US precision therapy company Blueprint Medicines said that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has adopted a positive opinion for avapritinib as a monotherapy for certain types of gastrointestinal stromal tumors (GIST).
CHMP has recommended conditional marketing authorization of the kinase inhibitor in the European Union for the treatment of unresectable or metastatic gastrointestinal stromal tumors harboring the PDGFRA D842V mutation in adult patients.
The committee’s opinion will now be considered by the European Commission, which is likely to take a final decision on the marketing authorization application for avapritinib by the end of September 2020.
The positive opinion adopted by CHMP was based on the efficacy results from the phase 1 NAVIGATOR clinical trial and also the combined safety results from the NAVIGATOR and phase 3 VOYAGER clinical trials. Treatment with avapritinib in the clinical trials showed deep and durable clinical responses apart from being well-tolerated in patients having PDGFRA D842V mutant gastrointestinal stromal tumors.
Avapritinib, if approved by the European Commission, will become the first treatment in the European Union indicated for patients having PDGFRA D842V mutant gastrointestinal stromal tumors and will be commercialized under the AYVAKYT brand.
Andy Boral – Chief Medical Officer at Blueprint Medicines said: “Avapritinib has shown unprecedented clinical activity in patients with PDGFRA D842V mutant GIST, who have traditionally had poor prognoses.
“Today’s positive CHMP opinion reflects important progress toward our goal of making this highly effective treatment option available in the EU. For patients with PDGFRA D842V mutant GIST, avapritinib is designed to fundamentally change the treatment paradigm by selectively inhibiting an oncogenic driver shown to be resistant to existing GIST therapies.”
The kinase inhibitor is approved in the US under the AYVAKIT brand for the treatment of adults having unresectable or metastatic gastrointestinal stromal tumors harboring a PDGFRA exon 18 mutation, which includes the PDGFRA D842V mutations.
Recently, Blueprint Medicines and Swiss pharma giant Roche signed a worldwide licensing and collaboration deal potentially worth up to $1.703 billion for the former’s cancer drug candidate pralsetinib.