Two Republican senators want the Food and Drug Administration to approve a new drug to treat a rare, fatal disease despite concerns about whether it actually works.
Sens. Ron Johnson, R-Wis., and Dan Coats, R-Ind., are disappointed that a panel of experts did not recommend a new drug called Sarepta for Duchenne muscular dystrophy, a fatal disease that mainly affects young boys. The senators’ call follows a massive online grassroots effort to get the agency to approve the drug despite questions about its safety.
“Parents are crying out for the FDA to hear them: They are engaged and knowledgeable and only want the agency to do what is already within its power,” according to the letter released Monday.
The senators said the letter isn’t limited to “one disease or one drug, though there is no less urgency with respect to the case of Duchenne muscular dystrophy.”
An FDA advisory committee voted against the Duchenne drug made by the small biotech firm Sarepta last month due to concerns about its effectiveness.
The FDA does not have to abide by the decisions of its advisory panels, but it usually does.
It isn’t uncommon for lawmakers to wade into drug approvals. Back in 2013, several lawmakers bashed the FDA for approving a powerful opioid called Zohydro without abuse-deterrent technology.
Several lawmakers on both sides of the aisle called for the agency to pull its approval for the drug, which the agency declined to do.
