant unmet needs. Strongbridge’s rare endocrine franchise includes RECORLEV™ (levoketoconazole), a cortisol synthesis inhibitor currently being studied in Phase 3 clinical studies for the treatment of endogenous Cushing’s syndrome, and veldoreotide extended release, a pre-clinical next-generation somatostatin analog being investigated for the treatment of acromegaly and potential additional applications in other conditions amenable to somatostatin receptor activation. Both RECORLEV and veldoreotide have received orphan drug designation from the FDA and the European Medicines Agency. The Company’s rare neuromuscular franchise includes KEVEYIS® (dichlorphenamide), the first and only FDA-approved treatment for hyperkalemic, hypokalemic, and related variants of primary periodic paralysis. KEVEYIS has orphan drug exclusivity in the United States.
KEVEYIS® (dichlorphenamide) is indicated for the treatment of primary hyperkalemic periodic paralysis, primary hypokalemic periodic paralysis, and related variants. In clinical studies, the most common side effects of KEVEYIS were a numbness or tingling, difficulty thinking and paying attention, changes in taste, and confusion. These are not all of the possible side effects that you may experience with KEVEYIS. Talk to your doctor if you have any symptoms that bother you or do not go away. You are encouraged to report side effects to Strongbridge Biopharma at 1-855-324-8912, or to the FDA at 1-800-FDA-1088 or visit www.fda.gov/medwatch. For additional KEVEYIS important safety information and the full prescribing information visit www.keveyis.com.
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i Sansone VA, et al. Neurology. 2016;86(15):1408-1416.
Source: Strongbridge Biopharma