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Gene Therapy LentiGlobin Granted Priority Medicines Designation for Sickle Cell Disease in the EU

LentiGlobin is already approved in Europe under the brand name 'Zynteglo' for the treatment of transfusion-dependent β-thalassaemia

Bluebird Bio’s investigational gene therapy LentiGlobin has been granted priority medicines (PRIME) designation by the European Medicines Agency (EMA) for the treatment of sickle cell disease (SCD).

Given to candidate therapies with promising clinical data for diseases of high unmet medical need, the designation is designed to speed clinical development and review of medicines, helping them to reach patients earlier.

“The PRIME designation shows that the EMA recognizes the importance of bringing innovative medicines to patients with SCD efficiently and will allow us to work even more closely with the Agency to help expedite the development and review of LentiGlobin for SCD,” stated Anne-Virginie Eggimann, senior vice president of regulatory science at Bluebird Bio.

LentiGlobin is a gene therapy designed to increase the levels of hemoglobin, the protein in red blood cells that carries oxygen and is defective in people with SCD due to mutations in the HBB gene.

Specifically, the therapy delivers a modified, but functional, copy of the HBB gene into a patient’s red blood cell precursors, or hematopoietic stem cells.

Once matured, the red blood cells start producing an anti-sickling version of hemoglobin, called HbAT87Q. The gene therapy is expected to lower the proportion of defective hemoglobin in the red blood cells, ultimately preventing their destruction and other complications associated with SCD.

The EMA’s decision was based on promising data from the clinical program of LentiGlobin, including the completed HGB-205 Phase 1/2 trial (NCT02151526) and the ongoing HGB-206 Phase 1/2 trial (NCT02140554), and their long-term follow-up study, LTF-303 (NCT02633943).

Results from the SCD patients, ages 13–21, showed that the gene therapy induced HbAT87Q production in all three patients, with levels reaching 50% of total hemoglobin in one patient after a median follow-up of 31.7 months. The remaining two patients had not yet reached the 30% of anti-sickling hemoglobin predicted to have therapeutic impact, but their follow-up was still short at 6.1 and 3.4 months.

In addition to the recent PRIME designation, LentiGlobin has also received orphan drug designation from the U.S. Food and Drug Administration (FDA) and the European Commission, as well as the FDA’s fast trackregenerative medicine advanced therapy, and rare pediatric disease designations for the treatment of SCD.

Read the Bluebird Bio’s Full Press Release HERE.


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