Saturday, September 20, 2008

Court says teen with muscular dystrophy must wait for decision about experimental drug

From The AP:


NEWARK, N.J. - A Minnesota teenager suffering from a rare form of muscular dystrophy will have to wait to learn if he can get access to an experimental drug.

In a ruling made public Sept. 18, a federal judge in Philadelphia granted a stay to South Plainfield-based PTC Therapeutics. That means the company won't have to provide the drug to 16-year-old Jacob Gunvalson while it appeals an earlier court ruling.

Last month, a federal judge ordered PTC to supply the drug to Gunvalson, who suffers from Duchenne muscular dystrophy, a terminal illness. PTC appealed the ruling, and the two sides have until early November to file briefs. 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. Gunvalson uses a wheelchair because the disease has left him unable to walk.

Gunvalson's family had sued PTC, claiming the company led them to believe Jacob could participate in a clinical trial featuring the experimental drug PTC124, then went back on its word. PTC said no promises were made and that allowing the teen to join the clinical trial would be unsafe and would set a bad precedent.

The clinical trial of PTC124 included a 28-day preliminary phase in 2005 and a 96-week phase that is currently under way. Jacob was excluded from the preliminary trial because of medication he was taking at the time, both sides agree.

But the Gunvalsons claim they were assured by PTC employees that he could
still participate in the 96-week trial. They later discovered he could not because he hadn't participated in the preliminary trial.

U.S. District Judge William J. Martini's Aug. 20 ruling raised the possibility that Jacob could receive the drug from his personal physician, provided the physician passed an FDA approval process. That idea was opposed by PTC officials who said granting exceptions to individual patients through litigation would compromise the clinical trial process and delay the approval of drugs.

In January, the U.S. Supreme Court refused to review a lower court ruling that terminally ill patients have no constitutional right to be treated with experimental drugs _ even if that means the patient will likely die before the medicine is approved.

An attorney representing the Gunvalsons did not return messages Friday. John Hutchinson, an attorney representing PTC Therapeutics, declined to comment
on the matter.