The FDA granted the application priority review with an action date of May 22, 2023. The FDA recently accepted the company's supplemental new drug application for Ayvakit for the treatment of adults with indolent SM. In the past year, shares of Blueprint have lost 47.1% compared with the industry’s decline of 8.6%.īlueprint Medicines’ lead drug, Ayvakit (avapritinib), is a kinase inhibitor approved by the FDA for the treatment of adults with advanced systemic mastocytosis (SM), including aggressive SM (ASM), SM with an associated hematological neoplasm (SM-AHN) and mast cell leukemia (MCL), and adults with an unresectable or metastatic gastrointestinal stromal tumor (GIST) harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations. The company targets to present its initial dose escalation data from the VELA trial of BLU-222 in the first half of 2023. The company has stated that patients have been treated with BLU-222 at doses ranging from 50 mg BID to 800 mg BID to date, with evidence of clinical benefit observed and no discontinuations due to AEs.īlueprint Medicines will look to amend the VELA trial protocol to provide specific guidance to investigators on how to monitor for and manage these events, should they occur. The company claims to have resolved all events involving dose interruption or reduction. All events were Grade 1, except one Grade 3 event involving light sensitivity and blurred vision in a patient treated at 600 mg BID. The reported visual AEs consisted of transient, reversible episodes of light sensitivity and blurred vision. Patients currently enrolled in the trial are continuing to take the study drug at this time, but additional patients will not be enrolled until the partial clinical hold is resolved. The partial clinical hold was placed due to visual adverse events (AEs) observed in a limited number of patients. The regulatory body informed the company about the hold in a verbal communication.
0 Comments
Leave a Reply. |
AuthorWrite something about yourself. No need to be fancy, just an overview. ArchivesCategories |