Wednesday, December 17, 2008

Court: Company doesn't have to provide teen with MD with experimental drug

From The AP:

WASHINGTON — A pharmaceutical company does not have to provide an experimental drug to a Minnesota teen who is terminally ill with a rare form of muscular dystrophy, a federal appeals court ruled Dec. 16 in reversing a lower court decision.

The ruling by the U.S. Court of Appeals for the Third Circuit in Philadelphia was a blow to 17-year-old Jacob Gunvalson, who suffers from Duchenne muscular dystrophy.

The court ruled that U.S. District Judge William J. Martini in Newark erred in his August ruling that PTC Therapeutics of South Plainfield, N.J., must provide the drug to Gunvalson. That decision had been stayed pending the company's appeal.

"I just think it's really unfair that these drug companies get all these benefits from the federal government," said Jacob's mother, Cheri Gunvalson. "And then they're allowing boys to fall through the cracks and die." She said she would not give up her fight but didn't know what the next step would be.

In its ruling, the appeals court said it was "sympathetic to the plight of Jacob and his family," but that the lower court "abused its discretion" in ordering PTC to supply the drug to Gunvalson.

The Gunvalsons, who live in Gonvick, Minn., maintained that the company led them to believe that Jacob could participate in a clinical trial of the drug, which is being investigated as a possible treatment — and that the company then went back on its word.

The company countered that no promises were made, and that allowing Jacob to join the clinical trial would not be safe and would set a bad precedent that could hinder research.

In a statement, PTC president and CEO Stuart Peltz called the ruling "important not just for PTC124, but for the future of the clinical trial process for all experimental drugs for rare disease.

Former Minnesota Attorney General Mike Hatch, who is representing the Gunvalsons at no charge, called the ruling "a surprise, a huge disappointment, a real tragedy."

He said the legal team was weighing its options, including a request for a rehearing or pursuing a trial at the district court level.

Duchenne muscular dystrophy is a genetic, degenerative disease that mostly affects young boys. Typically, those who suffer from it die in their 20s because of weakness in their heart and lung muscles. There is no known cure.