EMA is approving Zynteglo gene therapy for patients with beta-thalassemia
The Committee for Medicinal Products for Human Use (EMA) recommends granting conventional marketing authorization for Zynteglo gene therapy.
This is a drug for patients with beta-thalassemia (from the age of 12) who need regular blood transfusions and who haven’t found suitable donors for stem cell transplantation.
Thalassemia is a group of hereditary diseases of the hematopoietic system, which are characterized by impaired hemoglobin synthesis. Therefore, the main symptom of thalassemia is anemia.
If the synthesis of β-chains is impaired, then it is a question of beta-thalassemia; at the same time, other hemoglobin variants are formed that do not contain β-chains. Beta-thalassemia is more common. There are major, intermediate and minor forms; the most serious disease is thalassemia of major form, while the minor one allows you to lead a near-normal life.
When developing the drug received the status of orphan. The development of gene therapy is carried out with the support of EMA in the framework of the PRIME program (PRIority MEdicines).
This platform is designed to establish a dialogue with the developers of promising innovative products in the early stages.
Such interaction contributes to the formation of a more complete set of documents, allowing to speed up the evaluation procedure and make a decision in just 150 days, which is a record time today.
The decision to grant conventional marketing permission to Zynteglo was made at the March EMA meeting.
Another 3 drugs also received a favourable decision on the expansion of indications.
In order to obtain a conventional sale authorization, the data on the drug must indicate that the benefits of the drug outweigh the possible risks. And the applicant must provide full clinical data in the future.