Rare Disease Portfolio
Strongbridge is dedicated to bridging treatment gaps in rare diseases like Primary Periodic Paralysis, Adult Growth Hormone Deficiency, 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 received orphan drug designation from the FDA and the European Medicines Agency, we have the U.S. and Canadian rights to MacrilenTM (macimorelin), the first and only FDA-approved oral drug for the diagnosis of Adult Growth Hormone Deficiency.
We also have the U.S. rights to KEVEYIS® (dichlorphenamide), the first and only FDA-approved treatment for hyperkalemic, hypokalemic, and related variants of Primary Periodic Paralysis. KEVEYIS and Macrilen have both been granted orphan drug exclusivity in the United States.
Please see KEVEYIS Important Safety Information and Full Prescribing Information.
Please see Macrilen Important Safety Information and Full Prescribing Information.
A Growing Portfolio of Products
Indication/ |
KEVEYIS®(dichlorphenamide) Primary Periodic Paralysis (FDA-approved; FDA orphan drug designation) |
Macrilen™(macimorilen) Adult Growth Hormone Deficiency diagnosis (FDA-approved; FDA orphan drug designation) |
RECORLEV™(levoketoconazole) Endogenous Cushing's syndrome (FDA and EMA orphan drug designation) |
veldoreotidemodified-release Acromegaly (FDA and EMA orphan drug designation) and other conditions that are modifiable through activation of somatostatin receptors, such as Cushing's disease and neuroendocrine tumors |
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.
Macrilen: The First and Only FDA-Approved Drug for the Diagnosis of Adult Growth Hormone Deficiency.
Macrilen, an oral growth hormone secretagogue receptor agonist, is indicated for the diagnosis of adult growth hormone deficiency (AGHD). Read more about Macrilen.
RECORLEV (levoketoconazole): Cortisol Synthesis Inhibitor for Endogenous Cushing’s Syndrome*
RECORLEV (levoketoconazole) is currently being evaluated in LOGICS and SONICS, phase 3 trials 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 RECORLEV (levoketoconazole) to treatment of endogenous Cushing’s syndrome in 2012. Read more about RECORLEV (levoketoconazole).
Veldoreotide extended release: Novel Somatostatin Analogue for Acromegaly*
Veldoreotide extended release: A pre-clinical next-generation somatostatin analog being investigated for the treatment of acromegaly and potential additional applications in other conditions amenable to somatostatin receptor activation.
* The safety and efficacy of RECORLEV (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 Important Safety Information and Full Prescribing Information.
Important Safety Information for Macrilen
What is Macrilen?
Macrilen (pronounced ma-kri-len) (macimorelin) is a prescription oral solution that is used to test for adult growth hormone deficiency (AGHD).
What should you know about Macrilen?
- Taking Macrilen with certain other medications may cause irregular changes to your heart rhythm. Before taking Macrilen, tell your healthcare provider about all your medications, as you may need to temporarily stop taking some medications before you take Macrilen.
- Some medications may cause a false positive result when taken with Macrilen. Before taking Macrilen, tell your healthcare provider about all the medications you take, including growth hormone.
- Tell your healthcare provider if you were recently diagnosed with hypothalamic disease, as this can cause a false negative result with Macrilen.
- You will need to fast (go without food) for at least 8 hours before taking Macrilen.
What are the most common side effects with Macrilen?
The most common side effects were changed sense of taste, dizziness, headache, fatigue, nausea, hunger, diarrhea, upper respiratory tract infection, feeling hot, excessive sweating, sore nose and throat, and decreased heart rate.
These are not all of the possible side effects of Macrilen. 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 Macrilen Important Safety Information and Full Prescribing Information.
References
- KEVEYIS® [package insert]. Trevose, PA: Strongbridge Biopharma; 2015.
- 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.