This segment focuses on the development and commercialization of therapies for rare diseases with significant unmet medical needs. Research and development activities include clinical trials for Recorlev, a cortisol synthesis inhibitor for Cushing's syndrome, and Veldoreotide, a somatostatin analog for acromegaly. The company utilizes small molecule drug development and clinical trial methodologies. The primary therapeutic area is rare endocrine disorders, specifically Cushing's syndrome and acromegaly. The patient impact is significant, as these therapies aim to improve the quality of life for patients suffering from these debilitating conditions. Strongbridge Biopharma's market positioning is as a specialist in rare disease treatments, with a competitive advantage in its focus on underserved patient populations. Future opportunities include expanding the indications for existing drugs and developing new therapies for other rare endocrine disorders. Regulatory and clinical aspects are crucial, with the company navigating FDA approval processes and conducting Phase III clinical trials. Partnerships and collaborations are essential for clinical trial execution and commercialization efforts.

This segment encompasses the commercialization and distribution of Keveyis, an oral carbonic anhydrase inhibitor for the treatment of hyperkalemic, hypokalemic, and related variants of primary periodic paralysis in the United States. The segment's activities include sales, marketing, and patient support services. The technology used is the formulation and delivery of an oral medication. The therapeutic area is neuromuscular disorders, specifically primary periodic paralysis. The patient impact is significant, as Keveyis provides a treatment option for a rare genetic disorder, improving the quality of life for patients. The market positioning is as a provider of a specific treatment for a rare disease, with a competitive advantage in its established presence in the US market. Future opportunities include expanding the market for Keveyis and exploring potential new indications. Regulatory and clinical aspects include post-market surveillance and ongoing monitoring of patient outcomes. Partnerships and collaborations may involve patient advocacy groups and healthcare providers.