FDA Rejects Drug For Duchenne Muscular Dystrophy

The maker of a drug designed to help treat a terminal muscular disease is appealing the FDA's decision to reject the medicine.

Duchenne muscular dsytrophy causes the muscles of young boys to degenerate.

Most don't live past their mid-twenties.

A Lewisville 15-year-old has been in the drug trial for years, and his family says they'll keep fighting for a treatment.

Unlike many pateints his age, Matthew Wood can run, walk, bathe himself and even jump."

"I feel good," he says. 

He's spent four years in the clinical drug trials for Ataluren, a protein restoration therapy designed to enable the formation of the protein that's missing in the body of duchenne muscular dystropy patients.

"Anything that he can do prior to the medicine, he can still do now. There's nothing that he's lost. And that is unheard of," says Matthew's mother Teresa.

She had hoped the FDA would go against the recommendations listed by an FDA committee last month, which said enough evidence doesn't exist to support recommending the drug's approval.

This week, the FDA followed the recommendations.

In the FDA reponse letter to the drug maker, PTC Therapeutics, the FDA said "evidence of effectiveness from an additional adequate and well controlled clinlical trial will be necesary at a minimum to provide substatnial evidence of effectiveness."

"If we feel that it's something that is working and is safe, then let us have it," says Wood.

In a statement from the drugmaker: “We are extremely disappointed for the Duchenne community and strongly disagree with the agency’s conclusions,” said Stuart W. Peltz, Ph.D., chief executive officer of PTC Therapeutics. “We believe that this decision fails to consider the benefit-risk of ataluren and the high unmet medical need. Therefore, we plan to file a formal dispute resolution request next week.”

Teresa and Matthew won't give up hope.

"This disease is not something that you can sit and wait. This is a disease that everyday, things are happening to their body and their muscles are wasting and they're going to lose abilities that they're never going to get back," says Wood.

They say they will be able to continue receiving the drug through the appeal process, would the drugmaker advised them could take a month.

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