Our Therapeutic Focus

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

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.

Primary Periodic Paralysis (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 often interferes with daily activities as attacks are unpredictable and vary in severity and duration. As they age, patients may experience permanent muscle weakness.3 Symptoms are often nonspecific with varying clinical presentations; PPP is not easily diagnosed and may go untreated for many years.3,4 Although there are related variants, the most common forms are hyperkalemic and hypokalemic PPP.5-9

Learn more about Primary Periodic Paralysis Learn about KEVEYIS Please see KEVEYIS Important Safety Information below and Full Prescribing Information.

Rare Endocrine Franchise

The focus of our rare endocrine franchise is Adult Growth Hormone Deficiency (AGHD), Cushing's syndrome, and acromegaly. Our commercial effort is on MacrilenTM (macimorelin), the first and only FDA-approved drug for the diagnosis of AGHD. Our clinical effort is on developing new therapies to treat the rare endocrine disorders of Cushing's syndrome and acromegaly.

Adult Growth Hormone Deficiency (AGHD) is typically caused by injury/insult to the pituitary and leads to cardiovascular, neuromuscular, metabolic, mental health, and skeletal issues.10

Learn more about Adult Growth Hormone Deficiency Learn more about Macrilen for the diagnosis of Adult Growth Hormone Deficiency Please see Macrilen Important Safety Information below and Full Prescribing Information.

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

Acromegaly can be difficult to diagnose, and difficult to treat. Patients with acromegaly face numerous health issues related to their condition, and have limited treatment options available.

Learn more about acromegaly Learn about veldoreotide, a somatostatin analogue 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

  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;3:374-38
  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;31:126–133
  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;260:2606-2613.
  5. National Institutes of Health. Hyperkalemic periodic paralysis. Available at: https://ghr.nlm.nih.gov/condition/hyperkalemic-periodic-paralysis. Accessed September 27, 2017.
  6. National Institutes of Health. Hypokalemic periodic paralysis. Available at: https://ghr.nlm.nih.gov/condition/hypokalemic-periodic-paralysis. Accessed September 27, 2017.
  7. National Institutes of Health. Anderson-Tawil syndrome. Available at: https://ghr.nlm.nih.gov/condition/andersen-tawil-syndrome. Accessed September 27, 2017.
  8. National Institutes of Health. Potassium-aggravated myotonia. Available at: https://ghr.nlm.nih.gov/condition/potassium-aggravated-myotonia. Accessed September 27, 2017.
  9. National Institutes of Health. Paramyotonia congenita. Available at: https://ghr.nlm.nih.gov/condition/paramyotonia-congenita. Accessed September 27, 2017.
  10. Gupta V. Adult growth hormone deficiency. Indian J Endocrinol Metab. 2011;15(Suppl 3):S197-S202.

© 2018 Strongbridge Biopharma plc
STRONGBRIDGE BIOPHARMA® is a registered trademark of the Strongbridge Biopharma plc. companies, which include Strongbridge Ireland Limited and Strongbridge U.S. Inc.
KEVEYIS® is a registered trademark licensed exclusively in the U.S. to Strongbridge Biopharma plc.
Macrilen is a trademark of Aeterna Zentaris GmbH, licensed exclusively in the U.S. and Canada to Strongbridge Ireland Limited.
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