FDA authorises first therapy to tackle cortisol overproduction in Cushing’s disease
pharmafile | March 9, 2020 | News story | Sales and Marketing | Cushing's disease, FDA, pharma
The FDA has moved to approve its first therapy to directly treat the overproduction of the hormone cortisol which is a hallmark of the rare condition Cushing’s disease. The US agency announced that it has authorised Isturisa (osilodrostat), manufactured by Italian firm Recordati, in patients who are ineligible for pituitary gland surgery or are still affected by the condition despite receiving surgery.
In clinical trials of 137 adult participants with a mean age of 41, around half of patients achieved normal levels of cortisol after 24 weeks of treatment. Additionally, 71 participants who tolerated the drug for the final 12 weeks and also do not require dosage increases were put into a further eight-week withdrawal period. By the end of this withdrawal period, 86% of participants had maintained normal cortisol levels, compared to 30% with placebo.
“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,” remarked Dr Mary Thanh Hai, Acting Director of the Office of Drug Evaluation II in the FDA’s Center for Drug Evaluation and Research. “By helping patients achieve normal cortisol levels, this medication is an important treatment option for adults with Cushing’s disease.”
Cushing’s disease occurs when a pituitary tumour releases an abundance of adrenocorticotropin, which in turn stimulates the adrenal gland’s production of cortisol. Isturisa works to counteract this by blocking the enzyme 11-beta-hydroxlase, preventing cortisol synthesis.
The condition affects three times more women than it does men, with those between 30 and 50 most commonly affected. It can lead to a number of health issues including type 2 diabetes, obesity, high blood pressure, a compromised immune system, depression, blood clots and bone fractures.
Matt Fellows
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