A few months ago, I went on a rant about the FDA’s failure to approve a drug that would benefit children with Duchenne muscular dystrophy, or DMD, a muscle-wasting disease.
What really made me angry was the fact the FDA had approved a drug where the patient population (world-wide) was 11 cases.
It made me wonder what was going on in their little cat brains when they denied approval of a drug that had the potential to keep kids out of wheelchairs before their 10th birthday.
I stand by my rant, but I’m happy to tell you the FDA has somewhat redeemed itself in my eyes by fast-tracking and approving a drug called Exondys 51 (eteplirsen) which is made by pharma company Serepta Therapeutics.
The drug’s specifically aimed at patients who have a confirmed mutation of the dystrophin gene.
There has been some controversy about this particular approval because of the heavy participation of DMD parents who put pressure on the federal agency.
Questions arose that perhaps the FDA had lowered its standard of scientific proof, but I can tell you this: If I was the parent of a child with DMD who was already losing muscle control by age three, I certainly would have been right there on the front line rattling my saber demanding approval for a drug that may help my child.
And, I’m pretty sure that would fall under the category of “good parenting.” Kudos to the real parents who helped make this drug approval happen!
