Hopes high for muscular dystrophy drug
A new drug designed to slow the progression of Duchenne muscular dystrophy, a devastating disease that overwhelmingly affects boys and typically leaves them needing wheelchairs by their teens and dead in their 20s, has received a long-awaited boost from the Food and Drug Administration.
Massachusetts-based Sarepta Therapeutics said Monday that FDA officials detailed a potential path forward for the drug, eteplirsen, and indicated a willingness to consider it for accelerated approval. The news puts eteplirsen one step closer to becoming the first approved treatment for Duchenne, a disease that affects about one in 3,500 boys globally.
“This provides the opportunity to get the drug approved and in the hands of all the boys who can benefit from it sometime in 2015,” Sarepta chief executive Chris Garabedian said. “I think that's a huge win for the [Duchenne] community and for Sarepta.”
The move follows a tumultuous year in which the FDA initially appeared headed toward swift approval of eteplirsen based on promising results from a trial involving 12 young boys, whose muscular deterioration seemed virtually halted by the drug. But the agency hit the pause button in November, saying it would be “premature” for Sarepta to seek approval of its drug with the failure of a separate trial involving a different Duchenne treatment that used similar technology.
Months of behind-the-scenes deliberations followed over the effectiveness of eteplirsen, as well as an aggressive public campaign waged by patients' families determined to keep pressure on the agency.
Show commenting policy
TribLive commenting policy
You are solely responsible for your comments and by using TribLive.com you agree to our Terms of Service.
We moderate comments. Our goal is to provide substantive commentary for a general readership. By screening submissions, we provide a space where readers can share intelligent and informed commentary that enhances the quality of our news and information.
While most comments will be posted if they are on-topic and not abusive, moderating decisions are subjective. We will make them as carefully and consistently as we can. Because of the volume of reader comments, we cannot review individual moderation decisions with readers.
We value thoughtful comments representing a range of views that make their point quickly and politely. We make an effort to protect discussions from repeated comments either by the same reader or different readers.
We follow the same standards for taste as the daily newspaper. A few things we won't tolerate: personal attacks, obscenity, vulgarity, profanity (including expletives and letters followed by dashes), commercial promotion, impersonations, incoherence, proselytizing and SHOUTING. Don't include URLs to Web sites.
We do not edit comments. They are either approved or deleted. We reserve the right to edit a comment that is quoted or excerpted in an article. In this case, we may fix spelling and punctuation.
We welcome strong opinions and criticism of our work, but we don't want comments to become bogged down with discussions of our policies and we will moderate accordingly.
We appreciate it when readers and people quoted in articles or blog posts point out errors of fact or emphasis and will investigate all assertions. But these suggestions should be sent via e-mail. To avoid distracting other readers, we won't publish comments that suggest a correction. Instead, corrections will be made in a blog post or in an article.
- Family of woman found dead in Texas jail files wrongful death suit
- GOP hopefuls take on illegal immigration in debate preview
- Manhunt under way for suspect in Memphis officer’s killing
- Milwaukee archdiocese wants to pay 300 abuse victims $21M; it’s not enough, some say
- Veterans notified of info breach in South Dakota
- Obama orders steeper emission cuts from power plants
- Hurricane Guillermo downgraded to tropical storm
- Medicare patients’ outcomes improve
- West Virginia on pace to issue record number of concealed-carry permits