FDA Approves New Biotherapies for Severe Aplastic Anemia, Marginal Zone Lymphoma, Wiskott–Aldrich Syndrome

The Food and Drug Administration recently approved new biotherapies for patients with rare blood cancers, bone marrow failure disorders and inherited diseases.

First Cellular Therapy Approved for SAA

FDA approved omidubicel-onlv (Omisirge, Gamida Cell Ltd), the first hematopoietic stem cell transplant (HSCT) therapy for patients with severe aplastic anemia (SAA). The therapy is indicated for adults and children aged six years and older who lack a compatible stem cell donor and undergo reduced-intensity conditioning.

Omidubicel-only is a stem cell therapy in which donated cord blood stem cells are chemically enhanced with nicotinamide to support faster neutrophil engraftment. In an ongoing single-arm study, 12 of 14 patients achieved neutrophil engraftment, with a median recovery time of 11 days. The most common side effects were febrile neutropenia, viral and bacterial infections, hyperglycemia, immune thrombocytopenia and pneumonia. Autoimmune cytopenias occurred in 25% of patients.  

Omidubicel-only also received Priority Review and Orphan Drug designation. Gamida Cell presented additional interim clinical results for omidubicel during the American Society of Hematology’s 2025 Annual Meeting this week in Orlando, Fla.

First CAR T-Cell Therapy Approved for MZL

FDA authorized a new indication for lisocabtagene maraleucel (Breyanzi, Juno Therapeutics) on Dec. 4, marking the first chimeric antigen receptor (CAR) T-cell therapy approved for adults with marginal zone lymphoma (MZL) who have relapsed or failed at least two prior lines of therapy.

The agency based its approval on findings from an open-label, single-arm trial, in which 95.5% of patients responded to a one-time infusion and 62.1% achieved a complete response. Responses were durable at a median follow-up of 21.6 months. The most common adverse reactions were cytokine release syndrome, diarrhea, fatigue, musculoskeletal pain and headache.

Lisocabtagene maraleucel received both Priority Review and Orphan Drug designation.

FDA Approves First Gene Therapy for Wiskott–Aldrich Syndrome

On Tuesday, FDA approved etuvetidigene autotemcel (Waskyra, Fondazione Telethon ETS), the first cell-based gene therapy for Wiskott–Aldrich Syndrome (WAS), for patients six months and older who lack an appropriate HLA-matched related donor.

WAS is a rare genetic disease caused by mutations in the WAS gene and is characterized by bleeding, recurrent infections and increased susceptibility to autoimmunity and lymphoreticular malignancies. Previously, treatment options were limited to symptom management and allogeneic hematopoietic stem cell transplant.

Etuvetidigene autotemcel uses the patient’s own hematopoietic stem cells, genetically modified to include functional copies of the WAS gene, to restore functional WAS protein expression in affected cells.

Data from two clinical trials and an expanded access program showed substantial and sustained clinical benefit, including a 93% reduction in severe infections and a 60% reduction in moderate and severe bleeding events.

The therapy received FDA’s Orphan Drug, Rare Pediatric Disease, and Regenerative Medicine Advanced Therapy designations.