Strongbridge Biopharma plc to Present KEVEYIS® (dichlorphenamide) Primary Periodic Paralysis Data at the 2018 American Academy of Neurology Annual Meeting
~ Post-Hoc Analysis of Phase 3 Placebo-Controlled Study of KEVEYIS Showed Adolescents had Similar Improvements in Short-Term Attack Rates as Adults ~
~ Pooled Analysis of Two Phase 3 Studies Further Demonstrate the Clinical Benefit and Use of KEVEYIS in Patients with Hypokalemic and Hyperkalemic Primary Periodic Paralysis ~
“These new analyses help to validate the overall clinical profile of KEVEYIS in a variety of patient populations and disease variants,” said
In a poster presentation entitled, “Efficacy and Safety of Dichlorphenamide in Adolescent Patients with Primary Periodic Paralysis,”
In an oral poster presentation entitled, “Efficacy of Dichlorphenamide in Primary Periodic Paralysis: Pooled-Data Analysis of Two Phase 3 Clinical Trials,”
• Poster #955 – Efficacy and safety of dichlorphenamide in adolescent patients with primary periodic paralysis;
Detailed findings include:
- Dichlorphenamide treatment resulted in median changes from baseline in weekly attack rate of −0.96 [CI: ‒1.63, ‒0.88] in adolescents vs ‒0.83 [CI: ‒2.75, ‒0.26] in adults.
- Median changes from baseline in severity-weighted weekly attack rate were ‒2.25 [CI: −3.50, −1.38] in adolescents vs ‒1.17 [CI: −3.38, ‒0.63] in adults.
- Three of six adolescents with safety data reported ≥1 solicited side effect with dichlorphenamide treatment; no side effects were dose-limiting or resulted in study withdrawal.
- The frequency of each side effect reported by the adolescents was similar in adults.
• Oral Poster #958 – Efficacy of Dichlorphenamide in Primary Periodic Paralysis: Pooled-Data Analysis of Two Phase 3 Clinical Trials;
Detailed findings include:
- Median weekly attack rate improved with dichlorphenamide vs placebo: Median change from baseline was −0.75 vs +0.13, P=0.0004; treatment-effect size −1.22 [CI: −3.15, −0.38]. Median relative change from baseline was −51.67% for dichlorphenamide and 0.00% for placebo, P<0.0001; treatment-effect size −65.31% [CI: −93.17%, −34.40%].
- Median severity-weighted attack rate also improved with dichlorphenamide: Median change from baseline was −0.67 vs +0.55, P=0.0049; treatment-effect size −1.63 [CI: −4.47, −0.09]. Median relative change from baseline was −45.18% vs +16.05%, P=0.0002; treatment-effect size −60.85% [CI: −88.46%, −23.53%].
Primary periodic paralysis is a rare inherited disorder characterized by recurrent, progressive, and debilitating episodes of muscle weakness and temporary paralysis. Primary periodic paralysis is very rare, affecting about 5,000 to 6,000 individuals in the United States.
About Strongbridge Biopharma plc
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 commercial portfolio within its rare neuromuscular and rare endocrine franchises includes KEVEYIS®(dichlorphenamide), the first and only
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
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Source: Strongbridge Biopharma plc