After a months-long review process that sparked a heated internal debate, the Food and Drug Administration Monday approved eteplirsen as the first treatment for Duchenne muscular dystrophy, a rare disease that causes muscle weakness, loss of movement and eventually death, reported the Chicago Tribune.
The approval set a new precedent for the development and regulation of drugs for rare diseases, the merit of which is also up for debate.
While FDA staff who reviewed the drug's application opposed approval saying there was no proof of the drug's efficacy, Janet Woodcock, MD, director of the Center for Drug Evaluation and Research, overruled the group. The agency approved eteplirsen under an accelerated approval program that requires Sarepta to conduct another clinical trial to validate the drug's efficacy. The move followed an intense and emotional campaignfrom patients, parents and physicians calling for the drug's approval.
"It sets a precedent for patient advocate involvement and potential power; it sets a precedent for the degree of flexibility that FDA can show if they want to," said Wall Street analyst Ritu Baral. She believes the decision makes rare disease drug development more unpredictable, the report states.
In contrast, Ellis Unger, MD, the FDA executive who led the drug's review process said, "this decision could be precedent-setting," as it lowers the standard for effectiveness "to an unprecedented nadir." He said it could also be the first time a center director overruled a review team and an outside advisory committee on the issue of whether a drug's effectiveness was demonstrated, according to the report.
FDA Commissioner Robert Califf, MD, said the industry should not read too much into the decision.
"Our understanding about how to include patients in the regulatory process is evolving," he said in a statement. "Serious shortcomings in the eteplirsen development program should not be allowed to establish broad precedent for therapeutic development in rare diseases."
More articles on supply chain:
Mylan CEO acknowledges company could have anticipated consumer concern over price hike
Senate committee to examine Mylan's Medicaid rebates
Active ingredient shortage puts Bristol-Myers Squibb cancer drug on backorder