FDA Approves Oral Solution of Imatinib for Certain Leukemias, Other Cancers

FDA

The FDA has approved an oral solution of imatinib (Imkeldi) for the treatment of patients with certain forms of leukemia and other types of cancer.¹

Imatinib (Gleevec) was initially approved by the FDA in 2001 and is indicated for use across a wide array of hematologic and solid tumor malignancies. The prescribing information for Imkeldi includes the following indications:^1,2

• Newly diagnosed adult and pediatric patients with Philadelphia chromosome (Ph)–positive chronic myeloid leukemia (CML) in chronic phase
• Patients with Ph-positive CML in blast crisis, accelerated phase, or chronic phase after failure of interferon-alpha therapy
• Adult patients with relapsed or refractory Ph-positive acute lymphoblastic leukemia (ALL)
• Pediatric patients with newly diagnosed Ph-positive ALL in combination with chemotherapy
• Adult patients with myelodysplastic/myeloproliferative diseases associated with PDGFR gene rearrangements
• Adult patients with aggressive systemic mastocytosis without c-KIT D816V mutations or with unknown c-KIT mutational status
• Adult patients with hypereosinophilic syndrome (HES) and/or chronic eosinophilic leukemia (CEL) who have the FIP1L1-PDGFRα fusion kinase (mutational analysis or fluorescence in situ hybridization [FISH] demonstration of CHIC2 allele deletion) and for patients with HES and/or CEL who are 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)
• Adjuvant treatment of adult patients following resection of KIT CD117–positive GIST

“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, chief executive officer of Shorla, stated in a news release.¹ “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.”

Imkeldi is an advanced liquid formulation of imatinib intended to provide improved dosing accuracy. The solution is flavored and has a stable formulation that does not require refrigeration, and it could improve treatment adherence and accessibility for patients.

“This milestone marks our fourth FDA approval as we advance our mission to make existing oncology treatments better through formulation re-innovation,” Orlaith Ryan, chief technical officer and co-founder of Shorla, added in a news release. “Our team is dedicated to creating more patient-friendly options that address real needs in those suffering from cancer.”

There are no contraindications listed for Imkeldi. The most common treatment-related adverse effects (TRAEs) reported with imatinib include edema, nausea/vomiting, muscle cramps, musculoskeletal pain, diarrhea, and rash. Edema primarily occurred around the eyes or in lower limbs; this TRAE was managed with diuretics and other supportive measures, or through dose reductions.

Notably, some AEs were tied to local or general fluid retention, including pleural effusion, ascites, pulmonary edema, and rapid weight gain with or without superficial edema. Fluid retention–related AEs may be dose-related, and they were more common in the blast crisis and accelerated phase studies at a dose of 600 mg per day. These AEs are also more common in older patients. Fluid retention–related AEs were managed by treatment interruptions, diuretics, or other supportive care.

“At Shorla, every innovation is driven by our commitment to put patients first,” Rayna Herman, chief commercial officer of Shorla, added in the news release. “Imkeldi is another step forward as we continue to expand our growing portfolio with products that prioritize accessibility and affordability.”

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 25, 2024. https://www.businesswire.com/news/home/20241125044117/en
2. Imkeldi. Prescribing information. Shorla Oncology. November 2024. Accessed November 25, 2024. https://shorlaoncology.com/pdf/Imkeldi_PrescribingInformation.pdf