Biotech company bluebird bio has been awarded a priority medicines (PRIME) designation by the European Medicines Agency (EMA) for its gene therapy LentiGlobin as a treatment for sickle cell disease (SCD).
The PRIME designation for LentiGlobin was based on a clinical data package from the completed phase 1/2 HGB-205 study, as well as the ongoing phase 1/2 HGB-206 study and long-term safety and efficacy follow-up LTF-303 study.
LentiGlobin is designed to add functional copies of a modified form of the β-globin gene, which does not work as it should in sickle cell patients, into haematopoietic stem cells.
After patients receive copies of the functional gene, their own red blood cells can produce anti-sickling haemoglobin which decrease the amount of faulty sickled haemoglobin in the blood.
In the HGB-206 study, LentiGlobin achieved a near complete elimination of vaso-occlusive crises (VOCs) and acute chest syndrome (ACS) in adolescents and adults with sickle cell disease, along with a 99.5% reduction on the annualised rate of 14 patients with a documented history of attacks.
Researchers also followed up the patients for up to two years, showcasing the potential durability of LentiGlobin treatment. The therapy also increased the proportion of the anti-sickling form of haemoglobin (HbAT87Q) from 44% of total haemoglobin at six months to more than 50% among patients treated 24 months previously.
The PRIME initiative provides enhanced support partnered with an intent to optimise development plans and accelerate regulatory evaluations for potentially transformative therapies.
“Even with recent progress to deliver new medicines for sickle cell disease, there remains unmet need for people living with SCD,” said Anne-Virginie Eggimann, senior vice president, regulatory science at bluebird bio.
“The PRIME designation shows the importance of expediting the development and review of treatment options for patients with SCD,” she added.
LentiGlobin is approved under the brand name Zynteglo in Europe for the treatment of dependent beta thalassaemia (TDT) who do not have a matching donor for a stem cell transplant.
TDT is a hereditary disease caused by mutation in the β-globin gene that results in significantly reduced or absent adult haemoglobin. Those who suffer with this condition depend on blood transfusions to maintain haemoglobin levels throughout their lifetime.
Due to the coronavirus pandemic, however, bluebird bio had to push back the commercial launch of the gene therapy in this indication, as patients are required to receive the treatment in hospital.