The current clinical focus of our rare endocrine franchise is on developing new therapies to treat rare endocrine disorders, such as Cushing's syndrome. Our lead product candidate is levoketoconazole, an investigational adrenal steroidogenesis inhibitor for endogenous Cushing's syndrome.
Endogenous Cushing’s syndrome, characterized by sustained elevated cortisol levels, presents unique clinical challenges to patients and healthcare providers.
The safety and efficacy of 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.
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 is not easily diagnosed and may go untreated for many years.3,4
As they age, patients may experience permanent muscle weakness.3
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.
Please see KEVEYIS Full Prescribing Information.