The U.S. Food and Drug Administration (FDA) has approved ziftomenib to treat adults with relapsed or refractory acute myeloid leukemia (AML) with a susceptible NPM1 gene mutation who have no satisfactory alternative treatment options.
The once-daily oral therapy, developed by Kura Oncology and Kyowa Kirin, will be sold under the brand name Komzifti.
“Together with our partner, Kyowa Kirin, we remain committed to advancing development of Komzifti across the treatment continuum for AML, where its best-in-class profile offers potential for even greater impact in combination regimens and earlier lines of therapy,” Troy Wilson, PhD, president and CEO of Kura, said in a press release from the companies. “We are fully prepared to launch Komzifti today and deliver this new medicine to patients in need.”
Takeyoshi Yamashita, PhD, executive vice president and chief medical officer at Kyowa Kirin, said the FDA approval “underscores our commitment to advancing precision medicines to address the genetic drivers of disease in hematology and oncology.”
Kura offers a support program called Kura RxKonnect to help eligible patients navigate access and insurance coverage for the newly approved treatment.
A ‘major step forward’ for severe, hard-to-treat disease
AML is an aggressive form of blood cancer. In about 30% of cases, the cancer cells carry mutations in the NPM1 gene. According to Kura and Kyowa, about 20% of these cancers won’t respond to treatment at all, and most that do respond initially will relapse (come back) within a few years.
“In AML, where many patients face severe disease progression and limited treatment options, the evolution toward targeted therapies such as Komzifti represents a major step forward and offers potential to transform existing standards of care,” Yamashita said.
Komzifti is designed to block the activity of menin, a protein that contributes to abnormal cell growth in certain cancer cells. FDA approval was based mainly on data from the clinical trial KOMET-001 (NCT04067336), which tested the therapy in more than 100 people with NPM1-mutated AML whose cancer had relapsed or was refractory (meaning it failed to respond to initial treatment or returned after initially responding). Results showed nearly a quarter of patients achieved complete remission after being treated with Komzifti.
Eunice Wang, MD, chief of leukemia service at Roswell Park Comprehensive Cancer Center, said the clinical data “demonstrate deep and durable responses with a manageable safety profile, including no drug-drug interactions.”
“This approval equips physicians with a new oral therapy to integrate into care and improve outcomes for this vulnerable patient population,” Wang said.
According to its prescribing information, common side effects of Komzifti include infection, bleeding, diarrhea, nausea, fatigue, swelling, pain, itching, increased liver enzymes, and febrile neutropenia (a condition marked by fever and low levels of certain immune cells).
The therapy also carries a boxed warning — the FDA’s most stringent safety notice — that it may cause differentiation syndrome, a type of severe reaction to blood cancer treatments that can cause life-threatening complications such as trouble breathing and organ failure.
Kura and Kyowa highlighted that the therapy does not carry boxed warnings for QTc prolongation or Torsades de Pointes, which are forms of abnormal heart rhythms that can be fatal and are associated with certain other blood cancer drugs. While it’s not included as a boxed warning, the prescribing information does note that heart rhythm should be monitored regularly while patients are on Komzifti.
“Komzifti combines compelling efficacy, a favorable safety profile, compatibility with concomitant medications, and convenient once-daily oral administration in a population with few effective treatment options,” Wilson said. “These features highlight Komzifti’s potential to serve as the menin inhibitor of choice in its approved indication.”
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