The FDA Approves IMKELDI, the First Oral Liquid Form of Imatinib, for Leukemia and Other Cancers

The U.S. Food and Drug Administration (FDA) has approved imatinib (IMKELDI), the first oral liquid tyrosine kinase inhibitor (TKI) for treating leukemia and a range of cancers, including CML, GIST, and Ph+ ALL. Developed by Shorla Oncology, this new liquid formulation aims to improve patient adherence by offering precise dosing and a more accessible treatment option.

A Patient-Friendly Solution

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

Broad Applications in Cancer Care

IMKELDI has been approved for various indications, including:

• Chronic Myeloid Leukemia (CML): Newly diagnosed adult and pediatric patients in the chronic phase, as well as those with blast crisis or advanced disease.
• Acute Lymphoblastic Leukemia (ALL): Adults with relapsed or refractory Philadelphia chromosome-positive ALL and pediatric patients in combination with chemotherapy.
• Gastrointestinal Stromal Tumors (GIST): Unresectable, recurrent, or metastatic cases, as well as post-surgical adjuvant treatment.
• Myelodysplastic/Myeloproliferative Disorders (MDS/MPD): Cases linked to platelet-derived growth factor receptor (PDGFR) gene rearrangements.
• Other Cancers: Aggressive systemic mastocytosis, hypereosinophilic syndrome, and chronic eosinophilic leukemia.

This liquid formulation is also approved for rare tumors, such as dermatofibrosarcoma protuberans, expanding its utility across diverse oncology indications.

A History of Innovation

Imatinib first gained FDA approval in 2001 for treating Philadelphia chromosome-positive CML and later for advanced or metastatic GIST. Over the years, it has become a cornerstone in targeted cancer therapy. However, challenges with treatment adherence and accessibility have persisted, particularly for vulnerable patient populations. IMKELDI addresses these gaps by providing a stable, easy-to-administer formulation that does not require refrigeration, ensuring broader usability in diverse healthcare settings.

“This milestone marks our fourth FDA approval as we advance our mission to make existing oncology treatments better through formulation re-innovation,” said Orlaith Ryan, chief technical officer and co-founder of Shorla.

Therapeutic Benefits and Risks

Imatinib targets the BCR-ABL genetic anomaly associated with the Philadelphia chromosome, a key driver in certain leukemias. As a monotherapy or in combination with chemotherapy, it has demonstrated efficacy in achieving complete hematologic response in various studies, particularly among older patients.

However, imatinib carries risks, including hematologic toxicities such as thrombocytopenia, neutropenia, and anemia, along with fluid retention and edema. These risks are heightened in patients over 65 or at higher doses. Liver, renal, and cardiovascular complications are also possible, requiring close monitoring.

Pediatric Applications and Limitations

Although IMKELDI is approved for children as young as one year old, its safety and efficacy in pediatric patients with Ph+ ALL have yet to be fully established. This limitation highlights the need for further research to expand treatment options for younger populations.

About Author: Tracy Mokwe

With over 3 years of experience as a medical laboratory scientist and writer, I am passionate about translating complex scientific information into clear and accessible content. My expertise lies in medical sciences, laboratory science and research, digital health, AI, and cybersecurity in healthcare.