Posted by Michael Wonder on 03 Aug 2019
FDA approves first therapy for rare joint tumour
2 August 2019 - Today, the U.S. FDA granted approval to Turalio (pexidartinib) capsules for the treatment of adult patients with symptomatic tenosynovial giant cell tumour associated with severe morbidity or functional limitations and not responsive to improvement with surgery.
The approval of Turalio was based on the results of a multi-center international clinical trial of 120 patients, 59 of whom received placebo.
The FDA granted this application breakthrough therapy designation and priority review designation. Turalio also received orphan drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases. The FDA granted the approval of Turalio to Daiichi Sankyo.
