FDA Approves First Oral Liquid Imatinib for Leukemia and Other Cancers

Molecular structure of cancer cells under microscope, realistic macro indoor part of body.: © annamaria - stock.adobe.com

• The FDA has approved imatinib oral solution (Imkeldi) for the treatment of certain forms of leukemia and other cancers.
• This is the first oral liquid form of imatinib for the treatment of select cancers.
• This advanced liquid formulation of imatinib was made to assist with dosing accuracy.

The FDA has approved the first oral liquid form of imatinib to treat patients with certain forms of leukemia and other cancers.¹

In 2001, imatinib (Gleevec), a tyrosine kinase inhibitor, was approved for the treatment of patients with Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML). The agent was then granted approval from the FDA for the treatment of advanced or metastatic gastrointestinal stromal tumors (GIST) in 2002, followed by its accelerated approval for adjuvant use in patients with resectable GIST in 2008. Full approval from the FDA was granted to the agent as an adjuvant treatment following surgical removal of CD117-positive GIST in adult patients in 2012.²

This oral liquid form of imatinib aims to provide dosing accuracy and can help slow or prevent the growth of specific cancers, including CML, acute lymphoblastic leukemia (ALL), myelodysplastic syndrome /myeloproliferative disease (MDS/MPD), and GIST.¹

“We are thrilled to offer an oral solution option for patients with leukemia and other cancers, [which is] a meaningful advancement for thousands in need,” said Sharon Cunningham, chief executive officer of Shorla, in a press 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.”

The oral solution of imatinib features a convenient and palatable strawberry flavor with a stable formulation that does not require refrigeration. This patient-friendly option enables precise dosing, treatment adherence, and accessibility.

The agent has been approved for several indications, including for the treatment of adult and pediatric patients newly diagnosed with Ph+ CML in the chronic phase, as well as for patients with Ph+ CML in blast crisis, accelerated phase, or chronic phase following interferon-alpha therapy failure. It is also approved for adult patients with relapsed or refractory Ph+ ALL and for pediatric patients with newly diagnosed Ph+ ALL in combination with chemotherapy.

Additionally, the formulation is indicated for adult patients with MDS/MPD linked to platelet-derived growth factor receptor (PDGFR) gene rearrangements, aggressive systemic mastocytosis without the D816V c-Kit mutation or with unknown c-Kit status, and hypereosinophilic syndrome or chronic eosinophilic leukemia associated with FIP1L1-PDGFRα fusion kinase or with unknown kinase status.

Further, this oral formulation of imatinib is approved for treating unresectable, recurrent, or metastatic dermatofibrosarcoma protuberans and Kit (CD117)-positive GIST in unresectable or metastatic cases, as well as for adjuvant treatment following resection of CD117-positive GIST.

“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, in the press release. “Our team is dedicated to creating more patient-friendly options that address [the] real needs of those suffering from cancer.”

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

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://tinyurl.com/bdh22bmv

2. Gleevec FDA approval history. Drugs. Updated February 25, 2021. Accessed November 25, 2024. https://www.drugs.com/history/gleevec.html