Daiichi Sankyo’s Turalio becomes first FDA-approved therapy for rare tumour

pharmafile | August 5, 2019 | News story | Sales and Marketing Cancer, Daiichi Sankyo, FDA, Turalio, US, pharma, rare cancer 

Daiichi Sankyo has seen its CSF1R inhibitor Turalio (pexidartinib) approved by the FDA in the treatment of symptomatic tenosynovial giant cell tumours (TGCT) in adult patients whose disease is associated with severe morbidity or functional limitations and cannot be improved with surgery,  making it the first and only therapy available in the US for this indication.

The drug was approved in the rare tumour based on Phase 3 data which demonstrated that Turalio produced a 38% tumour response rate in patients according to the Response Evaluation Criteria v1.1 in Solid Tumors (RECIST) after 25 weeks of treatment, compared to 0% with placebo. Overall response rate by tumour volume score (TVS) was 56% over the same period, once again compared to 0% with placebo, while mean change from baseline in range of motion was shown to undergo a “statistically significant improvement”.

Despite these positive results, the approval of Turalio comes with a boxed warning over its hepatotoxicity and potential for fatal liver damage. During trials, two of the 768 participants suffered irreversible cholestatic liver injury, while one patient died as a result of advanced cancer and ongoing liver toxicity and one patient required a liver transplant.

Because of the risks posed by use of medicine, it will only be available through a restricted program called the TURALIO Risk Evaluation and Mitigation Strategy (REMS) Program, with only certified healthcare providers able to prescribe it.

“The FDA approval of Turalio represents a paradigm shift in the treatment of carefully selected TGCT patients who face significant disease morbidity and for whom surgery is not an option,” said Dr William D Tap, Chief of the Sarcoma Medical Oncology Service at Memorial Sloan Kettering Cancer Center and lead investigator for the Phase 3 ENLIVEN study. “We now have a new oral treatment option that can have a meaningful clinical benefit in select patients, including a reduction in tumour size.”

Dr Antoine Yver, Executive Vice President and Global Head, Oncology Research and Development at Daiichi Sankyo, also commented: “We are proud to be a part of today’s landmark approval and offer a much-needed treatment advancement for TGCT patients whose disease is not amenable to improvement with surgery, and who, until now, have had no approved systemic treatment options. With patients at the centre of everything we do, Daiichi Sankyo believes patient safety and providing effective medicines are our most important responsibilities. As such, we are committed to educating patients and the healthcare providers who care for them about the benefits and risks associated with Turalio to ensure appropriate prescribing and monitoring.”

Matt Fellows

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