An FDA panel of outside experts gave Novartis ($NVS) some good news going into its R&D day today. The panel provided a unanimous thumbs up for Signifor (pasireotide), a new therapy for Cushing's disease.
The unanimous recommendation tied to an internal FDA review endorsing the efficacy of the drug sets up a likely approval for Novartis, one of many biopharma companies looking to expand its presence in the rare disease field. The FDA review noted that the low dose in the pivotal 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--but 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 ?g bid and 900 ?g bid doses are effective and that they are not far apart on the dose response curve."
"We are encouraged by today's favorable advisory committee recommendation for pasireotide in Cushing's disease and will work closely with the FDA as it completes its review of our application," said Herve Hoppenot, president of Novartis Oncology. "There is a significant unmet medical need for Cushing's disease patients and Novartis is committed to providing the endocrinology community with a novel therapeutic approach for this rare and debilitating endocrine disorder."
Cushing's disease is a form of Cushing's syndrome, a rare endocrine disorder caused by excessive cortisol, which regulates metabolism, cardiovascular function and helps the body respond to stress.
- here's the press release
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