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news.Switzerland-based pharmaceutical company Vivendy Therapeutics has received the FDA orphan drug designation for its enzyme replacement therapy for mucopolysaccharidosis IVA.
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This designation, following potential marketing approval in the future will give Vivendy seven years of market exclusivity and could facilitate the recovery of certain regulatory filing fees.
Vivendy’s seeks to replace the lack of or deficient activity of the N-acetylgalactosamine-6- sulfatase (GALNS) enzyme in mucopolysaccharidosis IVA (MPS IVA- Morbus Morquio) by administering a recombinant human GALNS enzyme that has been specifically modified – potentially enhancing the efficacy of the therapy in MPS IVA significantly.
Compared to the native enzyme, Vivendy believes that the modification has the potential to maximize the delivery of the enzyme to efficiently clear the storage materials in target tissues and organs.
Roland Toder, Vivendy’s CEO, said: “This approval is an important regulatory step in the development of our unique modified enzyme treatment for Morquio patients. We look forward to a timely execution of our development program.”