Is the FDA sabotaging medical companies? UniQure’s valuation rollercoaster

UniQure is a Danish biotech-pharmaceutical company with a portfolio of breakthrough research and products. Just a few months ago, the company’s valuation was once again approaching its all-time high, only to lose 85% over the course of four months. Where does such extreme volatility come from?

Most of the investment thesis currently hinges on a revolutionary therapy with the working name “AMT-130.” It is the first gene therapy of its kind focused on treating Huntington’s disease, an incurable and fatal neurodegenerative disorder.
In its studies, the company showed that the drug can slow disease progression by 60–75%. This would hypothetically mean slowing the disease to a degree that could allow patients to live into old age. In early March, the FDA officially announced that it does not consider the study results to be reliable.

What the FDA expects is a large-scale, long-term, and complex testing and research procedure. However, given the rare and fatal nature of the disease, the requirements set by the FDA range from difficult to meet and unethical, to those that are virtually impossible.

So why the shift in sentiment and an approximately 30% rise in today’s session? The answer lies in the resignation of the head of CBER. Vinay Prasad, strongly linked to the Trump administration and the new U.S. Secretary of Health, Robert F. Kennedy Jr., was dismissed from his position for the second time after months of wreaking havoc on procedures and the decision-making process.

Prasad was personally involved in blocking several promising therapies without substantively addressing the study results.

After Prasad’s departure, the market is betting that the breakthrough therapy, now “in the final stretch,” will return to a path toward rapid market entry.

QURE.US (D1)

Source: xStation5