In a flurry of activity before the end of the year, the U.S. Food and Drug Administration approved two new drugs that—in a historic first for the embattled agency—had been developed internally by FDA scientists. In their spare time, the researchers had been trying to “show our colleagues and friends in industry a thing or two about how to do clinical research in the post-Vioxx era,” FDA administrator Andrew von Eshenbach said in a prepared statement.

In a new direction for the federal government, both drugs will be manufactured in Mexico and marketed under the auspices of the U.S. government. The research effort was a top-secret element of the FDA’s Critical Path research agenda, von Eschenbach noted. In coming years, off-label sales of the new drugs are expected to augment a tight FDA budget for basic research.

“With these two new compounds, our agency attempted to understand first-hand the challenges that the industry faces every day,” von Eshenbach said. “Our budget for both projects was limited to a few hundred dollars in an unspent travel account in one of our regional offices. But we think each of these compounds fulfills an important unmet medical need. We look forward to monitoring whether either of these drugs have any safety issues. We certainly hope not.”

Translational Research Success

The first drug approved this week is Documenta, to be administered in the emergency room as a 1-liter injection. The indication? A rare but debilitating psychiatric disorder in which a patient mistakenly believes that clinical trials or general medical practice could be made significantly more efficient using information technology.

“We had a nurse who really wanted to do everything on the computer,” said Phillip Courtland, chief of psychiatry at the University of Nebraska’s occupational health division. “She was shopping on eBay. Filling out her time card online. It got out of control. We needed to intervene. Documenta worked. She’s now making multiple photocopies of every email she sends.”

New Classification

The second drug approved in the pre-holiday rush was Blamexx, a spleen inhibitor. It is FDA-approved only for elected officials at the national level who criticize programs for which they themselves have not provided adequate funding or clear statutory language. The FDA said it determined that an exceptionally small target population of just 535 potential patients (100 in the U.S. Senate, 435 in the House of Representatives) warranted the creation of a new “ultra-orphan” drug status for Blamexx, a designation that it would clarify with draft guidance after an unspecified interval.

No risk management plan was deemed appropriate for Blamexx at this time. But an FDA advisory committee did stipulate that all patients taking the drug will be required to wear a nonremovable, bright red plastic bracelet (similar to the one popularized by Lance Armstrong) so that caregivers inside Congress can easily identify and monitor those taking Blamexx for self-harming behaviors, a side effect that was recorded during clinical trials.

“A brief, controlled, quadruple-blind clinical trial of Blamexx showed that this promising suppository has excellent onset and efficacy in Republicans and Democrats alike. It helps to halt bile production across even the most elderly patients,” noted Susan Jacobs, an internist at Walter Reed Army Hospital. “It appears to have sedative properties,” she added. “We’ve not yet had time to test Blamexx on the news media or on physicians in teaching hospitals, but believe it could work equally well in those subpopulations.”