Blueprint Medicines (BPMC) has announced that the European Commission (EC) has granted conditional marketing authorization to AYVAKYT (avapritinib) as a monotherapy to treat adults with unresectable or metastatic gastrointestinal stromal tumors (GIST) harboring the platelet-derived growth factor receptor alpha (PDGFRA) D842V mutation.
For the first time in Europe, there is an approved therapy for patients with PDGFRA D842V mutant GIST specifically designed to target the underlying molecular driver of their disease, the company says.
In trials, AYVAKYT demonstrated deep and durable clinical activity and was generally well-tolerated in patients. In the NAVIGATOR trial, AYVAKYT had an overall response rate of 95%, with 13% of patients achieving a complete response, and the median duration of response was 22.1 months. The median progression-free survival was 24 months, and the median overall survival was not reached.
GIST is a genomically driven sarcoma of the gastrointestinal (GI) tract, with PDGFRA D842V mutations implicated in a rare subset of patients.
“There have historically been no treatments offering hope for patients with PDGFRA D842V mutant GIST. AYVAKYT represents the first major therapeutic breakthrough for patients with GIST harboring this mutation, defining a new standard of care,” said Sebastian Bauer, M.D., an investigator on the NAVIGATOR trial.
“The NAVIGATOR trial confirmed that almost all patients with PDGFRA D842V mutant GIST achieved tumor shrinkage and clinical responses were durable. These patients have lived longer than what is expected based on historical outcomes, and side effects have been well-tolerated in most patients” he added.
In Europe, Blueprint Medicines plans to initiate its first commercial launch in Germany following the EC approval, and the timing of AYVAKYT availability will vary for other countries based on local reimbursement and access pathways.
As part of the conditional marketing authorization, Blueprint Medicines plans to conduct an observational, long-term study in patients with PDGFRA D842V mutant GIST treated with AYVAKYT.
Shares in Blueprint are up 10% year-to-date, and the stock boasts a bullish Strong Buy Street consensus. That’s with 13 recent buy ratings and just 1 hold rating. Meanwhile the average analyst price target of $103 indicates 17% upside potential from current levels.
“We continue to view Blueprint’s novel, rationally designed, targeted kinase inhibitor platform as attractive and reiterate our buy rating” cheered Canaccord Genuity analyst Arlinda Lee earlier this month.
She reiterated her $100 BPMC price target after the US FDA approved Blueprint/ Genentech’s Gavreto (pralsetinib) for RET+ non-small cell lung cancer (NSCLC). “Gavreto is the second of BPMC’s novel drugs to garner approval in 2020, and notably 3.5 years since the first patient was dosed” she noted. (See Blueprint stock analysis on TipRanks).