Trending Now - FDA Actions Raise Concerns for Experimental Drug Investments

WHO:

U.S. Food and Drug Administration (FDA)

UniQure, a biotechnology company

Robert Langreth, reporter for Bloomberg

Luca Issi, RBC Capital Markets analyst

Ryan Watts, CEO of Denali Therapeutics

Marty Makary, FDA Commissioner

Adam Feuerstein, senior writer and biotech columnist

Stifel analyst Paul Matteis

Dyne Therapeutics, Taysha Gene Therapies, Wave Life Sciences, Lexeo Therapeutics (biotech companies)

WHAT:

The FDA has denied or discouraged the applications of at least eight drugs in the past year, including:

A gene therapy for Huntington's disease from UniQure

A gene therapy for Hunter syndrome from Regenxbio

A drug for a blood condition from Disc Medicine

The FDA initially refused to review Moderna's flu shot before reversing its decision.

The FDA has asked UniQure to run a new study comparing its treatment to a placebo, contradicting previous guidance.

Denali Therapeutics' application for accelerated approval is based on a trial that wasn't randomized.

The FDA delayed its review of Denali's application by three months, with a new decision expected by April 5, 2026.

An FDA spokesperson stated that the agency "makes decisions based on the evidence, but does not make assurances about outcomes."

WHERE:

FDA headquarters in Silver Spring, Maryland

WHEN:

The media call occurred on Thursday, March 5, 2026.

The FDA's decision on Denali Therapeutics is expected by April 5, 2026.

The FDA has denied or discouraged applications in the past year (2025).

WHY:

Investors are concerned about the unpredictability of the FDA's decisions regarding experimental drugs.

The FDA's recent decisions have raised questions about the agency's standards for drug approvals, particularly for rare diseases.

HOW:

The FDA has taken issue with the evidence provided by companies, noting that some studies did not test drugs against a placebo and relied on biomarkers to predict efficacy.

The FDA official stated that randomized data is only required in specific circumstances, such as when the condition is heterogeneous or when the therapy is invasive or potentially harmful.

The FDA has recently denied or discouraged the applications of at least eight drugs over the past year, including a gene therapy for Huntington's disease from UniQure and a blood condition treatment from Disc Medicine. During a media call last Thursday, FDA officials highlighted concerns about the evidence provided by biotech companies, noting that some studies lacked placebo comparisons and relied on biomarkers to predict efficacy. As investors express worries about the unpredictability of the FDA's decisions, the agency is expected to announce its ruling on Denali Therapeutics' application by April 5, 2026.