Turalio Approved to Treat Rare Tumor of the Joints
August 2, 2019 – The U.S. FDA has approved Turalio™ (pexidartinib), manufactured by Daiichi Sankyo, to treat symptomatic tenosynovial giant cell tumor (TGCT) associated with severe morbidity or functional limitations and not amenable to improvement with surgery in adult patients. It is the first and only FDA-approved treatment for this indication.
TGCTs are rare, non-cancerous tumors that affect the synovium (a thin layer of tissue covering the interior surfaces of joint spaces), bursae (small, fluid-filled structures providing cushioning around joints), and tendon sheath (fibrous tissue connecting muscle and bone). They do not spread to other parts of the body, but can damage surrounding areas by causing the synovium, bursae, and tendon sheaths to overgrow. This leads to pain, inflammation, and limited mobility in the affected joints. Although surgery is often used to treat TGCTs, recurrent, difficult-to-treat, or diffuse TGCTs can wrap around nearby structures and make surgical resection a poor treatment option.
In a clinical study, the tumor response rate, as measured by Response Evaluation Criteria in Solid Tumors (RECIST) was 38% in patients who received Turalio and 0% in those who received placebo. RECIST is a standard measure of tumor response to treatment. The overall response rate as measured by tumor volume score (TVS) was 56% in patients who received Turalio versus 0% in patients who received placebo. Patients treated with Turalio experienced a statistically significant improvement in range of motion based on the mean change from baseline when compared to patients who received a placebo.
Recommended dosing is 400mg (two 200mg capsules) twice daily, taken on an empty stomach at least one hour before and two hours after consuming a meal or snack. A black box warning cautions that Turalio can cause serious and potentially fatal liver injury. Liver tests should be conducted before and during treatment with Turalio.
Daiichi Sankyo has launched the product at a wholesale acquisition cost (WAC) of $165 per capsule, or approximately $240,000 per year of therapy.