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Lumirexa (rexalogene autotemcel)

Transfusion-dependent beta-thalassemia (TDT) in patients 12 years and older without a matched sibling donor

Evidence Agent

Comparative effectiveness, PICO, citations

Asset context

Lumirexa

rexalogene autotemcel

Modality

Autologous ex-vivo lentiviral gene therapy

Indication

Transfusion-dependent beta-thalassemia (TDT) in patients 12 years and older without a matched sibling donor

Mechanism

One-time infusion of autologous CD34+ cells transduced with a lentiviral vector encoding a modified adult beta-globin gene (βA-T87Q), restoring functional hemoglobin production and eliminating transfusion dependence.

Pivotal trial · LUMIRA-301

Design

Open-label, single-arm, multicenter Phase 3, n=42

Primary endpoint

Transfusion independence ≥12 months (weighted average Hb ≥9 g/dL without RBC transfusion for ≥12 months)

Result

37/42 (88.1%) achieved transfusion independence at month 24 (95% CI 74.4–96.0)

Safety

No insertional oncogenesis events observed. Most common Grade ≥3 AEs related to busulfan conditioning (febrile neutropenia 71%, mucositis 52%).

Proposed price

$2.95M one-time, outcomes-based contract over 5 years

Comparators

Lifelong RBC transfusions + iron chelation (deferasirox/deferiprone) — standard of care
Allogeneic HSCT (matched sibling donor) — curative but unavailable for ~75% of patients
Casgevy (exa-cel, CRISPR) — competing one-time gene-edited therapy, FDA approved 2024
Zynteglo (beti-cel) — first-gen lentiviral gene therapy, $2.8M list price