The FDA has issued its stamp of approval to a new, cell-based option to treat Wiskott-Aldrich syndrome (WAS), marking the first therapy of its kind for the rare disease and making Italy’s Fondazione Telethon the first nonprofit to usher a gene therapy across the regulatory finish line in the US.
Branded as Waskyra, the drug is specifically indicated for children six months and older, as well as adults who have a mutation in the WAS gene. To be eligible for the ex vivo gene therapy, patients must have no available human leukocyte antigen-matched related stem cell donor and be cleared for hematopoietic stem cell transplantation, the FDA said in its recent announcement.
The drug was developed through decades of research at the San Raffaele Telethon Institute for Gene Therapy in Milan, Fondazione Telethon said in a press.
The organisation, an Italian nonprofit that specialises in rare and complex genetic diseases, is the first nonprofit to have successfully taken an ex vivo gene therapy from the lab to regulatory approval, it added.
Waskyra is made up of a patient’s own hematopoietic blood stem cells, which are genetically modified to include functional copies of the WAS gene and reinfused intravenously following chemotherapy.
