FDA OKs Oral Nilotinib Tablets for Ph+ CML With Acid-Reducer Flexibility

The FDA has approved a new orally disintegrating tablet formulation of nilotinib (Cavhanza) for the treatment of adult patients with Philadelphia chromosome–positive chronic myeloid leukemia (Ph+ CML).¹ This formulation is specifically designed to maintain bioavailability when coadministered with proton pump inhibitors (PPIs) or histamine H₂ receptor antagonists (H₂RAs) without timing restrictions, allowing concomitant use.

The new formulation is specifically indicated for patients with newly diagnosed Ph+ CML in chronic phase, and patients with chronic and accelerated phase Ph+ CML who have resistance or intolerance to prior therapy, including imatinib (Gleevec).

“We’re excited to be able to offer the CML community another TKI option that may better suit their needs,” Victoria Dickinson, chief product officer at Cycle Pharmaceuticals, stated in a news release.¹ “Finding the right treatment that fits a patient’s needs and lifestyle can be a stressful step in a patient’s treatment journey.”

Mechanism of Action and Clinical Significance

The approval introduces an alternative formulation of a well-established BCR::ABL tyrosine kinase inhibitor (TKI) and addresses a common medication management challenge in patients requiring acid-suppressive therapy. Patients with CML frequently require concomitant medications for gastroesophageal reflux disease and other acid-related conditions, particularly given the older age of many individuals receiving long-term TKI therapy. Historically, acid-suppressive agents have presented management challenges with certain kinase inhibitors because of pH-dependent absorption.

Designed with Flex Pharma’s proprietary ElectroNanoSpray™ platform, the new formulation may enable greater flexibility for patients receiving PPIs or H₂RAs, potentially reducing the need for complex medication scheduling, and could simplify supportive care management in routine clinical practice.

In addition to compatibility with PPIs and H₂RAs, clinical trial data showed that the orally disintegrating formulation of nilotinib had no food effect and can be administered without regard to meals,¹ according to the sponsor. The lack of food-related restrictions may reduce treatment burden and provide greater dosing flexibility compared with formulations that require fasting. Further, because the agent is available as an orally disintegrating tablet, it may offer an alternative administration option for patients who have difficulty swallowing tablets and can be taken with or without water.

Safety Profile and Contraindications

The prescribing information for nilotinib includes a boxed warning regarding QT prolongation and sudden death. The label recommends ECG and electrolyte monitoring and advises against use in patients with hypokalemia, hypomagnesemia, or congenital long QT syndrome, as well as concomitant administration with QT-prolonging agents and strong CYP3A4 inhibitors.

Background on Nilotinib

Nilotinib (Tasigna) is a second-generation BCR::ABL TKI approved since 2007 for the treatment of Ph+ CML.² The agent has been used across multiple CML treatment settings and remains an established therapeutic option for patients with chronic-phase disease.

Prior regulatory filings for alternative formulations of nilotinib have sought to address limitations associated with administration requirements and food effects. Investigators noted that food-related restrictions can affect treatment adherence and administration convenience for patients receiving long-term therapy.

The approval of the oral tablet formulation thus represents the latest effort to optimize delivery of nilotinib while preserving therapeutic activity and expanding administration flexibility. The decision ultimately reflects a growing trend in oncology toward reformulated oral therapies intended to improve administration, adherence, and compatibility with commonly prescribed concomitant medications.

REFERENCES

1. CAVHANZA™ (nilotinib) Orally Disintegrating Tablets: A New, FDA-approved Treatment Offering PPI and H₂RA Flexibility for Ph+ CML Patients. News release. Cycle Pharmaceuticals. June 2, 2026. Accessed June 2, 2026. https://tinyurl.com/2vhwdvhb

2. Hazarika M, Jiang X, Liu Q, et al. Tasigna for chronic and accelerated phase Philadelphia chromosome--positive chronic myelogenous leukemia resistant to or intolerant of imatinib. Clin Cancer Res. 2008;14(17):5325-5331. doi:10.1158/1078-0432.CCR-08-0308