FDA OKs New Drug Application for Momelotinib to Treat Myelofibrosis


The FDA has set a date to decide whether or not to approve momelotinib for myelofibrosis, a rare type of blood cancer.

The Food and Drug Administration (FDA) has accepted the new drug application for the novel drug momelotinib to treat myelofibrosis, a rare type of blood cancer.

The new development comes mere months after Sierra Oncology, the manufacturer of momelotinib, submitted the application to the FDA.

Of note, Sierra Oncology was acquired by GSK in July.

A new drug application, according to the FDA, is submitted by a pharmaceutical company when they are ready to formally seek approval from the agency to sell and market a new drug in the United States. In its application, the pharmaceutical company seeking a new drug approval must provide enough data to show that the drug is safe and effective in its targeted patient population. Moreover, the findings must show the benefits outweigh any potential risks from the drug.

Momelotinib is not currently approved by any agency in the world.

The new drug application was submitted using data from multiple studies, including the phase 3 MOMENTUM trial.

In this study, patients with myelofibrosis who presented with symptoms of the disease, were anemic and had been previously treated with an FDA-approved JAK inhibitor were randomized to receive either momelotinib or danazol.

The main focus of the trial was to assess if momelotinib was safe in this patient population, as well as if the study drug alleviated the symptoms associated with the rare blood cancer. Moreover, investigators aimed to see if momelotinib would reduce the need for blood transfusions in patients with anemia, and also decrease the size of a patient’s spleen.

According to the Mayo Clinic, an enlarged spleen may result in pain or fullness below a person’s ribs on their left side.

An analysis of the MOMENTUM trial at 24 weeks showed that 72.3% of the 130 patients who received momelotinib at the start of the trial had completed their course of treatment. Some of the patients in the momelotinib group had discontinued treatment for a variety of reasons, including side effects (16 patients), death (4 patients) and myelofibrosis progression to leukemia (2 patients).

As for the 65 patients who were prescribed danazol, only 58.5% of the group completed their treatment at 24 weeks.

More patients who received momelotinib experienced a 25% reduction in spleen size compared with those who were administer danazol (40% versus 6.2%).

Additionally, the investigators noted that 23.1% of patients in the momelotinib treatment group derived a 35% reduction in spleen size versus 3.1% of the group who received danazol.

Earlier this year, Dr. Srdan Verstovsek, a researcher on the MOMENTUM study and professor of medicine in the Department of Leukemia at The University of Texas MD Anderson Cancer Center in Houston, talked about the significant need of a drug like momelotinib for patients with myelofibrosis.

“Certainly if the drug is approved, as I would expect it to be in the very near future, momelotinib will be, by far, the No. 1 choice for therapy in the setting where it was tested,” Verstovsek said in an interview with CURE®.

The FDA announced that it plans to decide on the approval by June 2023.

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