The U.S. Food and Drug Administration approved a liquid form of imatinib to treat some forms of aggressive systemic mastocytosis (SM) and several cancer types, Shorla Oncology announced recently. Shorla markets imatinib under the brand name Imkeldi.
The oral flavored liquid solution will contain 80 milligrams of imatinib per milliliter and does not require refrigeration, the company said. The manufacturer said it expected the liquid to improve dosing accuracy and enhance treatment adherence.
What are the types of SM?
There are five subtypes of SM: 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., the most common and least severe subtype; smoldering SMSmoldering SM A subtype of nonadvanced SM, with a higher mast cell burden than the indolent subtype. Smoldering SM has a higher likelihood of progressing to an advanced form., which can progress to more serious illness; aggressive SMAggressive SM A subtype of advanced SM marked by a high mast cell burden that leads to organ damage. Aggressive SM accounts for less than 10% of SM cases.; SM with an associated hematologic neoplasm; and mast cell leukemiaMast cell leukemia The most severe subtype of SM, caused by the rapid buildup of immature mast cells in the bone marrow and blood. Mast cell leukemia accounts for less than 5% of SM cases and has a poor prognosis., the rarest and most severe subtype.
“We are thrilled to offer an oral solution option for patients with leukemia and other cancers, a meaningful advancement for thousands in need,” Shorla CEO Sharon Cunningham said in a statement. “Oral solutions may ensure more precise and consistent dosing, offering a convenient alternative to compounding for patients who have difficulty swallowing or require dosing tailored to body surface area.”
Imatinib is a 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. that was initially approved for the treatment of chronic myeloid leukemia (CML) in 2001. In the context of SM, imatinib is relevant for the minority of patients with aggressive SM without the KIT D816V mutation or having unknown KIT mutational status.
Several studies demonstrated that patients with KIT D816V negative aggressive SM improved significantly under imatinib treatment, experiencing a decrease in serum tryptaseTryptase A protein enzyme that is primarily produced by mast cells and stored in small pockets within the cells, known as granules. High levels of tryptase are a key indicator of SM. levels, 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. concentration in the bone marrow and meaningful symptom relief.
The drug is also valuable in other conditions such as CML, acute lymphoblastic leukemia (ALL), hypereosinophilic syndrome, chronic eosinophilic leukemia (CES) and malignant gastrointestinal stromal tumors (GIST).
Common adverse effects of imatinib are mild and include nausea, vomiting, muscle cramps, fatigue, diarrhea and abdominal pain. More severe adverse effects observed in a minority of patients include cardiac dysfunction, liver damage, anemia, immunosuppression and gastrointestinal hemorrhage.
The new form of imatinib should be administered with the help of a syringe or a press-in bottle adapter to ensure optimal dosing. Experts recommended taking it together with food and water.
