1. Pink Sheet
  2. >>Product Reviews
  3. >>Complete Response Letters

Mesoblast Plans Small Trial After Second CRL For GVHD Cell Therapy

 
• By Joseph Haas

Seeking approval for pediatric patients with acute graft-versus-host disease, Mesoblast gets a second US FDA complete response and now says it will seek to get needed data from a small trial in adults.

FDA Complete response stamp holder
Mesoblast is planning new trial after second FDA complete response • Source: Nielsen Hobbs; the Pink Sheet | Alamy image

For a second time, the US Food and Drug Administration has told Mesoblast Limited that a biologic licensing application for its cell therapy candidate for pediatric patients with steroid-refractory acute graft-versus-host disease (SR-aGVHD) lacks necessary data. On a 4 August analyst call, Mesoblast said it now plans to conduct a small study in high-risk adult GVHD patients that it hopes will meet the agency’s requirements for approval.

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 Complete Response Letters

US FDA’s Review Performance Held Steady Despite Stormy First Quarter

 
• By Bridget Silverman

Organizational turmoil has not yet affected reviews of novel agent applications, with Q1 2025 approvals coming in low, but the first quarter share of the entire year is variable.

Elevar/Hengrui’s Second US CRL A Bad Omen For Multiregional Trials In Asia-Dominant Cancers?

 
• By Dexter Jie Yan

While CMC glitches linger over a US NDA for Elevar/Hengrui’s novel liver cancer combination following a second complete response letter, the separate issue of underrepresentation of US patients in multiregional trials is looming large after new FDA draft guidance last year.

US FDA Spikes Vanda’s Hetlioz For Insomnia, Lack Of Well-Controlled Trial ‘Fatal’

 
• By Sue Sutter

Acting Commissioner Sara Brenner rejected the company’s request for a hearing, saying the lack of an adequate and well-controlled trial showing tasimelteon's effectiveness in treating sleep-onset insomnia is “fatal to the application.”

Better Luck Next Year: US FDA CRLs May Be Rising, But Are Not The End Of The Story

 
• By Bridget Silverman

Prospects are promising for many of the 16 novel candidates that received complete response letters in 2024 after half of the 2023 CRL class was approved the following year.

More from Product Reviews

Chikungunya At US CDC: Bavarian Nordic’s Vimkunya Recommended, Valneva’s Ixchiq Adjusted

 
• By Bridget Silverman

Bavarian Nordic's Vimkunya added the US CDC immunization committee's recommendation to its FDA approval, while Valneva's Ixchiq faces a precaution for use in people aged 65 and older.

US FDA Commissioner Makary Wants To Combine Adverse Event Reporting Systems

 
• By Sarah Karlin-Smith

In discussing FDA’s adverse event monitoring, Makary also seemed to falsely imply the agency did not fully investigate the myocarditis signal with COVID-19 vaccines.

US FDA User Fee Collection Safe In Preliminary Trump 2026 Budget Plan

 
• By Cathy Kelly

The preliminary White House 2026 budget plan cuts agency funding, but not so sharply that user fees are excluded from collection.