WASKYRA (Etuvetidigene Autotemcel)

WASKYRA (etuvetidigene autotemcel) is a one-time gene therapy for Wiskott-Aldrich syndrome (WAS), a rare inherited disorder of the immune system in which patients get frequent serious infections and bruise and bleed easily because they have too few normal platelets. The US FDA approved it on 9 December 2025 for children aged 6 months and older and adults who have a WAS gene mutation, for whom a stem cell transplant is appropriate but no suitable HLA-matched related donor is available. The EU authorised it on 9 January 2026 with the same scope.

The treatment uses the patient’s own blood stem cells (CD34+ cells). In the laboratory, a lentiviral vector that cannot replicate adds working copies of the WAS gene to these cells. After pre-treatment with rituximab and reduced-intensity chemotherapy (busulfan and fludarabine), the corrected cells are infused back. They settle in the bone marrow and produce blood cells that make the missing WAS protein.

In 27 patients aged 1 to 35 years followed for a median of about 5.7 years, the rate of severe infections fell from 2.0 to 0.2 per patient-year, and moderate-to-severe bleeding from 2.0 to 0.8 per patient-year. The FDA called it the first cell-based gene therapy for WAS and the first approved cell and gene therapy from a non-profit applicant. It was developed at the San Raffaele Telethon Institute for Gene Therapy in Milan.

Like Lenmeldy (atidarsagene autotemcel), it is a gene-corrected stem cell therapy made from the patient’s own cells, so it is produced individually for each patient.

• Not to be used after a donor stem cell transplant in the previous 6 months (or if donor cells remain), after a previous stem cell gene therapy, or when mobilisation or conditioning cannot be given.
• A back-up of the patient’s own untreated stem cells (at least 3 × 10⁶ CD34+ cells/kg) must be collected and frozen before conditioning, in case the gene-corrected cells do not engraft.
• Low blood counts can last several weeks; counts are checked for at least 8 weeks after infusion. Serious infections can occur because of rituximab and chemotherapy, and IgG levels are kept above 5 g/L.
• Hepatic veno-occlusive disease (liver vessel blockage) may occur after busulfan; liver tests are checked for one month.
• There is a theoretical lifelong risk of blood cancer from the vector (insertional oncogenesis). Patients need lifelong follow-up, with yearly checks for at least 15 years.
• Most common side effects in studies: rash, respiratory and catheter infections, diarrhoea, liver injury, small skin bleeds, vomiting, anaemia and fever. Patients may test falsely positive on some HIV PCR tests and must never donate blood, organs or cells.

Can WASKYRA be shipped to India like a normal medicine?

No. WASKYRA is made from each patient’s own stem cells, which are collected, sent to the manufacturing site, gene-corrected and returned frozen for that same patient. It is given only at treatment centres qualified by the manufacturer, so access from India normally means treatment at a qualified centre abroad, arranged with the patient’s immunologist or transplant team.

What information does the treating team need?

• Genetic report confirming a mutation in the WAS gene
• Letter from the paediatric immunologist or transplant physician confirming that a transplant is appropriate and no HLA-matched related donor is available
• Recent clinical summary (infections, bleeding, platelet counts, previous treatments)
• Passport details of the patient and caregiver for travel planning

How long does the whole process take?

It involves several stages: stem cell mobilisation with G-CSF and plerixafor, collection by apheresis, manufacturing, rituximab about 22 days before infusion, busulfan and fludarabine conditioning, the infusion itself, and then weeks of close monitoring while blood counts recover. Families should plan for an extended stay near the treatment centre.

How is the product stored?

Frozen in the vapour phase of liquid nitrogen below −130 °C, with a shelf life of 6 months. After thawing, each bag must be infused within 2 hours, so the infusion is timed precisely at the centre.

Because WASKYRA is a personalised gene therapy, IPN does not import it as a stock product. We help families and their doctors with the first steps: sharing the official product information, contacting the marketing authorisation holder about eligibility and qualified treatment centres, and organising the documents that centres ask for.

To discuss WASKYRA treatment options and cost: Call +91 96548 60915, WhatsApp +91 93100 90915 or email info@indianpharmanetwork.in.

Global Approval Status:

• United States: FDA approval on 9 December 2025 (BLA 125846; Orphan Drug, Rare Pediatric Disease and RMAT designations).
• European Union: Marketing authorisation on 9 January 2026 (orphan medicine).
• India: Not approved by CDSCO.

Sources:

• WASKYRA US Prescribing Information (FDA)
• FDA news release: first gene therapy for Wiskott-Aldrich syndrome
• WASKYRA – EU product information (EMA EPAR)
• Fondazione Telethon announcement of FDA approval

Information last checked: October 2026.

What is Wiskott-Aldrich syndrome?

A rare inherited disorder of the immune system caused by mutations in the WAS gene. People with WAS have frequent severe infections and bruise and bleed easily because they have too few normal platelets.

Is WASKYRA given only once?

Yes. It is a single infusion of the patient’s own gene-corrected stem cells, after preparation with rituximab and chemotherapy.

Does the patient need a donor?

No. WASKYRA is meant for patients without a suitable HLA-matched related donor, because it uses the patient’s own cells.

Who developed WASKYRA?

Fondazione Telethon, an Italian non-profit, together with the San Raffaele Telethon Institute for Gene Therapy in Milan.

What follow-up is needed after treatment?

Close blood-count monitoring for at least 8 weeks, infection prevention, and lifelong yearly check-ups for at least 15 years to look for any blood cancer.

Is WASKYRA FDA approved?

Yes, since 9 December 2025, for patients aged 6 months and older.