Etuvetidigene autotemcel
| Clinical data | |
|---|---|
| Trade names | Waskyra |
| Other names | GSK-2696275, OTL-103 |
| Routes of administration | Intravenous |
| ATC code |
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| Legal status | |
| Legal status | |
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Etuvetidigene autotemcel, sold under the brand name Waskyra, is a gene therapy medication used for the treatment of Wiskott–Aldrich syndrome.[2][3]
The most common side effects include rash, respiratory tract infection, febrile neutropenia, catheter related infection, vomiting, diarrhea, liver injury, and petechiae.[4]
The active substance of etuvetidigene autotemcel consists of genetically modified autologous CD34+ hematopoietic stem cell enriched population transduced ex vivo with a lentiviral vector encoding the human Wiskott–Aldrich syndrome gene.[3] Etuvetidigene autotemcel is a gene therapy that inserts the Wiskott–Aldrich syndrome corrected gene into the cell's genome, making the genetically modified cells capable of expressing the functional Wiskott–Aldrich syndrome protein.[3] The genetically modified cells engraft and repopulate the hematopoietic compartment.[3] They differentiate and produce biologically active lymphoid and myeloid progenitors whose progeny express Wiskott–Aldrich syndrome protein.[3]
Etuvetidigene autotemcel was approved for medical use in the United States in December 2025.[4]
Medical uses
Etuvetidigene autotemcel is indicated for the treatment of people with Wiskott-Aldrich syndrome who have a mutation in the WAS gene and for whom hematopoietic stem cell transplantation is appropriate and no suitable human leukocyte antigen-matched related stem cell donor is available.[2][4]
Wiskott-Aldrich syndrome is a rare, life-threatening genetic disease caused by mutations in the WAS gene.[4] The condition is characterized by bleeding, eczema, recurrent infections, and increased susceptibility to autoimmunity and lymphoreticular malignancies.[4]
Etuvetidigene autotemcel consists of the recipient's own hematopoietic (blood) stem cells, which have been genetically modified to include functional copies of the WAS gene.[4] Following reduced-intensity conditioning, the gene-corrected cells are infused intravenously to restore blood cell production.[4] Etuvetidigene autotemcel restores functional WAS protein expression in affected cells, addressing the underlying cause of the disease.[4]
History
The safety and effectiveness of etuvetidigene autotemcel was assessed based on two open-label, single-arm, multinational clinical studies and an expanded access program totaling 27 participants with severe Wiskott-Aldrich syndrome, which demonstrate substantial and sustained clinical benefit for people with severe Wiskott-Aldrich syndrome, with significant reductions in the primary disease manifestations that drive morbidity and mortality.[4]
The US Food and Drug Administration (FDA) granted the application for etuvetidigene autotemcel orphan drug, rare pediatric disease, and regenerative medicine advanced therapy designations.[4] The FDA granted approval of Waskyra to Fondazione Telethon ETS.[4] It is a first approved cell and gene therapy product from a non-profit applicant.[4]
Society and culture
Legal status
In November 2025, the Committee for Medicinal Products for Human Use of the European Medicines Agency adopted a positive opinion, recommending the granting of a marketing authorization for the medicinal product Waskyra, intended for the treatment of Wiskott–Aldrich syndrome.[3] The applicant for this medicinal product is Fondazione Telethon ETS.[3][5]
Etuvetidigene autotemcel was approved for medical use in the United States in December 2025.[4]
Names
Etuvetidigene autotemcel is the international nonproprietary name.[6]
Etuvetidigene autotemcel is sold under the brand name Waskyra.[2][4]
References
- ^ "Waskyra (etuvetidigene autotemcel) suspension, for intravenous use" (PDF). Fondazione Telethon ETS. December 2025. Retrieved 11 December 2025.
- ^ a b c d "Waskyra". U.S. Food and Drug Administration (FDA). 9 December 2025. Retrieved 11 December 2025.
- ^ a b c d e f g "Waskyra EPAR". European Medicines Agency (EMA). 14 November 2025. Retrieved 9 December 2025. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
- ^ a b c d e f g h i j k l m n "FDA Approves First Gene Therapy Treatment for Wiskott-Aldrich Syndrome". U.S. Food and Drug Administration (FDA) (Press release). 9 December 2025. Retrieved 11 December 2025. This article incorporates text from this source, which is in the public domain.
- ^ "First gene therapy to treat rare disease Wiskott-Aldrich syndrome". European Medicines Agency (EMA). 14 November 2025. Retrieved 9 December 2025. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
- ^ World Health Organization (2022). "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 87". WHO Drug Information. 36 (1). hdl:10665/352794.
External links
- "Etuvetidigene Autotemcel ( Code - C184832 )". EVS Explore.
- Clinical trial number NCT01515462 for "Gene Therapy for Wiskott-Aldrich Syndrome (TIGET-WAS)" at ClinicalTrials.gov
- Clinical trial number NCT03837483 for "A Clinical Study to Evaluate the Use of a Cryopreserved Formulation of OTL-103 in Subjects With Wiskott-Aldrich Syndrome" at ClinicalTrials.gov