After (mostly) skimming those "timeless raindrops, under the rocks from the basement of time"* -- on the beds of my icy rivers silently for lo these last four or five years, Salmon re-surfaced, arching from the golden flecked ripples, this week to mention a biologic called Exondys 51 -- and point us toward it, as part of our new non-Merck (did I say "new"?) coverage. Here is FierceBiotech on it -- but we will await Salmon's definitive take:
. . . .John Jenkins, who runs the FDA’s office for new meds, has not taken kindly to the regulator’s recent, and highly controversial, approval of Sarepta’s Duchenne med Exondys 51 (eteplirsen) after hitting out at bad trials with questionable data.
In a presentation given this week at the NORD summit in Arlington, called “Lessons learned from eteplirsen and other recent rare disease programs,” Jenkins spoke specifically of the biotech’s drug, saying: the “path taken by Sarepta NOT a good model for other development programs.”
A month ago, the FDA approved Exondys 51 against all odds. The approval was considered unlikely because many of the data used to back a speedy approval were based largely on a small study of a dozen children with no placebo control, comparing eteplirsen’s results against historical data in the muscle-wasting disease.
Back in April, an FDA panel of outside experts voted narrowly, 7 to 6, that Sarepta did not provide substantial evidence from “adequate and well-controlled” studies that the drug produced dystrophin at a level that was reasonably likely to produce a clinical benefit. . . .
And there you have it: an approval of -- at least arguably -- a poorly understood biologic. You are up, in comments, Dr. Salmon. Do hit it out of the park for us -- this is one fat hangin' juicy curve-ball, headed right out to the middle of Waveland Avenue. Smile.
* Apologies there -- to the immortal prose of one Norman MacClean.