Novartis has secured approval from the US Food and Drug Administration (FDA) for its Isturisa (osilodrostat) oral tablets to treat adults with Cushing’s disease.
The FDA approval allows Isturisa to treat adults with Cushing’s disease who either cannot undergo pituitary gland surgery or have undergone the surgery but still suffering from the disease.
Isturisa is claimed to be the first FDA-approved drug to directly control the cortisol overproduction by restricting the 11-beta-hydroxylase enzyme and prevent cortisol synthesis.
Cushing’s disease is induced due to pituitary tumour, which releases too much of adrenocorticotropin hormone that stimulates the adrenal gland to generate an excessive amount of cortisol.
The safety and effectiveness Isturisa to treat Cushing’s disease were assessed in a trial of 137 adult patients with a mean age of 41 years.
All patients secured a starting dose of 2mg of Isturisa twice a day that could be increased every two weeks up to 30mg twice a day in the 24-week, single-arm and open-label period study.
Around half of the patients had cortisol levels within normal limits at the end of this 24-week period
According to the FDA, 86% of patients receiving Isturisa maintained cortisol levels within normal limits compared to 30% of patients taking the placebo at the end of this withdrawal period.
The FDA’s Center for Drug Evaluation and Research acting director Dr Mary Thanh Hai said: “The FDA supports the development of safe and effective treatments for rare diseases, and this new therapy can help people with Cushing’s disease, a rare condition where excessive cortisol production puts them at risk for other medical issues.
“By helping patients achieve normal cortisol levels, this medication is an important treatment option for adults with Cushing’s disease.”
In February, Novartis secured priority review for its MET inhibitor capmatinib (INC280) from the US Food and Drug Administration (FDA) for the treatment of a type of non-small cell lung cancer (NSCLC).