The FDA has granted approval to Imkeldi (imatinib) oral solution, marking the first oral liquid formulation of imatinib for treating specific forms of leukemia and other cancers. Developed by Shorla Oncology, this new formulation aims to improve dosing accuracy and patient adherence, particularly for those who have difficulty swallowing or require tailored dosing based on body surface area. The approval signifies a meaningful advancement in providing accessible and patient-friendly treatment options.
Clinical Significance
Imkeldi is indicated for use in both adult and pediatric patients, offering a versatile treatment option across various age groups. The drug is approved for several conditions, including:
- Newly diagnosed Philadelphia chromosome-positive (Ph+) chronic myeloid leukemia (CML) in chronic phase.
- CML in blast crisis, accelerated phase, or chronic phase after failure of interferon-alpha therapy.
- Relapsed or refractory Ph+ acute lymphoblastic leukemia (ALL) in adults.
- Newly diagnosed Ph+ ALL in pediatric patients, in combination with chemotherapy.
- Myelodysplastic/myeloproliferative diseases (MDS/MPD) associated with platelet-derived growth factor receptor (PDGFR) gene rearrangements in adults.
- Aggressive systemic mastocytosis (ASM) without the D816V c-Kit mutation or with c-Kit mutational status unknown in adults.
- Hypereosinophilic syndrome (HES) and/or chronic eosinophilic leukemia (CEL) with or without the FIP1L1-PDGFRα fusion kinase in adults.
- Unresectable, recurrent, and/or metastatic dermatofibrosarcoma protuberans (DFSP) in adults.
- Kit (CD117)-positive unresectable and/or metastatic malignant gastrointestinal stromal tumors (GIST).
- Adjuvant treatment following resection of Kit (CD117)-positive GIST in adults.
Formulation and Benefits
Imkeldi features a convenient, palatable strawberry flavor and a stable formulation that does not require refrigeration. This addresses a critical unmet need for a more accessible, patient-friendly oral solution, as highlighted by Shorla Oncology. Sharon Cunningham, CEO of Shorla, noted that oral solutions ensure more precise and consistent dosing, offering an alternative to compounding for patients with swallowing difficulties or those needing body surface area-tailored doses.
Market and Unmet Needs
In 2024, approximately 9,280 individuals are expected to be diagnosed with CML, over 10,000 with MDS/MPD, and up to 6,000 with GIST in the U.S. Despite the proven clinical benefits of imatinib, patient adherence remains a challenge. The availability of Imkeldi aims to mitigate this issue by providing a more convenient and accessible treatment option.
Safety Profile
Common adverse reactions associated with imatinib include edema, nausea, vomiting, muscle cramps, musculoskeletal pain, diarrhea, and rash. Patients should be monitored for fluid retention, hematologic toxicities, and potential cardiac, hepatic, and renal complications. The prescribing information advises caution when co-administering Imkeldi with strong CYP3A4 inhibitors or inducers and includes warnings regarding embryo-fetal toxicity and potential growth retardation in children.
Company Statements
Orlaith Ryan, CTO and co-founder of Shorla Oncology, emphasized that this approval marks their fourth FDA approval, underscoring their commitment to improving existing oncology treatments through formulation re-innovation. Rayna Herman, CCO of Shorla, added that Imkeldi is another step forward in expanding their portfolio with products that prioritize accessibility and affordability, driven by a commitment to putting patients first.
