Gavreto Wins Second FDA Approval in Three Months

 

The U.S. Food and Drug Administration (FDA) approved Genentech and Blueprint Medicines’ Gavreto (pralsetinib) as a treatment for adults and pediatric patients 12 and above who have been diagnosed with rearranged during transfection (RET)-mutant medullary thyroid cancer (MTC).

This marks the second approval for Gavreto this fall. In September, the drug was approved for the treatment of adults with metastatic rearranged during transfection (RET) fusion-positive non-small cell lung cancer (NSCLC). Genentech said the latest approval demonstrates the value of Gavreto across multiple RET-altered tumor types.

Developed by Blueprint Medicines, Gavreto is a once-daily, oral precision therapy designed to selectively target RET alterations, including fusions and mutations. The drug was granted Breakthrough Therapy Indication for the latest indication it won approval.

Earlier this year, Genentech and Blueprint Medicines inked a $1 billion pact to develop and commercialize treatments for cancers driven by oncogenic RET alteration. Blueprint Medicines and Roche agreed to co-develop Gavreto globally in RET-altered solid tumors, including NSCLC, MTC and other thyroid cancers, as well as other solid tumors. The companies also plan to expand development of pralsetinib in multiple treatment settings and explore the development of a next-generation RET inhibitor as part of this collaboration.

RET gene alterations, such as fusions and mutations, are key disease drivers in many types of cancer, including NSCLC and several types of thyroid cancers. Approximately 10-20% of people with papillary thyroid cancer, which is the most common type of thyroid cancer, have RET fusion-positive tumors, and roughly 90% of people with advanced MTC, a rare form of thyroid cancer, carry RET mutations. Biomarker testing for RET fusions and mutations can help identify people who are eligible for treatment with Gavreto, Genentech said in its announcement.

For the latest indication, specifically, the FDA approved Gavreto for patients with advanced or metastatic RET- mutant medullary thyroid cancer (MTC) who require systemic therapy, or with advanced or metastatic RET fusion-positive thyroid cancer who require systemic therapy and who are radioactive iodine-refractory, if radioactive iodine is appropriate for treatment. The approval was based on data from the Phase I/II ARROW study, which showed patients treated with Gavreto demonstrated durable clinical activity in people with or without prior therapy, regardless of RET alteration genotypes. Treatment with Gavreto led to an overall response rate of 60% n 55 people with RET-mutant metastatic MTC previously treated with cabozantinib and/or vandetanib. In 29 people with RET-mutant advanced MTC who were cabozantinib and vandetanib treatment-naïve, the ORR was 66%. In nine people with RET fusion-positive metastatic thyroid cancer, Gavreto demonstrated an ORR of 89%.

Approval was conducted under the FDA’s Real-Time Oncology Review pilot program that accelerates the review process of cancer treatments that are deemed safe and effective.

Levi Garraway, Genentech’s chief medical officer and head of Global Product Development, said the latest approval for Gavreto underscores the company’s commitment to advancing personalized healthcare with treatments that target the underlying biology of each person’s cancer.