Ann: FDA Accepts Mesoblast's BLA Resubmission for Remestemcel-L, page-118

The BLA resubmission to the FDA for the treatment of children with SR-aGVHD has now been accepted by the FDA with PDUFA date of Aug 2, 2023

• Mesoblast has generated substantial new data, as previously discussed, that it believes establish the relevance of the proposed potency assay measuring remestemcel-L’s in vitro anti-inflammatory and immunomodulatory activity to the in vivo clinical effect of the product in the Phase 3 trial in children with SR-aGVHD, including survival and biomarkers of in vivo activity

• Mesoblast will provide these new data to FDA and address all chemistry, manufacturing and controls (CMC) outstanding items as required for the planned BLA resubmission. If the resubmission is accepted, CBER will consider the adequacy of the clinical data in the context of the related CMC issues noted above, hence I believe the agreed (with the FDA) CMC's, manufacturing batch consistency is de-risked 100%, because with the new control measures, batches will not be released that do not conform during the manufacturing process.

I believe that the FDA are seeking to approve their first stem cell medicine, however, they are just being cautious and seeking more clarity on CMC processes for manufacturing and will inspect our plant in Singapore soon. The FDA are well aware of the safety, efficacy and manufacturing quality of our cells based on years of use in Japan for both children and adults treating aGVHD.

Total royalty to MSB using our working cells that saved countless lives in Japan against aGVHD for period April 1 to Dec 31 2021 = $US7,490,000

https://hotcopper.com.au/posts/61137366/single

JCR Pharmaceuticals revenue for 9 months from April 1 to Dec 31 2021 was 2,648 million yen ~ US$20,362,000 annualised to ~US$27,150,000

USA market is about 4 times Japan however USA pricing will be much higher.

So minimum Revenue will be ~ US$110million, IMO, others may have a better understanding of pricing in USA.


JCR Pharmaceuticals in Japan is already demonstrating our manufacturing processes are extraordinarily repeatable and consistent in batch potency and effectiveness, our cells work and our manufacturing process is consistent - that is without dispute given multi-year success in Japan for successful treatment of aGVHD - Fact

I believe that the FDA want to approve Remestemcel-L for children with aGVHD given the significant unmet medical need and they have been working with MSB to do just that!

I look forward to MSB confirming approval by the FDA for Remestemcel-L for children with aGVHD well prior to August 2, 2023.

DYOR
GLTAH