FDA review endorses efficacy of Novartis' new drug for Cushing's disease

Novartis ($NVS) got a helpful assist in its quest to bring a new therapy for rare cases of Cushing's disease to the market. In an FDA review ahead of a scheduled discussion by outside experts on Wednesday, the agency's internal assessment concluded that there was clear efficacy data supporting the use of the drug from both doses that were studied in a pivotal trial.

Although the low dose in the study didn't hit the primary efficacy criterion in the study--at least a 15% improvement in urinary free cortisol normalized after 6 months of therapy compared to a prespecified threshold--the agency's reviewer concluded that it had an impact that was quite similar to the high dose: "The totality of data seems to suggest that both the 600 μg bid and 900 μg bid doses are effective and that they are not far apart on the dose response curve."

Signifor, or pasireotide injection, was designed to treat hypercortisolemia, a disruption in the normal ebb and flow of the hormone cortisol. In Cushing's disease, a tumor in the pituitary gland can generate ACTH, which spurs excess levels of cortisol. The FDA recently approved the first drug for Cushing's syndrome--Korlym, or mifepristone, from Corcept Therapeutics--to control hyperglycemia and hypertension. Approved in February, Korlym blocks cortisol, helping to control blood pressure in patients.

Only a few thousand patients suffer from Cushing's disease in the U.S. But as we've seen repeatedly, small patient populations can generate some sizeable revenue numbers for drug developers, making this a key target for a large group of biotech and pharma companies.

- here's the FDA review
- read the Bloomberg report