Gavreto is indicated for metastatic RET-mutant medullary thyroid cancer (MTC) in adult and paediatric patients, aged 12 years and above

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Genentech's sprawling headquarters campus in South San Francisco. (Credit: Coolcaesar/Wikipedia.)

Roche’s subsidiary Genentech and partner Blueprint Medicines have received the US Food and Drug Administration (FDA) approval for Gavreto (pralsetinib) to treat a type of advanced medullary thyroid cancer (MTC).

The US regulatory agency indicated Gavreto for metastatic rearranged during transfection (RET)-mutant medullary thyroid cancer (MTC) in adult and paediatric patients, aged 12 years and above, who require systemic therapy.

Gavreto is a once-daily oral precision therapy, developed by Blueprint Medicines to selectively target RET alterations that drive multiple tumour types.

Blueprint Medicines is commercialising Gavreto in the US, together with Genentech, under its $1.7bn collaboration and licensing agreement with Roche.

Roche chief medical officer and global product development head Levi Garraway said: “We are proud to partner with Blueprint Medicines to bring this important new option to people with certain types of RET-altered thyroid cancer.

“Gavreto is now approved across multiple RET-altered tumour types, underscoring our commitment to advancing personalised healthcare with treatments that target the underlying biology of each person’s cancer.”

FDA approval for Gavreto is based on data from Phase 1/2 ARROW study

According to the company, around 10 to 20% of people with papillary thyroid cancer, which is the most common type of thyroid cancer, have RET fusion-positive tumours, and nearly 90% of them with advanced MTC carry RET mutations.

FDA granted the regulatory approval for Gavreto under its accelerated approval programme, based on data from ARROW, a Phase 1/2, open-label, first-in-human clinical study.

The study is designed to evaluate the safety, tolerability and efficacy of orally administered Gavreto in people with RET fusion-positive NSCLC, RET-mutant MTC, and RET fusion-positive thyroid cancers, at multiple sites across the US, EU and Asia.

In the clinical trial, the drug demonstrated strong efficacy and was generally well-tolerated in patients with RET-altered thyroid cancers, regardless of previous systemic therapy.

The treatment using Gavreto resulted in 60% overall response rate (ORR) in 55 people with RET-mutant metastatic MTC previously treated with cabozantinib or vandetanib, and the median duration of response (DoR) was not reached.

The treatment showed 66% ORR in 29 people with cabozantinib- and vandetanib-naïve RET-mutant advanced MTC who were not eligible for standard therapies, and the median DoR was not reached.

Also, the ORR was 89% in nine patients with RET fusion-positive metastatic thyroid cancer, and the median DoR was not reached.

The most common adverse reactions in the ARROW trial include constipation, hypertension, fatigue, musculoskeletal pain and diarrhoea.

ARROW trial investigator Mimi Hu said: “Traditionally, we have treated patients with RET-altered thyroid cancers with multi-kinase inhibitors, non-selective therapies with modest efficacy and clinically significant side effects. The FDA approval of pralsetinib (GAVRETO), a once-daily RET-targeted therapy, advances the standard of care for these patients.

“As a clinical researcher with a focus on thyroid cancer, I am encouraged by the safety profile and durable responses shown by GAVRETO in RET-altered thyroid cancers in both treatment-naïve and previously treated patients.”