The review and subsequent approval of a controversial rare disease drug set off a debate in 2016 over whether the Food & Drug Administration has lowered its standards. In September, the agency went against the recommendation of its review panel and granted conditional approval to eteplirsen, a Duchenne muscular dystrophy treatment from Sarepta Therapeutics.
Eteplirsen has been closely watched for years as a test of the “accelerated approval” pathway, a mechanism that allows a drug for a serious, underserved disease to be marketed based on early signs of efficacy. Sarepta sought FDA’s stamp on the basis of a tiny trial—just 12 boys, all of whom received the drug—that yielded an even tinier amount of evidence that the drug is effective.
Sarepta’s drug works by enabling the production of dystrophin, a protein that cushions muscles from stress. But data presented to FDA’s advisory committee showed protein levels in boys taking the drug increased on average less than 1%. Families argued that their kids should have access to the drug while further studies are conducted.
Janet Woodcock, head of FDA’s Center for Drug Evaluation & Research, appeared to agree. Documents released by the agency show that she pushed the drug forward despite protests from colleagues. The decision unleashed a storm of criticisms, most centered on the worry that costly and marginally effective—or even ineffective—drugs now have a template for getting past the agency.
Even as FDA was accused of being more lenient, it was approving fewer new drugs than in recent years.
In the previous five years, new drug approvals had risen steadily, in part due to faster assessments of promising cancer and rare disease drugs. But as of Nov. 30, FDA had approved just 19 new molecular entities, including eteplirsen. In comparison, 39 new products had received the agency’s green light by the same time in 2015.
Although FDA tends to push through a spate of new drugs in the final weeks of the year, chances are slim that the industry will come anywhere near the approval peaks seen in the prior five years.