Avapritinib for unresectable or metastatic gastrointestinal stromal tumours (GIST) who have been treated with at least 3 prior lines of therapy and GIST harbouring the PDGFRA D842V mutation regardless of prior therapy

Avapritinib is currently in clinical development for the treatment of unresectable or metastatic gastrointestinal stromal tumours (GIST). GIST is a rare type of cancer that commonly develops in the gastrointestinal tract. The cause of GIST is unknown but the majority of cases are associated with a certain gene mutations, most commonly in the KIT or PDGFRA D842V genes. GIST with these mutations are very aggressive, can spread quickly to other parts of the body (metastatic) and are not easily removed surgically (unresectable). There are currently no approved therapies for patients with GIST who have exhausted treatment options with approved first, second, and third line agents. In addition, no treatment options are approved specifically for patients with PDGRFA D842V–mutated GIST, which is resistant to all currently approved therapies.
Avapritinib is designed to potently and selectively inhibit the active forms of the KIT and PDGFRA mutant enzymes, and has shown potent activity in GIST driven by these gene mutations. If licensed, avapritinib will offer a fourth-line treatment option for patients with unresectable or metastatic GIST who have exhausted all other approved treatment options. It will also be the first licensed treatment option specifically for patients with unresectable or metastatic GIST harbouring a PDGRFA D842V mutation.