In March, the FDA said uniQure's data was too weak. On Wednesday, it backed a path to clear the same drug.
What changed was not the science. It was who runs the FDA, and the stock jumped 70%.
uniQure makes a gene therapy for Huntington's disease, and the stock trades as QURE. Huntington's runs in families and slowly kills brain cells.
There is no cure for it today. That is part of why this news landed so hard, and why investors piled in fast.
Doctors put the drug right into the brain, and the surgery takes hours. In one early trial, it slowed the disease by about 75%.
uniQure did not run a normal trial with a control group. Instead, it matched its patients against records of past cases.
The FDA now says a three-year look at that trial is enough. It will back a faster OK based on that data.
A faster OK lets a drug reach patients sooner, but the maker still has to prove it works. uniQure plans to file by the end of September.
On Wednesday, the stock soared 70%. That kind of move in a day is a wild swing for a small biotech.
Before this news, uniQure was a high-risk bet. One FDA meeting can make or break a stock like this.
Stories like this, where a single decision moves a stock overnight, are what we unpack every morning in Market Briefs, which comes with a free investing masterclass when you join.
The turn came after a shake-up at the top. The FDA chief at the time, Marty Makary, had trashed the drug.
In a February CNBC talk, he knocked the treatment without naming it. He said the agency was pushed to clear a drug with "no benefit."
The FDA wanted a placebo trial instead. That means a fake surgery for some patients.
uniQure said that was wrong and refused. The FDA still says it wants "gold-standard science."
But it now backs the same data it once turned down. That is a big swing in just three months.
Makary left in May, and two other top officials left around the same time. They were Vinay Prasad and Tracy Beth Hoeg, who ran key drug-review units.
uniQure is not the only firm whose luck turned after they left. Replimune is now trying to clear its skin cancer drug for a third time.
The deal is not done yet, and uniQure still has to settle a new study with the FDA. This time it will test the drug against today's care, with no fake surgery.
The company says it will lock in that plan before it files. uniQure got a second chance, and investors will watch the next FDA meeting closely.
Now it has to do the work to keep it.
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