US FDA Orphan Exclusivity Would Revert To Pre-Catalyst Status Under Senate Legislation

Federal court decision said the FDA cannot grant orphan exclusivity for subpopulations of a disease, which agency officials fear could damage pediatric rare disease drug development.

Drugs and judge's gavel
A court ruling denies FDA from awarding orphan exclusivity for population subsets, such as adults or children. • Source: Shutterstock

Catalyst Pharmaceuticals, Inc.’s court victory, which scrambled the US Food and Drug Administration’s orphan drug exclusivity policy, could be undone in upcoming user fee reauthorization legislation.

A federal appeals court in September 2021 ruled that the FDA should withdraw approval of Jacobus Pharmaceutical Co. Inc’s...

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 Rare Diseases

Orphan Drug Trial Sponsors ‘Playing Catch-Up’ With Regulators – But Automation Can Help

 

Clinical trial sponsors in the rare disease space face “big safety demands” from regulators, which can be challenging for those with limited resources. A CRO founder makes the case for “embedding automation” into the clinical trial process to keep pace with regulatory requirements.

Elegy For SACHRP: US HHS Research Protection Panel Tackled Tough Pediatric Trial Questions

 

The termination of the HHS Secretary’s Committee on Human Research Protections closed an important forum for discussing pediatrics and pregnancy in clinical trial design, a former SACHRP chair said.

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.

More from Pink Sheet

Future Of US FDA’s Diversity Action Plan Guidance ‘Up In The Air’ As Statutory Deadline Looms

 
• By 

A 26 June deadline looms for the final guidance even though a draft guidance was removed from the agency’s website to comply with President Trump’s gender ideology executive order. The concepts underpinning the guidance are still relevant, legal experts say.

Pink Sheet Podcast: Prasad Staying Out Of Application Reviews, US FDA Proposed Budget Cuts

Pink Sheet reporter and editors discuss CBER Director Vinay Prasad’s expectation to stay out of routine application decisions, despite the agency’s top-down leadership approach, and the extent of proposed FDA budget cuts.

Semaglutide: EMA Safety Probe Confirms Rare Eye Disorder Risk

 

The EU product information for Novo Nordisk’s semaglutide medicines is to be updated to include non-arteritic anterior ischemic optic neuropathy as a side effect with a frequency of “very rare.”