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
Endogenous Cushing’s syndrome, characterized by sustained elevated cortisol levels, presents unique clinical challenges to patients and healthcare providers.
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.
The safety and efficacy of RECORLEV™ (levoketoconazole) and veldoreotide have not been established.
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
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?
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.
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?
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.
Learn more about this first and only FDA-approved treatment for patients with a rare disease.
© 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.
MLR-0010 V13 01/2018