Topline data show safety, efficacy of Cushing’s disease therapy
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Adults with Cushing’s disease who took the cortisol synthesis inhibitor osilodrostat were more likely to sustain a normal mean urinary free cortisol level vs. placebo, according to press release from Recordati Rare Diseases.
Data from the phase 3 LINC-4 study also demonstrate that osilodrostat (Isturisa, Novartis) was well tolerated with a manageable safety profile. The most common adverse events were arthralgia, decreased appetite, fatigue and nausea, according to the company.
“Cushing’s disease is a chronic and debilitating condition that can be extremely challenging to manage and, if left inadequately treated, can have a significant impact on patients’ quality of life and increase the risk of mortality,” Richard Feelders, MD, professor of endocrinology at Erasmus University Medical Center, Rotterdam, said in the release. “Data from this important phase 3 study show that Isturisa is an effective and well-tolerated therapy for Cushing’s disease, which significantly reduces and normalizes mean urinary free cortisol levels in most patients. These data are encouraging given the high unmet medical need for patients with this rare disorder.”
Osilodrostat, which was approved by the FDA in March, is an oral therapy originally developed for hypertension management. It is the first FDA-approved drug to directly inhibit 11-beta-hydroxylase, preventing cortisol synthesis, according to the FDA. The drug is indicated for the treatment of adult patients with Cushing’s disease for whom pituitary surgery is not an option or has not been curative.
In LINC-4, a significantly higher proportion of patients assigned osilodrostat achieved a normal mean urinary free cortisol level at 12 weeks compared with those assigned placebo (77% vs. 8%; P < .0001). Improvements in mean urinary free cortisol were sustained during 36 weeks of treatment for 81% of participants, according to the release.
“The compelling topline LINC-4 data confirm the effectiveness of Isturisa for the treatment of this rare, potentially life-threatening disease,” Andrea Recordati, CEO of Recordati Rare Diseases, said in the release. “We are deeply grateful to the patients, investigators, clinicians and study staff whose ongoing participation in the clinical development of Isturisa has helped bring this therapy to patients in need.”
As Healio previously reported, the therapy joins two other approved treatments for Cushing’s disease. In June 2018, the FDA approved pasireotide (Signifor, Novartis) for the treatment of people with Cushing’s disease for whom pituitary surgery is not an option or has not been curative. In 2012, the FDA approved mifepristone (Korlym, Corcept Therapeutics) as a once-daily oral medication to treat adults with Cushing’s syndrome who had elevated blood glucose due to type 2 diabetes.
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