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 acquired 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.

A Growing Portfolio of Products

Target Disease
Phase I
Phase II
Phase III



Primary Periodic Paralysis (FDA Approved) (FDA orphan drug designation)




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

Phase III



Acromegaly (FDA and EMA orphan drug designation)

Phase II

KEVEYIS®: The First and Only FDA-Approved Treatment for Hyperkalemic, Hypokalemic, and Related Variants of Primary Periodic Paralysis1

KEVEYIS is proven to reduce attacks of primary periodic paralysis.1 Read more about KEVEYIS®.

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

RECORLEV (levoketoconazole) is being evaluated in SONICS, a phase 3 trial 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.

KEVEYIS® Indication

KEVEYIS® is indicated for the treatment of primary hyperkalemic periodic paralysis, primary hypokalemic periodic paralysis, and related variants.1

Important Safety Information

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.

KEVEYIS is not for everyone. Do not take KEVEYIS if you:

  • Are on a high-dose aspirin regimen
  • Are allergic to sulfa-based drugs
  • Have liver, kidney, or certain lung conditions
  • Are pregnant, planning to become pregnant, or nursing
  • Are under 18 years old

Taking KEVEYIS may cause a drop in the amount of potassium (an electrolyte) in your body, which can lead to heart problems. Ask your doctor if you need to eat foods that contain high amounts of potassium while taking KEVEYIS.

Your body may produce too much acid or may not be able to remove enough acid from body fluids while taking KEVEYIS. Your doctor will run tests on a regular basis to check for signs of acid buildup and may reduce your dose or stop your treatment with KEVEYIS.

KEVEYIS may also increase the risk of falls, especially in elderly patients and patients taking high doses of KEVEYIS. Use caution when driving, operating machinery, or performing any other hazardous activities while taking KEVEYIS, as this medication may cause drowsiness.

You are encouraged to report side effects to Strongbridge Biopharma at 1-855-324-8912, or to the FDA at 1-800-FDA-1088 or visit www.fda.gov/medwatch/. For more information, go to www.keveyis.com.

For additional KEVEYIS important safety information, please see full Prescribing Information (PDF).


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

© 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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Trevose, PA 19053
United States
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