WORLDSymposium 2024 New Treatment Award
WORLDSymposium strives to recognize important achievements in therapy for lysosomal diseases for treatments attaining regulatory approval. In recognition of achieving major milestones, WORLDSymposium reviews potential recipients for the New Treatment Award in 2023. This award honors “new treatments” that are viewed as providing value to patients with lysosomal diseases, and general acceptance as evidenced by approval by the U.S. Food and Drug Administration, European Medicines Agency, and other regulatory authorities.
The WORLDSymposium New Treatment Award for 2023 was presented to Sanofi for olipudase alfa-rpcp (Xenpozyme™) which provided clinical data meriting approval by the U.S. Food and Drug Administration (FDA) as the first disease-specific treatment of non-central nervous system (non-CNS) manifestations of acid sphingomyelinase deficiency (ASMD) in adult and pediatric patients. The New Treatment Award was presented on Saturday, February 25, 2023, at 7:45 AM EST, followed by a full day of Contemporary Forum abstract presentations.