1. Pink Sheet
  2. >>Geography
  3. >>North America
  4. >>United States

Biomarker Approvals At Pre-IND Stage Requested For Orphan Drugs

 
• By Derrick Gingery

Updated draft white paper on expanding use of accelerated approval suggests FDA approve the use of biomarkers as surrogate endpoints in the pre-IND stage, which could enhance sponsors’ abilities to raise funds for trials and manufacturing.

A rare disease group is pressing FDA for more systematic biomarker qualification, including a pre-IND stage approval process, arguing that it holds the key to expanding orphan drug development.

An updated draft white paper on the expansion of the use of accelerated approval (AA) continues to ask...

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
Start Free Trial

Already a subscriber?

More from United States

US & EU Regulatory Support Growing For Animal Testing Alternatives, But Validation Remains A Hurdle

 
• By Eliza Slawther

In this first of a two-part series of articles, US Pharmacopeia CEO Ronald T. Piervincenzi tells the Pink Sheet how the use of alternatives to animal testing in drug development is gaining traction and discusses the challenges that exist.

Senate Panel Approves US CDC Director Candidate Monarez With Measles Outbreak Growing

 
• By Cathy Kelly

The Senate Health, Education, Labor and Pensions Committee voted to advance Monarez's nomination to lead the CDC as the agency announced the largest US measles outbreak in 22 years.

US FDA Advisory Committee Supports Vaccine Safety, Scrutinizes AE Reporting Quality

 
• By Sue Sutter

The Pediatric Advisory Committee supported routine safety monitoring for three vaccines and 21 drugs, but the panel’s consumer representative said adverse event reporting needs improvement and FDA literature reviews should include lawsuits as well as social media.

US FDA Will Not Pull Teva And MSN Hetlioz ANDAs, Rejects Vanda Citizen Petitions

 
• By Dean Rudge

The FDA concluded that generic tasimelteon products held by Teva and MSN Labs meet all statutory bioequivalence requirements and there was no “mistake” or untrue statement in the original ANDA approvals.

More from North America

US FDA Staff Stability Should Be A Congressional Priority, Former Chief Of Staff Says

 
• By Derrick Gingery

Lawmakers should address FDA staff upheaval and help the agency “get back on track,” former agency Chief of Staff Elizabeth Jungman said, while CBER Director Vinay Prasad told staff he is asking ex-colleagues to return to the agency.

US FDA ‘Commissioner’s Voucher’ As Public Relations Tool

 
• By Michael McCaughan

NRx Pharma is trying to capitalize on the headlines about a new US FDA hyper-fast review program, illustrating that the lack of details in the FDA’s announcement is an opening for sponsors to try to grab some attention.

HHS Wants Employees (And Public?) To Report Diversity, Equity And Inclusion Discrimination

 
• By Sue Sutter

A survey asks FDA staff to disclose whether DEI policies caused discrimination or resulted in disciplinary action against career employees over the past five years, but uses a web form that the general public also can access.