Rare Disease Portfolio

Dedicated to bridging neuromuscular and endocrinology treatment gaps

Our franchise-based model allows us to leverage company expertise—in both the clinical and commercial development realms—to provide a range of therapeutically aligned products.

Rare Disease Assets

In addition to our lead drug candidates, which have begun clinical development and have received orphan drug designation from the FDA and the European Medicines Agency, we have the US rights to KEVEYIS® (dichlorphenamide), the first and only FDA-approved treatment for hyperkalemic, hypokalemic, and related variants of Primary Periodic Paralysis. KEVEYIS has been granted orphan drug exclusivity in the United States.

Please see KEVEYIS Important Safety Information and Full Prescribing Information.

A Growing Portfolio of Products

Levoketoconazole: Adrenal Steroidogenesis Inhibitor for Endogenous Cushing’s Syndrome*

Levoketoconazole, previously studied in SONICS and LOGICS, and currently being studied in the OPTICS phase 3 trial for patients with endogenous Cushing's syndrome. Originally developed as a potential treatment for patients currently with diabetes, Strongbridge pivoted the focus of clinical development for levoketoconazole to treatment of endogenous Cushing’s syndrome in 2012. Read more about levoketoconazole.

Learn about levoketoconazole for Cushing's syndrome

Veldoreotide Modified Release: Novel Somatostatin Analogue*

Veldoreotide modified release—a next-generation somatostatin analogue with potential applications in endocrine and non-endocrine conditions that are amenable to somatostatin receptor activation.

Read about veldoreotide

KEVEYIS: The First and Only FDA-Approved Treatment for Hyperkalemic, Hypokalemic, and Related Variants of Primary Periodic Paralysis

KEVEYIS is proven to reduce the number, severity, and duration of attacks in patients with Primary Periodic Paralysis.1,2 Read more about KEVEYIS.

Discover KEVEYIS

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

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. KEVEYIS [package insert]. Feasterville-Trevose, PA: Strongbridge Biopharma.
  2. Sansone VA, Burge J, McDermott MP, et al; for the Muscle Study Group. Randomized, placebo-controlled trials of dichlorphenamide in periodic paralysis. Neurology. 2016;86(15):1408-1416.