Yesterday, an advisory panel of the U.S. Food and Drug Administration announced its verdict regarding Eteplirsen, an experimental drug used to treat a deadly degenerative disease that mostly afflicts boys.
Duchenne’s is a rare genetic disorder characterized by progressive muscular weakness. The disease is caused by a lack of dystrophin, a protein needed to keep muscles healthy. Eteplirsen is designed to increase the production of dystrophin.
The disease typically emerges in boyhood, causing weakness in the arms and legs and eventually the lungs and heart. Patients typically lose the ability to walk during adolescence and frequently die in their 20s or 30s, according to the National Institutes of Health.
Hundreds of patients and family members pleaded with the panel to approve the drug.
“FDA, please don’t let me die early,” urged 15-year-old Billy Ellsworth, who has the disease.
The panelists said they were moved by the patients’ testimony and that in some cases, it swayed their votes.
But all that pleading did not sway enough votes, as the panel voted against approving the drug.
The panel voted 7-3, with three abstentions, that the clinical trial of 12 patients did not provide substantial evidence the drug was effective for muscular dystrophy patients with a specific genetic mutation.
The vote followed a skeptical presentation by FDA reviewers, who questioned the validity of the data. They said the results were hard to interpret and there was no clear evidence the drug slowed progression of the disease…
The panel’s decision reflects the malign influence of the 1962 Kefauver-Harris Amendments which required drug companies to demonstrate not just safety, but also efficacy. Failure to demonstrate efficacy was the FDA panel’s justification for voting down the Duchenne’s drug this week.
The drug may or may not be effective, but we fail to see how patients who are facing death are better off not being able to try the drug at all. The decision to take this drug, it seems to us, should rest in the hands of the patients and their doctors, not panels of bureaucrats.
In any event, the panel’s vote was only ‘advisory’ and the FDA has not yet reached a final decision. Although it seems unlikely, the FDA might still be persuaded to approve the drug. Maybe the company that produces the drug just needs to make the right political contributions.
FDA delenda est