Vivendy's Treatment For Morbus Morquio (MPS IVA) Granted Orphan Drug Designation By FDA

Main Category: Bones / Orthopedics
Article Date: 18 Sep 2008 - 3:00 PDT

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Vivendy Therapeutics Ltd. today announced that its enzyme replacement therapy (ERT) for Mucopolysaccharidosis IVA, (MPS IVA-Morbus Morquio) has been granted Orphan Drug designation by the United States Food and Drug Administration (FDA). 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.

"This approval is an important regulatory step in the development of our unique modified enzyme treatment for Morquio patients", said Dr. Roland Toder, Vivendy's Chief Executive Officer, "We look forward to a timely execution of our development program".

About Vivendy's Enzyme Replacement Therapy (ERT)

Vivendy's seeks to replace the lack of or deficient activity of the N-acetylgalactosamine-6- sulfatase (GALNS) enzyme in MPS IVA 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.

About Mucopolysaccharidosis (MPS) IVA

Mucopolysaccharidosis (MPS) IVA (MPS IVA, also known as Morbus Morquio A) is a rare lysosomal storage disease characterized by a gene mutation that causes a lack or deficient activity of the N-acetylgalactosamine-6- sulfatase (GALNS) enzyme. This in turn causes excessive lysosomal storage of keratin sulfate (KS) and Chondroitin-6-Sulfate (C6S) which leads to multiple systemic skeletal, spine and joint abnormalities as well as malformations of the chest. Additionally, patients may suffer hearing loss, vision impairment, and heart valve disease. Accurate epidemiological data regarding the rate of incidence of MPS IVA is only sporadically available, but estimates vary between 1 in 250,000 live births to 1 in 500,000 live births.

About Vivendy Therapeutics Ltd.

Vivendy Therapeutics Ltd., a pharmaceutical company developing an enzyme replacement therapy (ERT) for Mucopolysaccharidosis (MPS) IVA (Morbus Morquio) was founded in March 2006, as a spin-off of Inotech Biotechnologies AG. The company's mission is the development of an enzyme replacement therapy (ERT) for Morbus Morquio. In June, 2008, Saint Louis University (SLU), Office of Innovation and Intellectual Property, St. Louis, Missouri, USA and Vivendy Therapeutics Ltd., finalized and signed a License Agreement where SLU granted to Vivendy Therapeutics the exclusive license to use its patents for the research, development, manufacturing, marketing, sales and other use of the licensed products in the field of Enzyme Replacement Therapy (ERT) compositions intended for the treatment of patients having MPS IVA (Morbus Morquio). http://www.vivendy.ch

Disclaimer

This press release does not constitute or form part, or all, of any offer or invitation to sell or issue, or any solicitation of any offer to purchase or subscribe for, any securities, nor shall part, or all, of these materials or their distribution from the basis of, or be relied on in connection with, any contract or investment decision in relation to any securities. This press release contains forward-looking statements based on the currently held beliefs and assumptions of the management of Vivendy, which are expressed in good faith and, in their opinion, reasonable. Forward-looking statements involve known and unknown risks, uncertainties and other factors, which may cause the actual results, to differ materially from the results, financial condition, performance or achievements expressed or implied by such forward-looking statements. Given these risks, uncertainties and other factors, recipients of this document are cautioned not to place undue reliance on these forward-looking statements. Vivendy disclaims any obligation to update these forward-looking statements to reflect future events or developments.

Vivendy Therapeutics Ltd.

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