EMA recommends approval of Waskyra, a gene therapy treatment for Wiskott-Aldrich syndrome

EMA recommends approval of Waskyra, a gene therapy treatment for Wiskott-Aldrich syndrome

The European Medicines Agency (EMA) has issued a positive opinion on Waskyra (etuvetidigene autotemcel), a gene replacement therapy for patients aged 6 months and older with a confirmed WAS gene mutation who are candidates for hematopoietic stem cell transplantation (HSCT) but do not have a compatible donor.

What is Wiskott-Aldrich syndrome?

Wiskott-Aldrich syndrome (WAS) is an extremely rare X-linked inherited disorder that occurs in boys.

Worldwide prevalence: 1–10 cases per 1 million male newborns.

Cause: WAS gene mutations leading to severe immunodeficiency and hematopoietic disorders:

• Chronic thrombocytopenia and bleeding

• Frequent and severe infections

• High risk of lymphomas and other malignancies

Standard of therapy is HSCT, but with a suitable It's not always possible to find a donor.

How Waskyra Works

Waskyra is an autologous gene therapy based on the patient's own CD34+ cells.

In the laboratory, the cells are modified to produce a functional WAS protein. After a single injection, the modified cells populate the bone marrow and form populations of healthy immune and hematopoietic cells.

Clinical Data

The EMA relied on the results of 27 patients (aged 1 to 35 years) from clinical trials and early access programs.

Key treatment effects:

• severe infections decreased from 2.0 to 0.15 events/year after 1–2 years

• the rate remains low at 0.12 events/year after 2–3 years

• moderate and severe bleeding decreased from 2.0 to 0.16 after 2–3 years

Adverse events were mainly related to the preparatory regimen before cell infusion.

EMA assessment and next steps

The Committee for Advanced Therapies (CAT) and the Committee for Medicinal Products for Human Use (CHMP) concluded that the benefits of the therapy outweigh the potential risks in patients who are candidates for HSCT but do not have an available donor.

The project was developed as part of the EMA's pilot project to support academic and non-profit developers of ATMPs.

The positive CHMP opinion will be submitted to the European Commission for a decision on granting marketing authorization. Once granted, pricing and reimbursement conditions will be regulated individually by EU countries.