Rare Disease Portfolio

Strongbridge is dedicated to bridging treatment gaps in rare diseases like Primary Periodic Paralysis, endogenous Cushing’s syndrome, and acromegaly.

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 been granted orphan drug designation from both the FDA and EMA, 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 also been granted orphan drug designation by the FDA.

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

A Growing Portfolio of Products

Target Disease
Phase 1
Phase 2
Phase 3



Primary Periodic Paralysis (FDA-approved; FDA orphan drug designation)




Endogenous Cushing's syndrome (FDA and EMA orphan drug designation)

Phase 3



Acromegaly (FDA and EMA orphan drug designation)

Phase 2

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

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

RECORLEV (levoketoconazole): Cortisol Synthesis Inhibitor for Endogenous Cushing’s Syndrome*

RECORLEV (levoketoconazole) is being evaluated in LOGICS and SONICS, phase 3 trials for patients with endogenous Cushing’s syndrome. Originally developed as a potential treatment for patients with diabetes, Strongbridge pivoted the focus of clinical development for RECORLEV (levoketoconazole) to treatment of endogenous Cushing’s syndrome in 2012. Read more about RECORLEV (levoketoconazole).

Veldoreotide: Novel Somatostatin Analogue for Acromegaly*

Veldoreotide is a somatostatin analogue (SSA) with a differential somatostatin receptor subtype binding profile.

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

Indication and Important Safety Information

What is KEVEYIS?

KEVEYIS (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 sulfur 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 Important Safety Information and Full Prescribing Information.


  1. KEVEYIS® [package insert]. Trevose, PA: Strongbridge Biopharma; 2015.
  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;86:1408-1416.

© 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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