FDA approves two treatments for rare blood cancers in same week

Adults with a rare form of non-Hodgkin lymphoma and people over the age of 75 who are newly diagnosed with acute myeloid leukemia (AML) now have new treatment options. Two cancer drugs have received approval this week.

First, the FDA has granted accelerated approval to BeOne Medicine’s Beqalzi (sonrotoclax) to treat adults with relapsed or refractory (R/R) mantle cell lymphoma (MCL). It is indicated for patients who have already received at least two lines of systemic therapy, including a Bruton’s tyrosine kinase (BTK) inhibitor.

Mantle cell lymphoma is an aggressive form of non-Hodgkin lymphoma (NHL), representing roughly 5% of non-Hodgkin lymphoma cases. Mantle cell lymphomas are more common in men and in older adults. In mantle cell lymphoma, relapse after initial treatment is common.

“For people living with relapsed or refractory mantle cell lymphoma, each progression can bring uncertainty and questions regarding remaining treatment options,” Meghan Gutierrez, CEO of the Lymphoma Research Foundation, said in a news release. “The FDA approval of sonrotoclax represents significant progress for the U.S. mantle cell lymphoma community, offering renewed hope for patients and families who have exhausted other available therapies. Advances like this underscore why continued research and innovation in this disease remain so critical.”

Beqalzi inhibits the BCL-2, a protein that plays a key role in preventing apoptosis, or cell death, in cancer cells. It will be available in the second half of this year. Pricing will be available closer to launch, a spokesperson told Managed Healthcare Executive.

“We are actively engaged with U.S. payers as part of our launch planning for Beqalzi,” the spokesperson said. “Consistent with BeOne’s long‑standing ‘patients first’ philosophy, our focus is on supporting timely, appropriate patient access through proactive payer dialogue and robust patient support infrastructure. As we move closer to launch, we expect to share more details on coverage status and access programs.”

The accelerated approval of Beqalzi is supported by data from the phase 1/2 study, BGB-11417-201, which was presented at the 67th American Society of Hematology (ASH) Annual Meeting & Exposition in December 2025. The single-arm study recruited 125 patients.

The study found that the overall response rate was 52%, and the complete response rate was 16%. The median duration of response was 15.8 months. The most common grade ≥3 treatment-emergent adverse events (TEAEs) in greater than 10% of patients were neutropenia (19.1%), infections (16.5%), and pneumonia (10.4%).

A confirmatory, CELESTIAL-RRMCL trial (NCT06742996) is under way, which is a global, randomized, double-blind study evaluating the efficacy and safety of Beqalzi plus BeOne’s Brukinsa (zanubrutinib) compared with Brukinsa plus placebo in adult patients with relapsed or refractory (R/R) mantle cell lymphoma.

Brukinsa is already approved to treat mantle cell lymphoma, as well as Waldenstrom’s macroglobulinemia, marginal zone lymphoma, chronic lymphocytic leukemia and follicular lymphoma.

New AML treatment

The FDA has also approved Taiho Oncology’s Inqovi (decitabine and cedazuridine) in combination with venetoclax as a treatment for adults with newly diagnosed acute myeloid leukemia (AML) who are 75 years or older or who are ineligible for intensive induction chemotherapy.

In 2026, an estimated 22,720 people in the United States will be diagnosed with AML, a cancer of the blood and bone marrow. More than half of those patients are likely to be ineligible for intensive induction chemotherapy due to advanced age or health concerns.

Inqovi is an orally administered hypomethylating regimen that is also approved in the United States and Canada to treat adults with myelodysplastic syndromes (MDS) and chronic myelomonocytic leukemia. Taiho Oncology provides a $0 copay program enrollment for commercially insured patients

Approval is based on data from the ASCERTAIN-V clinical trial demonstrating that Inqovi in combination with venetoclax met complete response endpoints, with no new safety concerns reported. The trial met its primary endpoint with a complete response rate of 46.5% of 47 patients. Median overall survival was estimated to be 15.5 months. At 12 months, median duration of response had not been reached, and more than 75% of patients achieving complete response status remained in complete response.

No new safety concerns were reported. Adverse events (AEs) of grade 3 and higher were reported in 98% of patients of 99 patients; most commonly, febrile neutropenia (49.5%), anemia (38.6%), and neutropenia (35.6%) were reported. No drug-drug interactions were observed between Inqovi and venetoclax.