Johnson Statement on FDA Panel Decision to Not Recommend Potential Promising Therapies

WASHINGTON — On Monday, a Food and Drug Administration (FDA) advisory committee voted to not recommend approval of a potentially promising therapy for Duchenne muscular dystrophy. The committee voted to not approve, despite compelling testimony at its meeting from dozens of parents, medical experts and patients’ groups in favor of approving the therapy.

One of those parents who spoke was Laura McLinn, who testified earlier this year — along with her 6-year-old son, Jordan, who has Duchenne — before the Senate Homeland Security and Governmental Affairs Committee.

The Senate committee’s chairman, Sen. Ron Johnson (R-Wis.), had this to say about the FDA advisory committee’s decision:

“I was disappointed in the results of the vote by the FDA committee, as Duchenne is a severely debilitating and very often fatal disease for which there is no available cure or effective therapy. The final decision is now up the FDA itself. Dr. Janet Woodcock, who is in charge of the drug division, was quoted as saying the agency has ‘flexibility and that’s where we should take the views of the community into account.’ I could not agree more. I encouraged her to use that flexibility and deference to patients’ views in a letter I sent to Dr. Woodcock last month, co-signed by three of my Senate colleagues.

“I’ve also asked the FDA about its failure to streamline the expanded access program’s application process. This program gives patients access to investigational products for life-threatening conditions where no alternative exists. It’s been more than a year since the agency announced it would streamline the process. But so far, my inquiry from February 19, 2016, has gone unanswered.

“The cost of delays in approving or granting access to therapies is paid in human lives. I urge the FDA to consider those lives when making final decisions on this and all potential life-saving drugs.  It is time to give these patients hope.”