Avapritinib treatment improves survival outcomes and symptom management in patients with systemic mastocytosis (SM), according Blueprint Medicines Corp., which presented new data at the American Society of Hematology Annual Meeting and Exposition. Blueprint makes avapritinib under the trade name Ayvakit.
Blueprint presented the results of several datasets, including a study that found that patients with advanced SM treated with avapritinib had significantly better survival outcomes than those treated with the best available real-world therapy.
What is SM?
Systemic mastocytosis (SM) is a rare hematological disease characterized by mast cells that are overactive and accumulate in different parts of the body such as the bone marrow, liver, spleen, gastrointestinal tract and lymph nodes.
Avapritinib was also associated with better symptom management and improved quality of life in those with less severe disease, the company said.
Company research also showed that treatment with avapritinib led to sustained improvements in bone density for advanced SM patients who had low bone mass at baseline.
Avapritinib “has fundamentally changed the treatment paradigm by targeting the disease at its source, showing prolonged survival outcomes for patients with advanced SM, as well as durable symptom control and quality-of-life improvements for patients with ISM [indolent SM],” said Becker Hewes, Blueprint’s chief medical officer, in a statement.
SM is a rare and progressive blood disorder in which the body produces too many mast cells (a type of immune cell). The excess mast cells build up in the body and can affect multiple organs, including the skin, liver, spleen, bone marrow and intestines.
The severity of the disease ranges from nonadvanced forms such as indolent SM to advanced SM. The latter is typically associated with poor prognosis and decreased survival rate because of resulting organ damage.
The U.S. Food and Drug Administration approved avapritinib for treatment of both indolent and advanced SM. The therapy targets the KIT D816V mutation, which is the main driver of the majority of SM cases.
Based on the datasets Blueprint gathered over the last decade, the company also announced a new trial for elenestinib, a next-generation KIT D816V inhibitor, which will “rigorously assess a broad range of endpoints reflecting disease-modifying impact,” Hewes said.
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