IPOPI Applauds EMA’s Positive Opinion on Waskyra™ for Wiskott-Aldrich Syndrome

IPOPI has welcomed the positive opinion issued by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA). The opinion recommends granting marketing authorisation in the European Union for Waskyra™, a groundbreaking ex vivo gene therapy for Wiskott-Aldrich Syndrome (WAS), a rare and life-threatening primary immunodeficiency.

“This marks a significant milestone for gene therapy in the European Union, offering new hope for patients with Wiskott-Aldrich Syndrome,” said Johan Prévot, Executive Director of IPOPI and Board Member of the AGORA Foundation. “This positive opinion will not only increase access to life-saving therapies for those affected by WAS but could also pave the way for the development of more innovative treatments for other primary immunodeficiencies and rare disorders,” added Martine Pergent, IPOPI President. For more information, see here.