CAVHANZA™ (nilotinib) Orally Disintegrating Tablets: A New, FDA-approved Treatment Offering PPI and H2RA Flexibility for Ph+ CML Patients1

CAMBRIDGE, England--(BUSINESS WIRE)--Cycle Pharmaceuticals (Cycle) announces the FDA approval of CAVHANZA (nilotinib) Orally Disintegrating Tablets. Launching soon, the CAVHANZA formulation is specifically designed to improve solubility and dissolution rate, enabling maintained bioavailability with concomitant use of acid-reducing agents such as proton pump inhibitors (PPIs) and/or H₂ antagonists (H₂RAs) without timing restrictions.¹

This will be a first in the nilotinib market, addressing a known challenge in Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML) treatment, where 25% of patients are co-prescribed PPIs or H₂RAs – medications that can compromise treatment efficacy.³ In clinical trials, CAVHANZA demonstrated no food effect and can be taken without regard to meals, reducing food-related burden and supporting more flexible dosing, potentially improving treatment outcomes.^1,2,4 Additionally, as an orally disintegrating tablet, CAVHANZA may support patients who have difficulty swallowing while also offering flexibility to take the treatment with or without water.¹

CAVHANZA is indicated for the treatment of:

• Newly diagnosed adults with Ph+ CML in chronic phase; and¹
• Adults with chronic and accelerated phase Ph+ CML with resistance or intolerance to prior therapy, including imatinib.¹

“We’re excited to be able to offer the CML community another TKI option that may better suit their needs,” said Victoria Dickinson, Chief Product Officer at Cycle. “Finding the right treatment that fits a patient’s needs and lifestyle can be a stressful step in a patient’s treatment journey. Bringing another option to market – backed by Cycle Vita^TM, our dedicated patient support* program – is an exciting prospect for Cycle.”

To prepare CAVHANZA for market, Cycle partnered with Flex Pharma (Flex), who specialize in advanced formulation solutions and has developed an improved formulation of the second-generation TKI – built on years of established nilotinib efficacy.

Christian Wertz, PhD, President of Flex, added “This collaboration reflects Flex’s strategy as a technology-driven company advancing differentiated 505(b)(2) oncology therapies. Leveraging our proprietary ElectroNanoSpray™ (ENS) platform, we reformulate established oncology molecules to enhance bioavailability and optimize dosing performance. Together with Cycle’s rare-disease commercialization expertise, we aim to efficiently deliver improved treatment options for patients with rare leukemias.”

Cycle has been delivering patient-focused treatments since 2017. The approval, and upcoming launch, of CAVHANZA represents the 10th product in Cycle's portfolio and the 2nd product to enhance treatment options for patients living with Ph+ CML.

Learn more about CAVHANZA at https://cyclepharma.com/chronic-myeloid-leukemia-new-treatment.

Warning: QT Prolongation and Sudden Deaths

Nilotinib prolongs the QT interval. Monitor for hypokalemia or hypomagnesemia and correct deficiencies. Obtain ECGs to monitor the QTc at baseline on day 7, and periodically thereafter, and following any dose adjustments.

Sudden deaths have been reported. Avoid use in patients with hypokalemia, hypomagnesemia or long QT syndrome.

Avoid concomitant drug use with QT-prolonging drugs and strong CYP3A4 inhibitors.

Indications

CAVHANZA (nilotinib) is a kinase inhibitor indicated for the treatment of:

Adult patients with newly diagnosed Philadelphia chromosome positive chronic myeloid leukemia (Ph+ CML) in chronic phase (CP).

Adults with CP Ph+ CML or accelerated phase (AP) Ph+ CML resistant to or intolerant to prior therapy.

Contraindications

CAVHANZA is contraindicated in patients with hypokalemia, hypomagnesemia, or long QT syndrome.

Important Safety Information

Warnings and Precautions:

• Substitution between nilotinib products requires dose conversion to avoid medication errors.
• Monitor for myelosuppression; manage with dose adjustments or interruptions.
• Cardiovascular and arterial occlusive events have been reported; assess and monitor cardiovascular risk.
• Pancreatitis risk: monitor serum lipase; interrupt dosing if abdominal symptoms occur with lipase elevations.
• Hepatotoxicity has been reported, with higher risk in pediatrics. Monitor liver function tests regularly.
• Correct and monitor electrolyte abnormalities throughout treatment.
• Tumor lysis syndrome has been reported; ensure hydration and correct uric acid before initiating treatment.
• Serious hemorrhage may occur; advise patients to report signs and symptoms of bleeding.
• Monitor for fluid retention and manage accordingly.
• Growth retardation has been reported in pediatric patients. Monitor growth and development.
• Can cause fetal harm; advise use of effective contraception during and 14 days after treatment.
• Monitor BCR-ABL transcript levels in patients discontinuing treatment and manage loss of response appropriately.

Adverse Reactions:

Common non-hematologic adverse reactions include rash, pruritus, headache, nausea, fatigue, alopecia, myalgia, abdominal pain, constipation, diarrhea and vomiting. Hematologic adverse reactions include thrombocytopenia, neutropenia, and anemia. Serious adverse reactions include QT prolongation, sudden deaths, febrile neutropenia, and intracranial hemorrhage. Postmarketing reports include thrombotic microangiopathy, facial paralysis, and osteonecrosis.

Drug Interactions:

Avoid concomitant use with strong CYP3A inhibitors or inducers. Dose reduction is recommended if strong CYP3A inhibitors cannot be avoided. Avoid drugs that prolong the QT interval, including anti-arrhythmics.

Use in Specific Populations:

Pregnancy: CAVHANZA can cause fetal harm when administered to a pregnant woman.

Lactation: Breastfeeding is not recommended during treatment with CAVHANZA and for 14 days after the last dose.

Pediatric use: CAVHANZA is not approved for use in pediatric patients. Monitor growth and development if used.

Due to Novartis Pharmaceuticals Corporation’s marketing exclusivity rights, this drug product is not labeled with that pediatric information.

Geriatric use: No major differences for safety were observed in patients ≥65 years taking CAVHANZA.

For more detailed information, please refer to the full Prescribing Information at https://cyclepharma.com/nilotinib-pi/

To report SUSPECTED ADVERSE REACTIONS, contact Cycle Pharmaceuticals at 1-855-831-5413, or the FDA at: 1-800-FDA-1088 or www.fda.gov/medwatch.

About Cycle Pharmaceuticals

Cycle Pharmaceuticals was founded in 2012 with the sole aim of delivering drug treatments and product support to the underserved rare disease community. Cycle focuses on rare genetic conditions in metabolic, immunology, urology, and oncology. In neurology, we focus on multiple sclerosis. Cycle is headquartered in Cambridge, UK. For more information, please visit www.cyclepharma.com and follow us on X, LinkedIn and Facebook.

About Flex Pharma

Flex Pharma is a specialty pharmaceutical company focused on transforming how complex oncology drugs are formulated and delivered. Flex develops next-generation oncology formulations that enhance drug performance using its proprietary ElectroNanoSpray™ (ENS) platform technology, a non-thermal spray drying process that uses electric-field forces to create fine, uniform, and amorphous particles. Flex is located in New Brighton, Minnesota. For more information, please visit www.flexpharmausa.com and follow on LinkedIn.

References:

1. CAVHANZA™ (nilotinib) Orally Disintegrating Tablets. Prescribing Information. Flex Pharma.
2. Data on file: REF-00096
3. Data on file: REF-00097
4. Boons, C.C.L.M., Timmers, L., Janssen, J.J.W.M. et al. Response and Adherence to Nilotinib in Daily practice (RAND study): an in-depth observational study of chronic myeloid leukemia patients treated with nilotinib. Eur J Clin Pharmacol 76, 1213–1226 (2020). https://doi.org/10.1007/s00228-020-02910-3 [Accessed March 26 2026].

*Some areas of support may not be accessible to all patients. Eligibility criteria may apply to ensure compliance with all applicable federal and state requirements, and benefits may be limited to commercially insured patients only. For more detailed information about eligibility, terms and conditions, please contact the Cycle Vita team at 888-360-8482.

© 2026 Cycle Pharmaceuticals Limited. All rights reserved.

Cycle Vita™ is a trademark of Cycle Pharmaceuticals Limited in the United States.

© Flex Pharma 2026. All rights reserved

US-NIL-2600029. April 2026.