Gene Therapy: Years After Accelerated Approval, Will US FDA Still Be Asking ‘Does It Work?’

Retiring Office of Tissues and Advanced Therapies’ Director Wilson Bryan expects the expedited pathway will be used more frequently moving forward, but worries that ambiguous confirmatory trial results many years down the line will still leave efficacy questions unanswered.

Hourglasses
The FDA's Wilson Bryan worries that answers to gene therapy efficacy questions will not emerge with the passage of time. • Source: Shutterstock

Accelerated approval is likely to be used more extensively in the future for gene therapies that treat very rare diseases, but the slowly progressive nature of some conditions means there may be many years of uncertainty about the clinical benefits provided by the one-time treatments, the head of the US FDA’s Office of Tissues and Advanced Therapies said.

OTAT Director Wilson Bryan opined on the future use of accelerated approval for gene therapies, worries about ambiguous confirmatory trial...

Read the full article – start your free trial today!

Join thousands of industry professionals who rely on Pink Sheet for daily insights

  • Start your 7-day free trial
  • Explore trusted news, analysis, and insights
  • Access comprehensive global coverage
  • Enjoy instant access – no credit card required

More from Cell & Gene Therapies

HHS Secretary Favors Regenerative Medicine Over Traditional Pharma: Blessing And Curse?

 

Cell and gene therapy developers may not have to contend with HHS Secretary Robert F. Kennedy Jr.’s animosity like the traditional pharma industry, but his disinterest in distinguishing between the good and bad actors in the space could backfire.

To Compete With China, US FDA Must Deregulate Early Gene Therapy Studies, CAR-T Inventor Says

 

FDA regulation was painted as an obstacle to US dominance in the cell and gene therapy space even as panelists at an agency event praised the Office of Therapeutic Products' track record under Nicole Verdun.

Cell/Gene Therapy Cost Recovery Options Could Include Pre-Approval Public ‘Bridge’ Funding

 
• By 

Expanded funding for cost recovery could dovetail with FDA Commissioner Martin Makary’s idea for a “conditional approval” pathway based on a plausible mechanism of action.

Prasad Says Involvement In US FDA Product Approvals Will Mirror Prior CBER Directors

 

The remarks from the new CBER director suggest Prasad will not be involved in most product-specific decisions.

More from Advanced Technologies

MHRA Backs Bacteriophage Innovation To Fight Antimicrobial Resistance

 

The UK regulator wants to help companies to develop bacteriophages for the treatment of infections. Its first guidance on this topic offers advice to researchers and sets out the regulatory requirements they will need to meet.

Prasad Says Involvement In US FDA Product Approvals Will Mirror Prior CBER Directors

 

The remarks from the new CBER director suggest Prasad will not be involved in most product-specific decisions.

Scientists Work With AI To Uncover ‘Unexpected’ Drug Combos For Breast Cancer

 

A UK research team has used artificial intelligence to find new treatments for cancer using existing US Food and Drug Administration-approved medicines that are not normally not used for the disease.