BioMarin Pharmaceutical Inc. announced that The WORLDSymposium 2018 awarded Brineura® (cerliponase alfa) the New Treatment Award, which recognizes important achievements in therapeutic advancements for lysosomal diseases. The award was presented on Monday, February 5.
“We are honored that Brineura has been recognized by the WORLDSymposium as the first approved therapy to treat a form of Batten disease,” said Hank Fuchs, M.D., President of Worldwide Research and Development at BioMarin. “The approval of Brineura was a direct reflection of an unwavering commitment and partnership between families, advocates and physicians. We share this award with those communities and look forward to continuing to provide our support to advance the care of children with CLN2 disease.”
Brineura is the only therapy approved by the U.S. Food and Drug Administration and European Union to treat children with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase 1 (TPP1) deficiency. In the U.S., Brineura is approved to slow the loss of ambulation in children over 3 with late infantile CLN2. CLN2 disease is an ultra-rare, rapidly progressive fatal brain condition, which affects an estimated 1,200 to 1,600 children worldwide. Affected children first show symptoms around the age of 3 and completely lose the ability to walk and talk around 6 years of age. During the later stages of the disease, feeding and tending to everyday needs become very difficult with death usually occurring between 8 and 12 years of age.
Brineura was also awarded the 2017 Popular Science “Best of What’s New” award in the health category. Each year, Popular Science reviews thousands of new products and innovations across 11 categories for its annual “Best of What’s New” issue, selecting those that represent a significant step forward in their category.
This is also the second time BioMarin has received the WORLDSymposium New Treatment Award. BioMarin received the award in 2015 for the first and only therapy approved to treat mucopolysaccharidosis type IVA, also known as Morquio A syndrome.