By Fineline Cube 
The US Food and Drug Administration (FDA) approved Omisirge (omidubicel-onlv) as the first hematopoietic stem cell transplant (HSCT) therapy to treat patients with severe aplastic anemia (SAA). The approval expands Omisirge’s indication to include adults and pediatric patients six years and older with SAA following reduced intensity conditioning, for whom a compatible donor is not available.
Regulatory Milestone & Clinical Data
| Attribute | Details |
|---|---|
| Product | Omisirge (omidubicel-onlv) |
| Developer | Gamida Cell Ltd. |
| Regulatory Status | FDA approved (first HSCT therapy for SAA) |
| Indication | Severe aplastic anemia (SAA) in patients ≥6 years |
| Study Design | Ongoing, open-label, prospective, single-arm study |
| Efficacy Population | 14 patients |
| Neutrophil Engraftment | 12 of 14 patients (85.7%) |
| Median Time to Recovery | 11 days (range 7-20 days) |
Mechanism of Action & Product Differentiation
Omisirge Technology:
- Stem Cell Source: Donated umbilical cord blood stem cells
- Enhancement: Chemically enhanced with nicotinamide (vitamin B3)
- Mechanism: Restores blood and immune system function
Addresses Key Limitations:
- Delayed Hematopoietic Recovery: Omisirge provides early and sustained neutrophil engraftment
- Infection Risk: Reduces infection risk vs. standard umbilical cord transplant
- Donor Availability: Provides graft option when matched sibling or related donor unavailable
Market Context & Unmet Medical Need
Severe Aplastic Anemia (SAA):
- Rare, life-threatening blood disorder where bone marrow fails to produce red blood cells, white blood cells, and platelets
- Treatment Options: Immunosuppressive therapy and/or HSCT (preferably from matched sibling donor)
- Donor Limitation: ~70% of patients lack compatible matched donor
- Current Gap: Umbilical cord transplant limited by slow recovery and infection risk
Clinical Impact:
- Omisirge offers first approved alternative for donor-unavailable SAA patients
- Provides rapid neutrophil recovery (median 11 days vs. 21-28 days with standard UCB)
- Reduces transplant-related mortality
Strategic Outlook & Gamida Cell Positioning
- Manufacturing: Leverages established Omisirge production platform
- Commercial Infrastructure: Builds on existing hematologic malignancy distribution network
- Revenue Expansion: SAA indication doubles addressable patient population
- Next Milestones: Real-world evidence generation; potential pediatric expansion studies
- Competitive Moat: First and only approved product for SAA HSCT; 7+ years patent protection
Forward‑Looking Statements
This brief contains forward‑looking statements regarding Omisirge’s commercial launch timeline, market penetration, and real-world outcomes. Actual results may differ due to manufacturing capacity, competitive dynamics, and reimbursement uncertainties.-Fineline Info & Tech