A new oral drug for use in patients with certain blood cancers has been approved by the US Food and Drug Administration (FDA). The drug, duvelisib (Copiktra, Verstem Oncology), is an oral inhibitor of phosphoinositide 3–kinase (PI3K) and is the first to act as a dual inhibitor of PI3K-delta and PI3K-gamma.
The FDA approved for the use of duvelisib in adult patients with relapsed or refractory chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (CLL/SLL) who have received at least two prior therapies. This approval was based on data from the DUO trial, which showed a benefit in progression-free survival.
The FDA also granted accelerated approval for the treatment of adult patients with relapsed or refractory follicular lymphoma (FL) who had undergone at least two prior systemic therapies. This accelerated approval was based on overall response rate from the DYNAMO clinical trial. Further data showing clinical benefit are required.
These three disorders CLL/SLL and FL are common types of indolent non-Hodgkin lymphomas. It is estimated that 681,000 people are living with non-Hodgkin lymphoma in the United States, including nearly 350,000 patients with CLL/SLL or FL, notes the manufacturer.
Many of these patients will eventually experience relapse or develop the refractory disease, it adds. The drug had orphan drug designation and was approved after a priority review.
"Duvelisib is an important addition to the evolving treatment paradigm for patients with CLL/SLL and FL," said Ian Flinn, MD, director of the Lymphoma Research Program at Sarah Cannon Research Institute. He was lead investigator of the pivotal studies that led to this approval. Together, the studies involved more than 400 patients.
"I believe it will address an unmet need for patients who have limited options once they have progressed after two prior therapies," he said in a company press release.
Boxed Warning and Adverse Effects
The product carries a boxed warning for four fatal and serious toxicities: infections, diarrhea or colitis, cutaneous reactions, and pneumonitis. The manufacturer is implementing an informational risk evaluation and mitigation strategy to provide appropriate dosing and safety information to physicians.
The drug is also associated with adverse reactions that may require dose reduction, treatment delay, or discontinuation. These may include infections, diarrhea or colitis, cutaneous reactions, pneumonitis, hepatotoxicity, neutropenia, and embryo-fetal toxicity.
The most common adverse reactions (reported in ≥20% of patients) were diarrhea or colitis, neutropenia, rash, fatigue, pyrexia, cough, nausea, upper respiratory infection, pneumonia, musculoskeletal pain, and anemia.