Our Therapeutic Focus

Strongbridge Biopharma is building a portfolio of vertical, therapeutically aligned rare disease franchises

Rare Endocrine Franchise

The current clinical focus of our rare endocrine franchise is on developing new therapies to treat rare endocrine disorders, such as Cushing's syndrome. Our lead product candidate is levoketoconazole, an investigational adrenal steroidogenesis inhibitor for endogenous Cushing's syndrome.

Endogenous Cushing’s syndrome, characterized by sustained elevated cortisol levels, presents unique clinical challenges to patients and healthcare providers.

Learn more about endogenous Cushing’s syndrome.

Learn about LOGICS and SONICS, phase 3 clinical trials for patients with endogenous Cushing’s syndrome.

Learn about veldoreotide modified release, a preclinical next-generation somatostatin analogue.

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

Rare Neuromuscular Franchise

The focus of our rare neuromuscular franchise is Primary Periodic Paralysis (PPP). Our commercial effort is on KEVEYIS® (dichlorphenamide), the first and only FDA-approved treatment for hyperkalemic, hypokalemic, and related variants of PPP.

PPP is a spectrum of rare and chronic genetic disorders that cause recurrent, progressive, and debilitating attacks of muscle weakness and temporary paralysis.1,2

PPP is not easily diagnosed and may go untreated for many years.3,4

As they age, patients may experience permanent muscle weakness.3

Learn more about Primary Periodic Paralysis.

Learn about KEVEYIS.

Please see KEVEYIS Important Safety Information below and Full Prescribing Information.

Important Safety Information for KEVEYIS

What is KEVEYIS?

KEVEYIS (pronounced keh-VAY-iss) (dichlorphenamide) is a prescription drug used to treat primary hyperkalemic periodic paralysis, primary hypokalemic periodic paralysis, and other similar diseases.

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 KEVEYIS Full Prescribing Information.


  1. Cannon SC. Channelopathies of skeletal muscle excitability. Compr Physiol. 2015;5:761-790.
  2. Arya SN. Periodic paralysis. Journal, Indian Academy of Clinical Medicine. 2002;3:374-382.
  3. 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(2):126-133.
  4. Charles G, Zheng C, Lehmann-Horn F, Jurkatt-Rott K, Levitt J. Characterization of hyperkalemic periodic paralysis: a survey of genetically diagnosed individuals. J Neurol. 2013;260(10):2606-2613.