Blueprint Medicines Corporation announced AYVAKIT data showing high, durable response rates and prolonged overall survival (OS) in patients with advanced systemic mastocytosis (Advanced SM), including SM with an associated hematological neoplasm (SM-AHN). The findings are being reported in two presentations, including an oral presentation featuring updated follow-up from treatment-naïve patients in the PATHFINDER trial, at the 64th American Society of Hematology (ASH) Annual Meeting and Exposition in New Orleans. AYVAKIT is approved by the U.S. Food and Drug Administration (FDA) for the treatment of adults with Advanced SM.
SM is a rare hematologic disorder driven by the KIT D816V mutation in approximately 95 percent of cases. In advanced SM, the median OS has ranged from less than six months to approximately 3.5 years, depending on the subtype.1 AYVAKIT was designed to potently and selectively inhibit D816V mutant KIT.
Also Read: First SyncAR for Spine Surgery in the World Performed at Hoag
“AYVAKIT has demonstrated significant clinical benefits in patients with advanced systemic mastocytosis, including complete remissions and durable responses that have translated into a highly meaningful impact on overall survival,” said Deepti Radia, M.D., a hematologist and an investigator on the PATHFINDER and EXPLORER trials. “As a physician specializing in SM and other myeloproliferative neoplasms, I am encouraged that treatment-naïve patients with SM-AHN had a 95 percent response rate and an estimated two-year survival rate of 86 percent in the PATHFINDER trial. Before the introduction of KIT D816V-targeted therapy, physicians often focused on treating the AHN component of SM-AHN. AYVAKIT represents a practice-changing advancement for this patient population, and updated PATHFINDER data highlight the importance of treating the SM for early therapeutic intervention in SM-AHN patients with measurable ‘C’ findings, excluding those with aggressive AHNs.”
“AYVAKIT has become the standard of care treatment for advanced systemic mastocytosis in the U.S., with a growing number of patients receiving the therapy early in the course of their disease. This position is further reinforced by updated AYVAKIT results in the treatment-naïve setting, which include overall response, complete remission and survival rates that establish a new benchmark for patients with advanced SM,” said Becker Hewes, M.D., Chief Medical Officer at Blueprint Medicines. “Through our long-term partnership with the SM community, we have amassed datasets reflecting hundreds of patient years of clinical experience across advanced and non-advanced forms of the disease. This breadth of data highlights strong clinical execution and reflects our leadership in developing a portfolio of targeted treatment options designed to address the medical needs of patients with SM.”
SOURCE: PR Newswire