A new expanded access program (EAP) has been launched for qualifying patients with systemic mastocytosis (SM), allowing patients whose disease is not adequately controlled with current therapies to receive the investigational drug bezuclastinib. Details on the program were recently posted on clinicaltrials.gov.
Bezuclastinib is a selective tyrosine kinase inhibitorTyrosine kinase inhibitor Targeted therapies designed to block the KIT D816V mutation that drives most cases of SM. Includes avapritinib, midostaurin and imatinib., targeting specific mutations in the KIT gene, particularly the KIT D816V mutation that is commonly found in patients with SM. It is currently being tested in clinical trials but has not yet been approved for routine medical use.
Although early trial results are promising, showing that bezuclastinib can lower mast cellMast cell A type of white blood cell produced in the bone marrow. They help defend against infections and play a key role in allergic reactions. In SM, mast cells become overactive and build up throughout the body. counts and improve symptoms with relatively mild side effects, it is still considered experimental. Current treatment guidelines recommend approved drugs, such as avapritinib, midostaurin, cladribine and peginterferon alfa-2a.
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The new EAP is meant for adult patients who have either not responded to existing treatments or cannot tolerate them, and who do not qualify for ongoing clinical trials. Eligibility criteria includes being over 18 years old, having a confirmed diagnosis of SM, and being able to swallow tablets. Patients previously removed from bezuclastinib studies due to side effects or personal choice are not eligible.
Both patients with non-advanced forms of SM (such as 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.) and 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. subtypes can qualify for the program.
While bezuclastinib is not yet part of standard treatment guidelines, this EAP offers a therapeutic alternative to patients with no other remaining options.
“The purpose of this expanded access program (EAP) protocol is to provide investigational bezuclastinib to patients with a diagnosis of nonadvanced 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. (NonAdvSM) or advanced systemic mastocytosis (AdvSMAdvanced 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.) who have received and failed or been intolerant to at least one standard approved therapy and/or have no comparable or satisfactory alternative therapy options,” the authors wrote.
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