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 will be 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 arimoclomol (MIPLYFFA™) 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 will be presented on Friday, February 7, 2025, at 08:00 PST, followed by Late-breaking Science, Contemporary Forum abstract presentations and the Rapid Fire Competition.

Past Award Recipients:

2024 cipaglucosidase alfa and miglustat (Pombiliti® and Opfolda®, Amicus Therapeutics)
2024 pegunigalsidase alfa-iwxj (Elfabrio®, Chiesi Global Rare Diseases)
2024 velmanase alfa-tycv (Lamzede®, Chiesi Global Rare Diseases) 
2023 olipudase alfa-rpcp (Xenpozyme™, Sanofi) 
2022 avalglucosidase alfa-ngpt (Nexviazyme®, Sanofi Genzyme)
2022 pabinafusp alfa (IZCARGO®, JCR Pharmaceuticals)
2018 cerliponase alfa (Brineura™, BioMarin)
2018 vestronidase alfa (MEPSEVII™, Ultragenyx)
2017 migalastat (Galafold™, Amicus)
2016 sebelipase alfa (Kanuma™, Alexion)
2015 eliglustat (Cerdelga™, Sanofi Genzyme)
2015 idursulfase beta (Hunterase®, GC Pharma)
2015 cysteamine bitartrate (PROCYSBI™, Horizon Therapeutics)
2015 elosulfase alfa (Vimizim®,  BioMarin)