FDA Approves Imatinib Oral Solution for Treatment of Various Cancers

Yesterday, Marks Shorla Oncology’s announced the FDA’s approval of imatinib (Imkeldi). With this decision, the agent becomes the first oral, liquid tyrosine kinase inhibitor (TKI) indicated for the treatment of various cancers including gastrointestinal tumors (GIST), myelodysplastic syndromes (MDS), or myeloproliferative disease (MPD), as well as chronic myeloid leukemia (CML) and Philadelphia chromosome–positive acute lymphoblastic leukemia (Ph+ ALL).¹

Imatinib word concept | image credit: lexiconimages - stock.adobe.com

“We are thrilled to offer an oral solution option for patients with leukemia and other cancers, a meaningful advancement for thousands in need,” Sharon Cunningham, CEO of Shorla, 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.”

Oral imatinib was designed to create a more accessible and patient-friendly treatment option and can be administered in patients as young as 1 year. With over 9000 people being diagnosed with CML in 2024, more than 10,000 with MDS or MPD, nearly 6000 with GIST, and upwards of 6500 expected to be diagnosed with ALL by the end of the year—leading to an anticipated 1300 or more deaths—the availability of oral imatinib is set to address a great need for these populations.^1,2

ALL diagnoses are not very common and make up less than 0.5% of all cancer diagnoses throughout the US. Males have a slightly higher risk for ALL than females, and children under 5 years old are thought to carry the greatest risk for ALL. While approximately 60% of ALL cases occur in children, adults tend to experience worse outcomes, accounting for nearly 80% of deaths in ALL.²

Imatinib may be prescribed as a monotherapy or a dual therapy in conjunction with forms of chemotherapy. To date, the drug has shown considerable efficacy as a first-line treatment in older patients, especially when combined with chemotherapy compared with chemotherapy on its own. Imatinib targets the BCR-ABL anomaly that occurs with the Ph chromosome and has led to patients achieving complete hematologic response in various studies.³

Currently, not even studies have been conducted to establish the safety and efficacy of imatinib in pediatric populations with Ph+ ALL.⁴

For patients pursuing this treatment, close monitoring remains important because imatinib may increase their risk for retaining serious fluid or developing edema. In some studies, researchers observed edema risks increase in patients 65 years or older and those with greater imatinib dosing. Other data reveal the risk for hematologic toxicity such as thrombocytopenia, neutropenia, or anemia, as well as the potential for certain liver, renal, or cardiovascular complications. For more information on adverse events, patient risks, and to review the list of drug interactions for imatinib, please visit here.

References

1. Shorla Oncology announces FDA approval of IMKELDI (imatinib) oral solution, an oral liquid for the treatment of certain forms of leukemia and other cancers. News release. Shorla Oncology. November 25, 2024. Accessed November 26, 2024. https://www.businesswire.com/news/home/20241125044117/en/Shorla-Oncology-Announces-FDA-Approval-of-IMKELDI-imatinib-Oral-Solution-an-Oral-Liquid-for-the-Treatment-of-Certain-Forms-of-Leukemia-and-Other-Cancers

2. Key statistics for acute lymphoblastic leukemia (ALL). American Cancer Society. Updated January 17, 2024. Accessed November 26, 2024. https://www.cancer.org/cancer/types/acute-lymphocytic-leukemia/about/key-statistics.html

3. Ravandi F. Managing Philadelphia chromosome-positive acute lymphoblastic leukemia: role of tyrosine kinase inhibitors. Clin Lymphoma Myeloma Leuk. 2011;11(2):198-203. doi:10.1016/j.clml.2011.03.002

4. Imatinib (oral route). Mayo Clinic. Updated February 1, 2024. Accessed November 26, 2024. https://www.mayoclinic.org/drugs-supplements/imatinib-oral-route/description/drg-20068331