Vtesse.

When, less than 2 years later, Vtesse had moved the drug into the new, pivotal trial, the bench-to-bedside center pointed to the drug’s quick progress as a “huge win” for patients—an example of how the U.S. biomedical agency can develop drugs in their earliest stages to the point where industry takes them over.

Then in March 2017, privately held Vtesse was acquired for $200 million by the global biopharmaceutical company Sucampo Pharmaceuticals.

It was shepherded through early development by an NCATS-orchestrated collaboration and then handed off to the small Gaithersburg, Maryland, company, Vtesse.

Some parents and scientists charge that CTD is promising brain improvements its drug cannot deliver and siphoning rare patients who might otherwise enroll in the Vtesse clinical trial.

"If you are going to try to start selling parents on 'You don't need to do intrathecal,' you are putting children at risk in my opinion. I got very upset," says Philip Marella, who lost his daughter at 19 to the disease and whose 17-year-old son is receiving cyclodextrin intrathecally as part of Vtesse's trial.