I often equate this process of getting Trofinetide to market to baseball. The teams, the hits, the homers, the errors, the running of the bases-this is a metaphor that I find very appropriate. And now, given the incredible, almost unbelievable win by the Cubs in a Series that gave its all-seven games, going into extra innings in the last game, I get to continue the metaphor!
The Pediatric Trial (technically Game 3 of this series) went into extra innings. Enrollment was so high and quick that the original target of 64 was blown out of the water with a total of 82 being the final count, with one withdrawing. And, now we are coming down to the wire. Neuren has announced that the trial will complete in January with results out in March. I’m a bit jealous because those of us with children who participated in the adult trial had to wait until APRIL ;).
So, will we win? Will these extra innings be enough for the FDA to just go ahead and approve Trofinetide? It sure would be awesome. We might have to Game 4 to win it all. But, wouldn’t it be great if, just this once, a series sweep could be won in three.
And now we wait for March and collectively sit in the stands, hands clasped close to our mouths, much like our girls and boys, knees going up and down as we wait for that last batter to see if our team wins it all.
Thank you to the parents and girls who braved the unknown for the sake of all the girls and boys suffering and waiting for this treatment. You have all been heroes on the field.
See Neuren’s announcement, here.