Genzyme licenses rights to genetic disorder drug

The deal with privately-held PTC gives Genzyme rights to market PTC124 outside of the United States and Canada. The compound is in late-stage development for a type of muscular dystrophy and for cystic fibrosis.

In addition to the $100 million upfront, PTC is eligible to receive up to $337 million in milestone payments and royalties on sales. PTC is responsible for funding current Phase IIb clinical trials, but the companies will split costs on further development.

"PTC124 is a powerful new approach that holds great potential to help cystic fibrosis and Duchenne muscular dystrophy patients and many others with a variety of devastating diseases," said Henri A. Termeer, Genzyme's chairman and chief executive, in a statement.

Genzyme said its experience with the drug Myozyme, which treats the genetic disorder Pompe disease, will be directly applicable to the deal for PTC124, since Myozyme patients are treated by the same specialist physicians. The company's best-selling drug is Cerezyme, a treatment for the genetic disorder Gaucher disease. Sales of that drug reached $304.3 million in the first quarter, followed by the kidney disease treatments Renagel and Renvela, with sales of $168.7 million.

Shares of Genzyme fell 9 cents to $78.88 in morning trading.