FDA Approves Oral Liquid Solution of Imatinib for Certain Forms of Leukemia and Other Cancers

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The FDA approved an oral solution of imatinib (Imkeldi; Shorla Oncology) for the treatment of certain forms of leukemia as well as other cancers. This approval marks the first oral liquid form of imatinib to be approved by the FDA to treat cancers.¹

This oral liquid formulation of imatinib is designed to provide dosing accuracy. Imatinib can help slow or prevent the growth of specific cancers. The solution is a flavored and stable formulation that does not require refrigeration. Experts note that the oral formulation may improve treatment adherence and accessibility for patients. Further, each milliliter of the oral formulation contains the equivalent to 80 mg of imatinib.^1,2

Initially approved by the FDA in 2001 for the treatment of chronic myeloid leukemia (CML) in the chronic phase (CP) following the failure of interferon-alpha (INF-alpha) therapy, the kinase inhibitor imatinib (Gleevec; Novartis). Currently, it has indications in the following disease states^1,2:

• newly diagnosed adult and pediatric patients with Philadelphia chromosome positive (Ph+) CML in blast crisis, accelerated phase, or in CP following failure of INF-alpha therapy
• Adult patients with relapsed or refractory Ph+ acute lymphoblastic leukemia (ALL); pediatric patients with newly diagnosed Ph+ ALL in combination with chemotherapy
• Adult patients with myelodysplastic/myeloproliferative diseases associated with platelet-derived growth factor receptor gene rearrangements; adult patients with aggressive systemic mastocytosis without the D816V c-Kit mutation or with c-Kit mutational status unknown
• Adult patients with hypereosinophilic syndrome (HES) and/or chronic eosinophilic leukemia (CEL) who have the FIP1L1-PDGFRα fusion kinase, negative, or unknown
• Adult patients with unresectable, recurrent and/or metastatic dermatofibrosarcoma protuberans
• Patients with Kit (CD117) positive unresectable and/or metastatic malignant gastrointestinal stromal tumors (GIST)
• For adjuvant treatment of adult patients following resection of Kit (CD117) positive GIST

The manufacturer recommends that all doses of imatinib should be taken with food and water. Doses of 400 mg or 600 mg are recommended to be administered once per day, but 800-mg doses should be administered as 400-mg doses twice per day. Additionally, they emphasize that imatinib must be measured accurately, such as with a press-in bottle adapter and oral dispensing syringe that can be obtained by a pharmacist.²

The most frequently reported adverse events (AEs) include edema, nausea, vomiting, muscle cramps, musculoskeletal pain, diarrhea, rash, fatigue, and abdominal pain. More severe AEs may include hematologic toxicities (eg, anemia, neutropenia, and thrombocytopenia), congestive heart failure and left ventricular dysfunction, hepatotoxicity, hemorrhage, gastrointestinal perforations, dermatologic toxicities (eg, erythema multiforme and Stevens-Johnson syndrome), among others.²

“We are thrilled to offer an oral solution option for patients with leukemia and other cancers, a meaningful advancement for thousands in need,” said Sharon Cunningham, CEO of Shorla in a news release. “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.”¹

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. Businesswire. News release. November 25, 2024. Accessed November 25, 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. Shora Oncology. Highlights of prescribing information. Accessed November 25, 2024. https://shorlaoncology.com/pdf/Imkeldi_PrescribingInformation.pdf