Roche’s Genentech unit and Blueprint Medicines have received full approval from the US Food and Drug Administration (FDA) for use of their targeted cancer therapy, Gavreto (pralsetinib), in a subset of non-small cell lung cancer (NSCLC) patients.
The drug was authorised for the NSCLC indication in 2020 through the FDA’s accelerated approval pathway, under which the regulator may approve drugs for serious conditions where there is an unmet medical need earlier.
This was based on initial overall response rate (ORR) and duration of response (DOR) data from 114 patients enrolled in the phase 1/2 ARROW trial.
The FDA’s latest decision, which specifically applies to adults with metastatic rearranged during transfection (RET) fusion-positive NSCLC, was supported by data from an additional 123 patients and 25 months of extra follow-up to assess durability of response.
Among 107 treatment-naïve patients, ORR was 78%, with a median DOR of 13.4 months. For 130 patients previously treated with platinum-based chemotherapy, ORR was 63%, with a median DOR of 38.8 months.
An estimated 2.2 million people worldwide are diagnosed with lung cancer each year, and NSCLC represents up to 85% of all lung cancer diagnoses.
Gavreto is an oral tyrosine kinase inhibitor designed to selectively and potently inhibit RET alterations, which are present in just 1% to 2% of NSCLC patients.
The therapy was also granted accelerated approval by the FDA in 2020 to treat patients aged 12 years and older with advanced or metastatic RET-mutant medullary thyroid cancer who require systemic therapy. Genentech, however, recently decided to withdraw Gavreto from use in the US for this indication.
Blueprint said Genentech will not pursue the late-stage study required to confirm the drug’s benefits due to its feasibility following consultation with the FDA.
According to Blueprint, Genentech previously said the use of the drug in this indication accounted for a fraction of its overall revenue in the US.
Gavreto was jointly developed and commercialised by Genentech and Blueprint in the US through a deal in 2020. Roche decided to terminate the deal earlier this year, with the termination set to be effective in February 2024.