Optimizing Treatment for Rare Genetic Condition: Cushing’s Syndrome

Cushing’s syndrome, a rare endocrine disorder and FDA approved treatments.
· 10 min read

Cushing’s syndrome (CS) is a rare endocrine disorder that results from chronic exposure to excessive amounts of the hormone cortisol that may come from inside the body or an external source. For example, the syndrome can be a side effect of prolonged use of high doses of a corticosteroid medications (e.g., prednisone, dexamethasone) for conditions such as lupus or rheumatoid arthritis. CS can also be caused by the body over-producing cortisol as a result of a pituitary or adrenal tumor. Cushing’s Disease, a specific type of CS, occurs when excess adrenocorticotropin hormone (ACTH) production from a benign pituitary tumor causes the adrenal glands to produce excessive amounts of cortisol.

Medications that are FDA approved for the treatment of CS include Korlym® (mifepristone), Signifor® and Signifor® LAR (pasireotide), Isturisa® (osilodrostat) and Recorlev® (levoketoconazole).

All the current medication options are very expensive, ranging from $170,000 to over $420,000 annually. Generics are not expected to enter the market for CS before at least 2026, so current pricing will likely continue for the near future.

AscellaHealth’s end-to-end Specialty Pharmacy solutions enhance provider and patient education, increase compliance and persistency and improve patient access to high-cost specialty medications.

To learn more about the potential cost-saving opportunities for rare disease drugs, click here.

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