I'm just a pill. Yes, I'm just a pill. And I'm sitting here ...
Oh, hi. My name is Nupil. I'm a new drug, or at least I hope to be. Right now, the FDA is deciding whether to approve me. See that big office building? That's the Center for Drug Evaluation and Research. It's as important as it sounds. The fate of all new medicines that want to be sold in the U.S. is decided here.
By Laura Beil
Christen Childs woke up on September 12, 2009, in the pitch dark of early morning with what she thought was a pulled muscle in her leg. She reached down to massage the cramp, trying to fathom how her left calf could be so achingly sore when she hadn't made it to the gym in weeks. This was a Saturday — by Monday, her leg was swollen and hot, and when she tried to stand, jolts of pain shot up to her spine. She consulted her brother-in-law, a doctor, and he told her to go to the ER immediately...
Inside, FDA reviewers are carefully examining all the information that's known about me and talking it over together. They sure are busy. There are more than 100,000 pages of data, and it will take a team of reviewers several months to review. I guess I'll just have to sit here and be patient.
How did I end up here? Why, I'm glad you asked. That's an interesting story.
A Molecule Stands Out
About 12 years ago, I started out as a molecule, one of thousands researchers created in a laboratory. The scientists screened us, one by one, looking for some special properties. I was added to some cells in a test tube to see what I would do.
It was a long time ago, but I remember I liked almost everything about those cells, except for one awful little enzyme -- an enzyme that could make people sick. That enzyme really annoyed me, so I blocked its production, but left everything else alone. Well, the scientists were plenty pleased. I only did what came naturally to me, but now I know it was exactly what they were hoping for.
I didn't have a name yet, just a number: ABCD-523.
The Testing Begins
The scientists then started testing me in laboratory rats. The purpose of this was to see if I did the same thing in live animals that I did in the test tube. They also wanted to know if I had any toxic effects. They measured how I was absorbed and passed though the animal's body.
As Alan Goldhammer, PhD, associate vice president of regulatory affairs for the Pharmaceutical Research and Manufacturers of America (PhRMA), told me, "It's easy to identify lots of things that work inside a test tube." The challenge is finding something that works in a living body.
The results of the experiments were good. It's pretty rare for that to happen. Only one in 50 promising compounds will pass these tests. The vast majority don't work as expected, or they prove to be too toxic.
Meanwhile, researchers studied how I could be made into a pill. They wanted to make sure I wasn't too fragile -- that I could exist within a wide range of temperatures without degrading. They also looked at how difficult it would be to manufacture me on a large scale. It seems I'm not fussy about the weather, and I'm not impractical to make in bulk.