The Biotech Scramble for Big Money in Muscular Dystrophy Drugs

Politicians and media observers are focusing on the spectacular prices some biotech companies seek to charge for drugs for rare diseases. The hunt for a treatment for the deadliest strain of muscular dystrophy is likely to return the issue to the spotlight and raise questions about how regulators evaluate cutting-edge drugs.

Three companies are competing for Food and Drug Administration approval of medicines for Duchenne, a disease that destroys muscle, robs victims of the ability to walk, causes respiratory complications, and typically leads to death by the mid-20s. One in 3,500 newborns has Duchenne, almost all of them boys. There's no cure. An FDA-approved Duchenne treatment would likely carry a price tag of $300,000 a year or more, making the drug a billion-dollar bonanza for its manufacturer.