FDA Delays Threaten Kid’s Life; Parents Plead With White House

Austin Leclaire, 15, and his brother
Max, 12, of Vermont, both suffer from a rare form of muscular
dystrophy that leaves them with a life expectancy of 25 years. In
2012, Max began showing signs of improvement while participating in
a clinical study with a drug called eteplirsen. Austin didn’t
qualify for the study, because he is
wheelchair-bound. His condition has continually
deteriorated: He can barely get himself out of bed or unlock a
door. Making matters worse, the Food and Drug Administration (FDA)
is now effectively blocking Austin’s ability to access the drug by
delaying its release. So, the Leclaire boys’ parents are
petitioning the White House.

The Boston Herald
reports:

Cambridge-based Sarepta Therapeutics has been testing its
[Duchemme Muscular Dystrophy] drug in 12 boys for more than two
years and says the results are good, but the FDA has stalled
approval until it sees results from a larger trial.

[…]

Sarepta spokesman Jim Baker said the company plans to launch a
new, larger drug study later this year, with input from the
FDA.

The process could take years.

But, “There is no reason in the world for this drug to not be
approved. It’s 100 percent safe,” Jenn McNary, mother of the
Leclaire boys, told the Herald. She isn’t alone:

Dr. Jerry Mendell of Nationwide Children’s Hospital in Columbus,
Ohio, who designed the Sarepta drug trial, said eteplirsen is a
first-of-its-kind therapy and has shown “superb” results so
far.

“I think the product is very good, and I think it deserves to be
approved. … It has absolutely no side effects,” he said.

Frustrated by the bureaucratic delays, the Leclaire family
teamed up with a campaign called “The Race To Say Yes.” They
filed a “We the People” petition at WhiteHouse.gov late in
February,
hoping to get at least 100,000 signatures in favor of
accelerating the approval process for the drug. They met that mark
this past Saturday.

Unfortunately, as the Herald notes, the White House has
not yet replied.

For more Reason coverage of the FDA stifling progress in
medicine, click
here or watch the video below: