FDA panel votes down new muscular dystrophy drug

On Monday, the Food and Drug Administration's Peripheral and Central Nervous System Drugs advisory committee determined there was not significant evidence to suggest that Sarepta Therapeutics' new drug for treating Duchenne muscular dystrophy is effective, according to a report from STAT.

The central question was whether the drug, called eteplirsen, can produce high levels of protein call dystrophin. Though some agreed that the drug does produce this essential protein lacking in those with DMD, the panel felt the drug failed to display clinical efficacy. The vote came back seven to six with three abstentions. Likely at issue was the small size of the trial — typically, smaller trials are met with skepticism by the FDA, according to STAT.

The door is not closed on approval, as the FDA has until May 26 to decide whether to follow the recommendation of the panel.

While some are skeptical about approval, others believe the emotional nature of the testimonies given at the hearing could spur ultimate approval by the FDA. Fifty-two testimonies were given in favor of the drug, and they sometimes got emotional, especially when children with DMD and their families spoke, according to STAT.

Steven Brozak, who runs WBB Securities and tracks biotech stocks, told STAT, "Everyone was visibly moved by the audience and their testimony, and that is not going to go unnoticed...it's not just political pressure. It's advocacy pressure of the sort that I've not seen since the days of HIV and AIDS. And that can make the difference here."

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