WORLDSymposium 2025 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 2025. 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 2025 New Treatment Awards were presented to:

IntraBio for the FDA approval of AQNEURSA™ (levacetylleucine) for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing ≥15 kg.
 

Orchard Therapeutics for receiving FDA Approval of LENMELDY™ (atidarsagene autotemcel) for the treatment of children with pre-symptomatic late infantile (PSLI), pre-symptomatic early juvenile (PSEJ), or early symptomatic early juvenile (ESEJ) (collectively referred to as early-onset metachromatic leukodystrophy (MLD)).
 

Zevra Therapeutics for receiving FDA approval of MIPLYFFA™ (arimoclomol) capsules for use in combination with miglustat for the treatment of neurological symptoms associated with Niemann-Pick disease type C (NPC) in adults and children 2 years of age and older.

Congratulations!

The WORLDSymposium 2025 New Treatment Awards were presented on Friday, February 7, 2025, at 07:30 PST, followed by Late-breaking Science, Contemporary Forum abstract presentations and the Rapid Fire Competition.