Do gene therapies, marketed for millions of dollars, and exon-skipping therapies administered monthly and sold for thousands of dollars, actually work well? Families campaigning under intense stress to access the therapies hope that DMD therapies will positively impact their children’s lives, but the data is less promising.
It might have been the end of the road for the medications when Sarepta Therapeutics announced this month that a confirmatory trial for two of its marketed exon skipping drugs for Duchenne muscular dystrophy had not met its primary endpoint. >> Sarepta’s DMD Exon-Skipping Therapies Fail Confirmatory Study, Stock Price Down
Leaders of the company, however, were unwilling to let the spirit go. On the basis of additional clinically significant changes observed in the research participants, who range in age from 6 to 13, they intend to meet with the FDA to talk about “converting from an accelerated to traditional approval.”
A failed trial is not always a death sentence, and Sarepta is not the first business to realize this; in fact, it has already saved one medication from certain death. Despite a late-stage trial failing to meet the primary endpoint, Elevidys, its DMD gene treatment, was fully approved and given a label expansion. >> Why Elevidys Was Not Approved by the European Medicines Agency (EMA)?
This adaptable strategy has its detractors. According to some experts, the company should indicate a hard halt when a medication trial fails.
“There’s no wiggle room in my mind,” said Dr. Joseph Ross, professor of medicine and public health at the Yale School of Medicine. “I cannot understand how a trial that’s designed to demonstrate whether a drug works or not, if it fails, how a drug could then get approved for use. Because what was the point of the trial?”
Trials are already designed to give products the best possible chance of demonstrating a benefit.
“And so if it’s null, it’s null,” he said.
Accelerated Approval Pathway
The FDA has approved scores of subpar medications in recent years. According to a research Ross co-authored, between 2018 and 2021, over 10% of all approvals involved a medicine that failed to meet at least one primary endpoint in a pivotal trial. >> Read The Article
When a drug is approved without clear evidence of a benefit, it raises several risks around safety and costs.
“Some of these very expensive gene therapies, biologics and other specialty drugs can bankrupt families,” he said. The broader public also shoulders some of the financial burden with higher insurance premiums and other costs.
There need to be more stringent FDA standards to protect patients, he said.
“My general feeling has been, if we as a society are going to allow products to get approved with less certain evidence so they can to reach patients sooner, we need to do a better job of ensuring that companies are completing the confirmatory trials in the post-market period within a reasonable period of time, so we know they work and that patients aren’t wasting their money,” Ross said.
Follow This Page >>> All Clinical Trials for Duchenne
