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# FDA Challenges Remestemcell-L Data
- URL: https://www.fdaweb.com/fda-challenges-remestemcell-l-data/
- Published: 2020-08-11T12:00:00.000Z
- Updated: 2026-09-14T16:24:30.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5147407

Briefing documents released by FDA in advance of an Oncologic Drugs Advisory Committee 8/13 meeting to consider the Mesoblast BLA for its Remestemcell-L as a treatment for steroid-refractory acute Graft Versus Host Disease (SR aGVHD) in pediatric patients challenge the biologic’s effectiveness and durability of patient responses. The committee is to discuss whether an additional clinical trial is required to confirm the product’s effectiveness and what trial design would be required to produce evidence of effectiveness.

In [one](https://www.fda.gov/media/140986/download?ref=fdaweb.com) of the briefing documents, FDA says it wants committee input on whether the results of one statistically-positive single-arm trial “in a landscape of the multiple negative clinical trials for the treatment of aGVHD, including randomized control trials, is adequate to allow one to conclude that remestemcell-L is effective in the treatment of SR aGVHD in pediatric patients.

The second briefing document asks committee members to consider the product attributes identified by Mesoblast as critical quality attributes and discuss whether they are adequate to ensure that the manufacturing process will continue to produce lots of consistent quality. The committee is also asked to discuss other product characteristics not previously identified as critical quality attributes that might provide more meaningful measures of product quality and potency and thus better assurance of product quality from lot to lot.

The reviewers noted that remestemcell-L has yet to be approved in the U.S. in more than 20 years of existence, despite multiple clinical trials for various inflammatory conditions.