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FDA Designates Orphan Drug Made From Tarantula Venom

Frederick Sachs and tarantula (University at Buffalo)

Frederick Sachs (University at Buffalo)

The U.S. Food and Drug Administration (FDA) has designated the peptide GsMTx4, made by Rose Pharmaceuticals of Buffalo, New York, as an orphan drug for Duchenne muscular dystrophy. Rose Pharmaceuticals is a one-year old company founded by University at Buffalo (UB) medical professor Frederick Sachs and Buffalo stockbroker Jeff Harvey.

While Sachs (pictured left) applied his research interests to develop GsMTx4, Harvey’s interest was more personal. His grandson, now 3 years old, has Duchenne muscular dystrophy (DMD), a fatal affliction that causes patients’ muscles to waste away.

DMD is one of a group of muscular dystrophies characterized by the enlargement of muscles. DMD is one of the most prevalent types of muscular dystrophy and is characterized by rapid progression of muscle degeneration that occurs early in life. The disease affects mainly males, an estimated 1 in 3,500 boys worldwide

GsMTx4 is a peptide found in tarantula venom. Research by Sachs and fellow UB biophysicist Thomas Suchyna found the peptide had potential against DMD, but could not find an established pharmaceutical company to commercialize it into a drug. Last year Sachs and Harvey founded Rose as a biotech company to develop the drug, soon joined by Suchyna.

The FDA’s orphan drug designation applies to drugs and biologics intended for the diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S. The status qualifies Rose for tax credits and other incentives that could help the firm move the product to the market more quickly.

Related: FDA Gives Muscular Dystrophy Therapy Orphan Drug Status

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