FDA Approves Danziten, a Nilotinib Formulation for CML Without Mealtime Restrictions

Key Insights

• The FDA has approved Danziten (nilotinib) for chronic myeloid leukemia (CML), offering a new treatment option without the strict fasting requirements of Tasigna.
• Danziten provides comparable efficacy to Tasigna with consistent pharmacokinetics, even without fasting, potentially improving patient adherence to treatment regimens.
• This tyrosine kinase inhibitor (TKI) is approved for newly diagnosed Philadelphia chromosome-positive CML and those resistant or intolerant to prior therapies, including imatinib.

The FDA has approved Azurity Pharmaceuticals' Danziten (nilotinib) for the treatment of chronic myeloid leukemia (CML), marking a significant advancement by eliminating the need for strict mealtime restrictions. This new formulation of nilotinib offers a more flexible treatment option for adult patients with newly diagnosed Philadelphia chromosome-positive (Ph+) CML in chronic phase, as well as those with chronic or acute phase CML who are resistant or intolerant to previous treatments, including imatinib.

Advantages of Danziten

Danziten distinguishes itself from the original nilotinib formulation, Tasigna, by removing the necessity for patients to take the medication in a fasted state. Richard Blackburn, CEO of Azurity Pharmaceuticals, emphasized that Danziten provides equivalent efficacy to Tasigna but liberates CML patients from mealtime restrictions. Tasigna's label carries a boxed warning requiring patients to take it on an empty stomach to avoid potential cardiotoxicity, specifically QT interval prolongation, which can occur if the drug is taken with food due to increased absorption.

Danziten's formulation ensures consistent pharmacokinetics and bioavailability regardless of food intake. This eliminates the risk of excess absorption and potential cardiac events associated with non-adherence to fasting requirements. The enhanced formulation also allows for a lower dose while maintaining efficacy, potentially improving patient adherence, a critical factor in managing CML.

Clinical Significance and Access

Optimal tyrosine kinase inhibitor (TKI) therapy, such as nilotinib, can lead to deep molecular responses and treatment-free remission in some patients. Studies suggest that newly diagnosed patients with chronic-phase CML, when treated appropriately, can achieve a life expectancy comparable to that of the general population. Azurity Pharmaceuticals is facilitating patient access through a network that includes Biologics by McKesson and a limited specialty distribution. The company has also established DanzitenCONNECT, a patient support program that offers prior authorization assistance, benefits investigation, a free initial month of therapy, potential copay reductions, and support through a patient assistance program.