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CepTor Receives Dual Orphan Drug Designation for Myodur (C101) for |
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26 Jan 2006 |
Hunt Valley, MD. US | Jan. 25, 2006 | CepTor Corporation (OTC BB: CEPO), a development-stage biopharmaceutical company focusing on cell-targeted therapeutic Products for neuromuscular and neurodegenerative diseases, announced that the Office of Orphan Products Development of the Food and Drug Administration (FDA) granted orphan drug designation for Myodur for both Duchenne and Becker muscular dystrophies.
The Orphan Drug
Act (ODA) of 1983 provides for the granting of orphan designation for products that address serious or life threatening disease that affect less than 200,000 people in the U.S. and where no other definitive therapy is currently available. The spirit of the ODA legislation is to provide incentives to promote research on orphan diseases. As such, compounds, like Myodur, that are granted orphan designation, provide their manufacturers tax breaks, potential regulatory expediency including accelerated reviews and seven years of market exclusivity after commercial approval. CepTor recently announced the submission of its Investigational New Drug application (IND) to initiate phase I/II clinical trials with Myodur in Duchenne boys. Duchenne is an X-linked defect of the dystrophin gene that affects about 25,000 boys in the U.S and EU. This devastating disease normally results in the need for walking braces by about ages 6-8, wheelchair dependence by ages 10-13 and death in late adolescence. Becker is a less aggressive form of the disease that affects adults and is believed to be about 10-15% as large as the Duchenne population. Earlier published work suggests a cause and effect relationship between abnormally high up regulation of calpain and these muscle wasting diseases. Calpain is a protease which causes the accelerated destruction of muscle tissue. CepTor’s Myodur is a muscle cell targeted product that inhibits calpain in an effort to preserve muscle tissue.
William Pursley, CepTor’s Chairman and CEO, said, “The orphan drug office reviewed our application quickly and did the right thing by granting dual orphan designation since Duchenne and Becker are caused by defects in the same gene. We believe this is an important milestone for several reasons. Indirectly, it provides a strong extension of the intellectual property around our technology. The competition free environment that the market exclusivity portends is an obvious advantage. Most importantly, it creates a significant opportunity to work in partnership with the FDA in an expedited manner to address this devastating disease.”
SOURCE: Ceptor Corporation |
