Kura Oncology and Kyowa Kirin Receive Approval for KOMZIFTI™ (ziftomenib for Relapsed or Refractory NPM1-Mutated AML
Friday, November 14, 2025
Kura Oncology and Kyowa Kirin have received full approval from the US Food and Drug Administration (FDA) for KOMZIFTI™ (ziftomenib), a once-daily oral therapy for adults with relapsed or refractory acute myeloid leukaemia (AML) carrying a susceptible NPM1 mutation.
KOMZIFTI continues to be evaluated for use in earlier lines of treatment and in combination with approved therapies for AML with NPM1 mutations, KMT2A translocations and FLT3 mutations, with the aim of improving outcomes across a broader patient population.
These patients often have limited treatment options, and KOMZIFTI is the first targeted menin inhibitor approved for this specific group.
NPM1 mutations are among the most common in AML, appearing in around 30% of cases. A significant number of patients do not respond to initial treatment, and many who do respond are likely to relapse within a short period. This highlights the need for more effective and durable treatment options.
The FDA approval is based on results from the pivotal KOMET-001 study, which assessed the safety and efficacy of the therapy in 112 adults with relapsed or refractory NPM1-mutated AML. The combined rate of complete remission and partial haematologic recovery was 21.4%. The median duration of remission was five months, and most responses occurred within six months of starting treatment.
KOMZIFTI showed a manageable safety profile. Common adverse reactions included liver enzyme increases, electrolyte changes, infections, haemorrhage, diarrhoea, nausea, fatigue, and differentiation syndrome. The therapy carries a Boxed Warning for differentiation syndrome, a known risk associated with drugs that restore cell differentiation. Clinically meaningful interactions with other medicines were not reported, which may support its use alongside other therapies.
To support access, Kura has launched Kura RxKonnect™, a programme designed to assist eligible patients with reimbursement and related services.
Source: kuraoncology.com
