EMA, Gene Therapy, Regulatory

Bluebird’s one-shot gene therapy for beta thalassemia officially secures EMA backing — approval expected in the second quarter

Days after two charities abruptly preannounced that the EMA had endorsed the approval of bluebird bio’s keenly anticipated gene-replacement therapy LentiGlobin, for beta thalassemia — a rare, inherited blood disorder — the European regulator made the recommendation official on Friday.

The one-shot therapy for this patient population — who usually require lifelong treatment with blood transfusions and medication — is expected to win EC approval by the second quarter, and US approval next year.

The Cambridge, Massachusetts-based company’s shares $BLUE lifted up nearly 2% in premarket trading to $156.02.

The therapy is expected to help patients who need regular blood transfusions and have no matching donor for a stem cell transplant become transfusion-independent.

In order for LentiGlobin to work, stem cells are harvested from the patient’s body, and the patient is given chemotherapy to prime their bone marrow to receive the therapy. A viral vector is then used to implant a healthy copy of the beta-globin gene into the bone marrow, stimulating the production of red blood cells.

Nick Leschly

The list price of the company’s gene therapy, branded as Zynteglo, has not been announced, but is expected to be in the seven-figure range, albeit below $2.1 million — which the company believes is the “intrinsic value” of the treatment. SVB Leerink analysts have modeled the launch price of LentiGlobin as $1.2 million in the US and $0.9 million in the EU.

Earlier this year, CEO Nick Leschly proposed a mortgage-like installments-based plan for the pricey therapy. It involves an upfront 20% of the cost of the treatment, while the rest is expected to come in 20% installments per year via the insurer if the drug does indeed work as intended. Except unlike a mortgage, the product (or in this case the treatment) is chosen for the individual, and payments are based on treatment outcomes.

Source: Bluebird Bio

SVB Leerink analysts forecast global peak sales of $1.2 billion in 2030 for LentiGlobin, in a note published on Friday. The therapy is also being evaluated for use in sickle cell disease, another blood disorder.

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Research Scientist - Immunology
Recursion Pharmaceuticals Salt Lake City, UT
Director of Operations
Atlas Venture Cambridge, MA

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