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 SMAdvanced SM In these subtypes of SM, mast cells begin to damage organs. Advanced SM includes the subtypes aggressive SM, mast cell leukemia and SM with an associated hematological neoplasm. treated with avapritinib had significantly better survival outcomes than those treated with the best available real-world therapy.
What is SM?
Systemic mastocytosisMastocytosis Rare disease caused by the buildup of mast cells. Cutaneous mastocytosis primarily affects the skin and is more common in children, while systemic mastocytosis affects internal organs and is more common in adults. (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 SMIndolent SM A subtype of nonadvanced SM caused by the abnormal accumulation of mast cells in the bone marrow and other organs. Indolent SM accounts for around 90% of SM cases.],” 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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