Ann: FDA Accepts Mesoblast's BLA Resubmission for Remestemcel-L, page-212

@Hawk70 Once the submission is accepted , the actual filing date starts the clock…hence the PDUFA date for this Class 2 is anticipated to be “on or before the PDUFA goal date of 7th January 2025.
It is worth noting that the resubmission has been accepted by the FDA as a complete response. Interestingly, the protocol for a resubmission is not the same as a standard submission. Although the FDA are a law unto themselves and may possibly drag this out for the full six months..i believe this is unlikely.
There are really two remaining issues…confirmation that the new potency assay is acceptable and then to conclude any remaining items not covered during the submissions…namely labelling and post marketing trial commitments.

The reason I am extremely confident of approval is that a formal meeting was held with the FDA in March this year. Mesoblast presented its new potency assay for use with a proposed adult trial for steroid refractory acute GVHD patients. The FDA was satisfied with the data provided and had also reviewed additional paediatric published data (such as four year survival data, validated paediatric MAGIC biomarker scores.,etc, etc) . As a result of the latter developments Mesoblast was told by the FDA to re submit and they could defer the adult trial until after approval. I reiterate my opinion from previous posts that the actual decision will be announced by the end of month 4 at the latest (Nov 7th ) but could come as early as September.

Assuming near term authorisation of Ryoncil , prospects for early sales are good. The top 15 GVHD centres account for almost 50% of the 500 or so paediatric patient that suffer from this horrific indication. Until recently Mesoblast had been treating under compassionate use, up to 2-3 patients a week. Pricing is expected to be somewhere between CART-T therapies such as Kymriah which sell at slightly under $500k and have long term survival rates close to Ryoncil …..and gene therapies who are often marketed above $1m + . I prefer to model on the basis of a list price of $700k and assume 30% market share within two years of marketing approval. On that basis we could be talking about over $105m of paediatric sales. With almost 50% of adult patients now refractory to Ruxolitinib, off label use in the adult market as a third line therapy for severe grade patients will be low hanging fruit. Mesoblast is expected to commence a single arm circa 60 patient trial for adults with steroid refractory acute GVHD shortly…within four years I believe sales in the US will exceed $400m for Ryoncil ..justifying a market cap of at least $5bn for the GVHD indication alone. OP

Let’s hope I am right !


Please do not rely on the facts or opinions expressed in the above post when making an investment decision.