Specifically, the product was examined for its use in subjects where pituitary surgery is not an option or has not be curative.
Recordati Rare Diseases announced on Wednesday that it has seen positive results from the Phase III LINC-4 study of ISTURISA, also known as osilodrostat, for the treatment of patients with Cushing’s disease. Specifically, the product was examined for its use in subjects where pituitary surgery is not an option or has not be curative.
Information gathered from the Phase III study showed that a significantly higher proportion of patients receiving ISTURISA achieved normal mean urinary free cortisol – the main treatment goal for Cushing’s disease – after 12 weeks of treatment versus placebo. These improvements were sustained over 36 weeks of treatment, and ISTURISA was well tolerated in patients.
“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,” said Richard Feelders, MD, Professor of Endocrinology at the Erasmus University Medical Center, Rotterdam. “Data from this important Phase III study show that ISTURISA (osilodrostat) is an effective and well-tolerated therapy for Cushing’s disease, which significantly reduces and normalizes mUFC levels in most patients. These data are encouraging given the high unmet medical need for patients with this rare disorder.”
The data from this study now reinforces the clinical benefits of ISTURISA as a potentially effective and well-tolerated oral treatment option for patients with Cushing’s disease. The most common adverse reactions to ISTURISA, a cortisol synthesis inhibitor, are adrenal insufficiency, fatigue, nausea, headache, and oedema.
“The compelling topline LINC-4 data confirm the effectiveness of ISTURISA for the treatment of this rare, potentially life-threatening disease,” stated Andrea Recordati, CEO. “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.”
Back on June 2, Recordati Rare Diseases announced the commercial availability of ISTURISA in France. It became the first EU country to launch ISTURISA, and Recordati Rare Diseases intends to make the product available across select European Union markets over the course of 2020.
“ISTURISA is a meaningful addition to the treatment options for Cushing’s syndrome and helps address the unmet need of patients affected by this rare and debilitating disease,” said Jacques Young, MD, PhD, Professor of Medicine at University Paris Saclay.
The European Commission based its approval on data from the product’s development program, including the LINC-3 study, which met its primary endpoint. A significantly higher proportion of patients continued being treated ISTURISA and maintained normal mean urinary free cortisol at the end of the eight-week trial period.
“We had the opportunity to benefit from ISTURISA as part of a special access scheme in France since April 2019,” Young added. “Response to the treatment has been extremely positive across the severity spectrum of Cushing’s patients, even more so when I consider my patients that have switched from current treatment options.”
Back in March, Recordati Rare Diseases also announced that it had submitted a Japanese New Drug Application to the Japan’s Ministry of Health, Labour, and Welfare, for the approval of ISTURISA for patients with Cushing’s.
“We are excited about this regulatory filing for osilodrostat in Japan which, if approved, will provide a new and convenient oral treatment option that will help address patients’ needs in this underserved patient population” said Andrea Recordati, CEO, at the time of the announcement.
Recordati Rare Diseases is dedicated to reducing the impact of rare diseases by providing urgently needed therapies and products, such ISTURISA.
