FDA: Ukoniq Under Investigation for Possible Risk of Death

The agency said data from a trial testing the PI3 kinase inhibitor as a treatment for chronic lymphocytic leukemia prompted the concern.

The FDA announced today that it is investigating the possibility of an increased risk of death associated Ukoniq (umbralisib) because of worrying data from a phase 3 trial assessing the drug as a treatment for chronic lymphocytic leukemia (CLL).

The agency’s statement says results from the Unity trial show “a possible increased risk of death” in patients receiving the combination of Ukoniq and ublituximab, an investigational monoclonal antibody.

“Because of the seriousness of this safety concern and the similarities between the two types of cancer for which this drug is approved and the type of cancer that was studied in the clinical trial, we are alerting patients and health care professionals that we are re-evaluating this risk against the benefits of Ukoniq for its approved uses,” the FDA statement says.

Ukoniq, an oral drug in the PI3 kinase class, has been approved as a treatment for two types of lymphoma. The FDA approved it in February 2021 as a treatment for adults with marginal zone lymphoma (MZL) with relapsed or refractory disease that had been previously treated with an anti-CD20 regimen. The agency has also approved Ukoniq as a treatment for relapsed and refractory follicular lymphoma (FL) after at least pervious treatments.

There have been similar safety concerns about other PI3 kinase inhibitors, the FDA statement said.

TG Therapeutics, a biotech company in Edison, New Jersey, distributes Ukoniq and is the sponsor of the Unity trial. The company, which is focused on developing therapies for B-cell cancers and autoimmune diseases, put out a press release in November 2021 announcing that the FDA’s Oncologic Drugs Advisory Committee was holding a meeting to discuss the Unity trial and its pending application for the UKONIQ-ublituximab combination. The company’s stock has lost two-thirds of its value since November.