The announcement of Recordati Rare Diseases comes shortly after the drug became fully available for sale and distribution to adults in the United States.
The company said the drug will be available elsewhere in the European Union later this year. In order to allow clinicians greater flexibility for individualized treatment of patients, three dosages (1, 5 and 10 mg tablets) are available.
“Isturisa is a significant addition to treatment options for Cushing’s syndrome and helps meet the unmet needs of patients,” said Jacques Young, MD, PhD, in a press release. Young is a professor at Paris-Saclay University and a researcher in clinical trials of the drug.
Isturisa was approved by the European Commission as a treatment for people with endogenous Cushing’s syndrome earlier this year. Recordati recently acquired the rights to market the therapy from the original developer Novartis.
Isturisa works by blocking the activity of an enzyme called 11-beta-hydroxylase. This enzyme is necessary for the production of the hormone cortisol; high cortisol levels are the defining characteristic of Cushing’s syndrome.
The EC approval of Isturisa was based on data from the LINC-3 phase 3 clinical trial (NCT02180217), which recruited 137 people with Cushing’s disease. The participants all received Isturisa initially; then, some participants – those who had reached normal cortisol levels – continued on Isturisa, while others received a placebo, for eight weeks.
Compared to those who switched to placebo, a significantly higher number of participants who continued treatment had normal levels of cortisol in their urine after this eight-week randomization period (86% versus 29%).
The most common side effects of Isturisa were adrenal insufficiency, fatigue, nausea, headache and edema.
” [Isturisa] is supported by data generated by prospective clinical trials as part of Isturisa’s development program, providing strong evidence in terms of helping patients reach normal cortisol levels while improving clinical signs and symptoms and their quality of life, ”said Young.
“We have had the opportunity to benefit from Isturisa as part of a special access program in France since April 2019. The response to treatment has been extremely positive across the spectrum of severity of patients from Cushing, all the more so when I consider my patients who have gone from current treatment options, ”he added.