Creating a New Therapy (IND Submission)

At MDA, we take a big picture perspective across the full spectrum of neuromuscular diseases to uncover scientific and medical breakthroughs that accelerate treatments and cures. The power in our research approach is that we can often apply learnings from one disease to progress in others to bring urgently-needed answers to our families.

IND Submission: Asking for Permission

At the end of all of this work, the developers think that the drug is ready to be tested in humans.

They can make it in a uniform manner in a form that can be given to people. They know a range of doses that are predicted to be safe in humans, and know that there are doses in this range that would be expected to have a therapeutic effect. They know how to administer the drug to participants and what markers to look for to see if it is having an effect. They have a feasible trial design, patients who want to take part and clinicians willing to run the trial.

At this point, the developer has the data that is needed to ask the FDA for approval to move forward.

If all this data is approved by the FDA, the drug will be allowed to start trials. That’s when it gets exciting — what happens to the drug in a human being? Will it work?

Next: Clinical Trial