Gavreto Receives New Thyroid Cancer Indications

December 1, 2020 – The U.S. FDA has approved two new indications for GavretoTM (pralsetinib), manufactured by Roche and Blueprint Medicines, to treat certain types of thyroid cancer. The drug is now indicated for use in patients who are at least 12 years old and diagnosed with:

RET activating fusions and mutations are drivers of the development of many types of cancer, including some thyroid cancers. RET fusions are associated with up to 20% of papillary thyroid cancers (the most common type of thyroid cancer), and RET mutations are implicated in approximately 90% of advanced MTC cases. Gavreto is designed to specifically target RET alterations associated with the patient’s disease, which can help reduce the occurrence of side effects and improve clinical efficacy compared to non- targeted treatments.

In a clinical trial, Gavreto produced an overall response rate (ORR) of 60% in patients diagnosed with RET- mutant MTC who had received prior treatment with cabozantinib or vandetanib (current standards of care). Gavreto also produced an ORR of 66% in patients with RET-mutant MTC who were cabozantinib and vandetanib treatment-naïve. For patients diagnosed with RET fusion-positive thyroid cancer, the ORR with Gavreto was 89%. To date, the median duration of response (DOR) has not yet been reached for patients in the trial who have responded to treatment.

Patients should not consume any food for at least two hours before and at least one hour after taking Gavreto. The recommended dosing is 400mg taken by mouth once daily until disease progression or unacceptable toxicity occur.

Gavreto first received FDA approval in 2020 to treat metastatic RET fusion-positive non-small cell lung cancer. All current indications are approved through the FDA’s accelerated approval pathway, meaning that continued approval may be contingent upon verification and description of Gavreto’s clinical benefit in confirmatory trials.