ORCHARD THERAPEUTICS PLC.

The EC granted full, or standard, marketing authorization for Libmeldy (OTL-200) (autologous CD34+ cell enriched population that contains hematopoietic stem and progenitor cells transduced ex vivo using a lentiviral vector encoding the human arylsulfatase-A (ARSA) gene) for the treatment of early-onset MLD. As of December 11, 2023, Libmeldy (OTL-200) is under Priority Review by the FDA, with a Prescription Drug User Fee Act (PDUFA) goal date of March 18, 2024.

Strimvelis was the first ex vivo autologous gene therapy approved by the EMA. It has not been approved by FDA. In March 2022, Orchard said it would discontinue investment in and seek alternatives for Strimvelis, OTL-103 for treatment of WAS and OTL-102 for treatment of X-CGD. The Group’s product sales of Strimvelis are currently distributed exclusively at the San Raffaele Hospital in Milan, Italy.