FDA approves new orphan drug for Cushing’s disease

Hannah Blake

pharmaphorum

The US FDA has approved a new orphan drug to treat patients with Cushing’s disease who cannot be helped through surgery.

Novartis’ Signifor (pasireotide diaspartate) injection has been approved to be administered twice daily. It is the first medicine to be approved in the US that addresses the underlying mechanism of Cushing’s disease, a serious, debilitating endocrine disorder caused by the presence of a non-cancerous pituitary tumor which ultimately leads to excess cortisol in the body. Cushing’s disease is a rare but serious disease that affects approximately one to two patients per million per year.

This approval follows a unanimous recommendation from the FDA Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) in support of the use of Signifor.

“The FDA approval of Signifor for Cushing’s disease brings a novel pituitary-directed therapy to patients with limited treatment options. Today's milestone reinforces Novartis’ commitment to addressing unmet needs and advancing treatments for rare pituitary-related disorders.”

Hervé Hoppenot, President, Novartis Oncology.

The approval was based on a clinical trial of 162 Cushing’s disease patients, evaluating the safety and effectiveness of Signifor. Trial participants were randomly chosen to receive one of two dose levels of Signifor over a six-month treatment period. Some patients who safely responded to the medication where allowed to continue treatment. Signifor resulted in decreased cortisol levels as measured in urine collected over a 24-hour period. This reduction was seen as early as one month after starting treatment. About 20% of patients in the clinical trial were able to reduce urine cortisol levels into the normal range.

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