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 2024. 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 2024 WORLDSymposium New Treatment Awards was presented to 3 outstanding new therapies:

Cipaglucosidase alfa (Pombiliti®), an enzyme replacement therapy (ERT) designed to deliver active GAA enzyme directly to the muscle cells for effective uptake, and miglustat (Opfolda®), the first and only oral enzyme stabilizer designed for late-onset Pompe disease (LOPD). This two-component therapy provided clinical data meriting approval by the European Commission (EC), the Medicines and Healthcare products Regulatory Agency (MHRA) of the United Kingdom (U.K.), and the U.S. Food and Drug Administration (FDA) as the first for adults with late-onset Pompe disease (LOPD). (Amicus Therapeutics)

Pegunigalsidase alfa-iwxj (Elfabrio®) which provided clinical data meriting approval by the European Commission (EC) and the U.S. Food and Drug Administration (FDA) as an enzyme replacement therapy (ERT) that has been shown to work safely and effectively in adults with Fabry disease. (Chiesi Global Rare Diseases)

Velmanase alfa-tycv (Lamzede®) which provided clinical data meriting approval by the U.S. Food and Drug Administration (FDA) as the first U.S. enzyme replacement therapy (ERT) approved for the treatment of non-central nervous system manifestations of alpha-mannosidosis (AM) in adult and pediatric patients. (Chiesi Global Rare Diseases)

The WORLDSymposium 2024 New Treatment Awards was presented on Thursday, February 8, 2024, at 7:45 AM PST, followed by a full day of Contemporary Forum abstract presentations.