The U.S. Food and Drug Administration has approved a new formulation of the leukemia drug nilotinib that could make treatment significantly easier for thousands of patients living with Philadelphia chromosome positive chronic myeloid leukemia (Ph+ CML). The approval of CAVHANZA, an orally disintegrating tablet, addresses a longstanding challenge in CML care by allowing patients to take the medication alongside common heartburn and acid reflux drugs without timing restrictions.
Approximately one in four CML patients also uses proton pump inhibitors (PPIs) or H2 receptor antagonists (H2RAs) for gastrointestinal issues. Standard nilotinib tablets can lose effectiveness when combined with these acid reducing agents, forcing patients to carefully time their doses or forgo relief. The new formulation uses an advanced technology called ElectroNanoSpray to improve the drug’s solubility and dissolution rate, maintaining consistent bioavailability regardless of acid reducer use. Clinical trials also showed CAVHANZA has no food effect, meaning patients can take it with or without meals, and the orally disintegrating tablet can be taken with or without water, which may help those who have difficulty swallowing.
The treatment is approved for adults with chronic or accelerated phase Ph+ CML who are resistant to or intolerant of prior therapy, including imatinib. The approval marks the 10th product in Cycle Pharmaceuticals’ portfolio and the second designed specifically for CML patients. The company partnered with Flex Pharma, which developed the reformulation using its proprietary ENS platform. “Finding the right treatment that fits a patient’s needs and lifestyle can be a stressful step in a patient’s treatment journey,” said Victoria Dickinson, Chief Product Officer at Cycle, in a statement. The drug will be supported by a dedicated patient assistance program called Cycle Vita.
As with other nilotinib products, CAVHANZA carries important safety warnings. The drug can prolong the QT interval, a heart rhythm measurement, and has been associated with sudden deaths. It is contraindicated in patients with low potassium or magnesium levels or long QT syndrome. Healthcare providers should monitor electrolytes, conduct regular ECGs, and avoid use with QT prolonging drugs and strong CYP3A4 inhibitors. Common side effects include rash, headache, nausea, fatigue, and changes in blood cell counts. The drug can cause fetal harm, so effective contraception is required during treatment and for 14 days after the last dose.
Cycle Pharmaceuticals expects to launch CAVHANZA in the coming months. For patients managing the daily challenges of CML therapy, this new option offers welcome flexibility and the potential for fewer disruptions to their treatment routine and quality of life.