New Treatment Awards

In recognition of achieving major milestones, WORLDSymposium honors “new treatments” that were 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. Two new treatments were recognized with the WORLDSymposium 2018 New Treatment Award:

Cerliponase alfa (Brineura™, BioMarin)
On April 27, 2017, cerliponase alfa (Brineura™, BioMarin) became the first approved treatment for CLN2, (a form of Batten disease), by the U.S. Food and Drug Administration. It is indicated to slow the loss of ambulation in symptomatic pediatric patients 3 years of age and older, with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase 1 (TPP1) deficiency. CLN2 disease is an ultra-rare and rapidly progressing brain disorder that affects an estimated 20 children born in the United States each year – less than one in a million Americans. Notably, cerliponase alfa is the first enzyme replacement therapy to be directly administered into the cerebrospinal fluid of the central nervous system. It is aimed at treating the underlying cause of CLN2 disease by replacing the deficient TPP1 enzyme missing in affected children.

Vestronidase alfa (MEPSEVII™, Ultragenyx)
On November 15, 2017, the U.S. Food and Drug Administration approved vestronidase alfa (MEPSEVII™, Ultragenyx) to treat pediatric and adult patients affected with mucopolysaccharidosis type VII (MPS VII), also known as Sly syndrome. MPS VII is an extremely rare, progressive condition that affects most tissues and organs.

WORLDSymposium presented the New Treatment Awards on Monday, February 5, 2018, at 5:15 PM in the Harbor Foyer, followed by the Opening Reception in the Exhibit Hall.