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Strongbridge Biopharma is a global commercial-stage biopharmaceutical company focused on the development and commercialization of therapies for rare diseases with significant unmet needs. Founded in 1996, the Company is publicly traded on the NASDAQ Global Market under the ticker symbol SBBP.

The primary focus of Strongbridge Biopharma is to build and advance a portfolio of vertical, therapeutically-aligned rare disease franchises. At the center of its rare neuromuscular disease franchise is KEVEYIS® (dichlorphenamide), the first and only U.S. Food and Drug Administration (FDA)-approved product to treat hyperkalemic, hypokalemic, and related variants of Primary Periodic Paralysis. The Company’s rare endocrine disease franchise includes RECORLEVTM (levoketoconazole), in Phase 3 clinical development for endogenous Cushing’s syndrome and veldoreotide, for acromegaly. Significant unmet needs remain in the diagnosis and treatment of these rare diseases.

The Company will continue to identify and evaluate the acquisition of products or product candidates that would be complementary to its existing rare neuromuscular and endocrine franchises or that would form the basis for new rare disease franchises.

Strongbridge Biopharma’s Product Portfolio

KEVEYIS is the first and only FDA-approved drug for the treatment of hyperkalemic, hypokalemic, and related variants of Primary Periodic Paralysis, a spectrum of rare and chronic genetic, neuromuscular disorders with autosomal dominant inheritance that cause recurrent, progressive, and debilitating episodes of muscle weakness and temporary paralysis.1 As they age, some patients may experience permanent muscle weakness2. KEVEYIS was approved by the FDA on August 7, 2015 and has orphan designation status through August 7, 2022. Primary periodic paralysis is very rare, affecting approximately 5,000 to 6,000 people in the U.S.3-7 The most common forms of the disease are hyperkalemic and hypokalemic Primary Periodic Paralysis, and time between symptom onset and diagnosis can take more than 20 years.8-9

RECORLEV, a cortisol synthesis inhibitor, is being evaluated in a global Phase 3 clinical program for the treatment of endogenous Cushing’s syndrome. SONICS, the first global Phase 3 study is fully enrolled and supported by LOGICS, a second global Phase 3 study designed to supplement the long-term efficacy and safety data from the SONICS study in a randomized, double-blind, placebo-controlled study. Endogenous Cushing’s syndrome is a rare but serious and potentially lethal endocrine disease caused by chronic elevated cortisol exposure. People with uncontrolled disease are seriously ill and have a 2- to 4-fold higher mortality rate than age- and gender-matched controls, mainly due to metabolic and cardiovascular complications. RECORLEV has been granted orphan drug designation by the FDA and the European Medicines Agency (EMA). The Company is developing RECORLEV, a single enantiomer of ketoconazole, as a new chemical entity, or NCE, under the FDA 505(b)(2) regulatory pathway, and intends to reference the FDA’s prior conclusions of safety for ketoconazole.

Veldoreotide is a novel, investigational somatostatin analogue (SSA) with proven and differentiated pharmacology in pre-clinical models and patients with acromegaly. Veldoreotide may provide the acromegaly community with a new treatment option offering a unique clinical profile and delivery system. Strongbridge has completed the screening of potential long-acting release (LAR) technologies for veldoreotide, and selected a proprietary formulation based upon PLGA microspheres for further development. PLGA is a well-known polymer, which has been widely applied in LAR formulations due to its biocompatibility, biodegradability, and favorable release kinetics. Veldoreotide has been granted orphan drug designation by the FDA and the EMA.

The safety and efficacy of RECORLEV and veldoreotide have not been established.

 

KEVEYIS Important Safety Information

What should you tell your healthcare provider before taking KEVEYIS?

Tell your doctor if you are allergic to dichlorphenamide or other sulfa drugs; if you take high doses of aspirin, or if you have lung or liver disease; if you are pregnant, plan to become pregnant, or are breastfeeding or plan to breastfeed.

 

What should you know about KEVEYIS?

  • Severe allergic and other reactions have happened with sulfonamides (drugs such as KEVEYIS) and have sometimes been fatal. Stop taking KEVEYIS at the first sign of skin rash, swelling, difficulty breathing, or any other unexpected side effect or reaction, and call 911 right away.

  • Tell your healthcare provider if you take aspirin or if another healthcare provider instructs you to begin taking aspirin. High doses of aspirin should not be taken with KEVEYIS.

  • KEVEYIS can cause your body to lose potassium, which can lead to heart problems. Your healthcare provider will measure the potassium levels in your blood before you start treatment and at certain times during treatment.

  • Tell your healthcare provider about all other prescription and over-the-counter medicines you take, including supplements, as some medicines can interact with KEVEYIS.

  • While taking KEVEYIS, your body may produce too much acid or may not be able to remove acid from the body. Your healthcare provider may run tests on a regular basis to check for signs of acid buildup.

  • KEVEYIS may increase your risk of falling. Tell your healthcare provider right away if you experience a fall while taking KEVEYIS.

  • The risks of falls and acid buildup are greater in elderly patients.

  • It is not known whether KEVEYIS is safe or effective for people younger than 18 years of age.

 

What are the most common side effects with KEVEYIS?

The most common side effects are a feeling of numbness, tingling or burning (“pins and needles”) in the toes, feet, hands or fingers; trouble with memory or thinking; feeling confused; and unpleasant taste in the mouth.

 

These are not all of the possible side effects of KEVEYIS. Call your healthcare provider for medical advice about side effects. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088.

Please see full Prescribing Information.

  1. Grieg SL. Dichlorphenamide: a review in primary periodic paralyses. Drugs. 2016;76:501- 507.
  2. Charles G, Zheng C, Lehmann-Horn F, Jurkatt-Rott, Levitt J. Characterization of hyperkalemic periodic paralysis: a survey of genetically diagnosed individuals. J Neurol. 2013;260:2606-2613.
  3. National Institutes of Health. Hyperkalemic periodic paralysis. https://ghr.nlm.nih.gov/condition/hyperkalemic-periodic-paralysis. Accessed December 5, 2016.
  4. National Institutes of Health. Hypokalemic periodic paralysis. https://ghr.nlm.nih.gov/condition/hypokalemic-periodic-paralysis. Accessed December 5, 2016.
  5. National Institutes of Health. Anderson-Tawil syndrome. https://ghr.nlm.nih.gov/condition/andersen-tawil-syndrome. Accessed December 5, 2016.
  6. National Institutes of Health. Potassium-aggravated myotonia. https://ghr.nlm.nih.gov/condition/potassium-aggravated-myotonia. Accessed December 5, 2016.
  7. National Institutes of Health. Paramyotonia congenita. https://ghr.nlm.nih.gov/condition/paramyotonia-congenita. Accessed December 5, 2016.
  8. Charles G, Zheng C, Lehmann-Horn F, Jurkatt-Rott, Levitt J. Characterization of hyperkalemic periodic paralysis: a survey of genetically diagnosed individuals. J Neurol. 2013;260:2606-2613.
  9. Cavel-Greant D, Lehmann-Horn F, Jurkat-Rott K. The impact of permanent muscle weakness on quality of life in periodic paralysis: a survey of 66 patients. Acta Myol. 2012;31:126-133.

 







© 2017 Strongbridge Biopharma plc
STRONGBRIDGE BIOPHARMA is a trademark of Strongbridge Biopharma plc.
KEVEYIS ® is a registered trademark licensed exclusively in the US to Strongbridge Biopharma plc.
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info@strongbridgebio.com

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