UniQure lost nearly half a billion dollars in market value Thursday, as comments made by the head of the Food and Drug Administration appeared to stoke investor fears that the company’s most advanced research project won’t be approved in the U.S.
Shares of UniQure dropped more than 30% shortly after FDA Commissioner Martin Makary appeared on CNBC to defend the agency’s approach to approving rare disease therapies. The agency has come under fire for multiple delays and regulatory setbacks involving these medications. Makary, though, said critics have been searching for a "boogeyman" to blame for recent rejections, when the bottom line is that some of these therapies just haven’t been proven effective.
He mentioned, as one example, how the FDA was pressured to approve a product that’s injected into the space surrounding the brain, through a burr hole drilled into patients’ skulls.
Makary didn’t name any company, but that didn’t stop some investors from interpreting his remarks as a reference to UniQure’s “AMT-130,” a gene therapy for Huntington’s disease administered via burr hole.
Wall Street analysts noted that Makary might not have been talking about UniQure, especially since other details he shared about the safety, delivery and review of this unnamed therapy didn’t align with AMT-130. Some speculated he could have been talking about a medicine from Regenxbio designed to treat a rare metabolic disorder.
In any case, what's notable from Makary’s appearance is his “steadfast defense” of the FDA, “which is discouraging for those hoping that the agency can be convinced to reconsider recent negative decisions, in the absence of specific political pressure,” wrote Paul Matteis, of Stifel, in a note to clients.
Under Makary, the FDA has taken pains to say it intends to make rare disease drug development quicker and easier. Just last week, the agency provided initial guidance on testing medicines for extremely uncommon conditions. Several rare disease treatments have also been the recipients of a new, controversial voucher program that drastically shortens approval review timelines.
Yet those efforts also contrast with other FDA actions, most notably a series of shifts in regulatory guidance that have left drugmakers and industry experts perplexed. One such example came in November, when UniQure said agency staff had a change of heart about AMT-130 even though the drug had just scored clinical data that wowed both investors and the Huntington’s research community.
The two parties had been meeting extensively in the months leading up to those results, to ensure UniQure was collecting the necessary data to support an approval application. They seemed to be on the same page until a meeting late last year, wherein, according to UniQure, agency staff indicated the company needed more evidence.
“This is a key shift from prior communications,” UniQure said in a statement, adding that the timing for an approval filing “is now unclear.”
Shareholders have been hoping the Netherlands-based biotechnology company can work out a path to approval with the FDA. Analysts expect an update on those proceedings Monday, when UniQure reports fourth quarter earnings.
