28 October 2019 - Given proximity of NDA action date to anticipated top-line data readout for Phase 3 VOYAGER trial, FDA requested VOYAGER top-line data to inform its review of fourth-line GIST indication.

Blueprint Medicines today announced an update regarding its new drug application for avapritinib for the treatment of adults with PDGFRA Exon 18 mutant GIST, regardless of prior therapy, and fourth-line GIST.

On October 25, 2019, Blueprint Medicines received written feedback from the U.S. FDA following its NDA mid-cycle review meeting. The FDA informed Blueprint Medicines that it intends to administratively split the proposed indications for avapritinib into two separate NDAs, one for PDGFRA Exon 18 mutant GIST, regardless of prior therapy, and one for fourth-line GIST. Given the acceleration of the ongoing Phase 3 VOYAGER clinical trial of avapritinib in patients with third- and fourth-line GIST and the anticipated availability of top-line data in the second quarter of 2020, the FDA requested top-line data from the VOYAGER trial. The FDA indicated these data would be informative in its review of the proposed fourth-line indication and potential clinical benefit in this population, including its evaluation of response rate and safety for the fourth-line indication. An extension of the review period for the fourth-line GIST NDA will likely be required to enable Blueprint Medicines to provide the top-line VOYAGER data to the FDA.

Read Blueprint Medicines press release