It’s true, but the wording is extremely deceptive and misleading as to what Orphan Drug Designation (ODD) actually means.
When they say: “the FDA will not approve another mesenchymal stromal or stem cell (MSC) products for this indication during the 7-year period from the approval of Ryoncil,” they don’t mean the entire condition of Graft vs Hose Disease. They mean ‘paediatric patients aged 2 months or older with Steroid-refractory aGvHD.’
Result: Who cares, Cynata is not targeting this sub-subset of patients with aGvHD. Cynata is running a trial in adults with High Risk aGvHD. This market is anywhere from 10-20 times bigger than what Mesoblast has approval in.
When they say: “Mesoblast has biologic exclusivity preventing another sponsor from referencing the Ryoncil® biologic license application (BLA) until December 2036, twelve years from its first approval which would prevent market entry by a biosimilar.” They don’t mean any company referencing the fact Ryoncil was granted approval will be denied entry into the market. They mean other MSC companies can’t use data from the Rem-L clinical trials to assist with their own approvals.
Result: The trials using Rem-L are unequivocally a clusterf*** of confounding data. Let alone the latest trial was only a single arm treatment with no placebo arm. Cynata is running their own gold-standard trials with appropriate blinding and placebo arms. They will not be relying on anything generated by Mesoblast (or Osiris).
When they say: “Mesoblast’s strong U.S. intellectual property position on MSC composition of matter, manufacturing and indications, including SR-aGvHD, that provide a commercial barrier to entry against competitors through 2044.” They don’t mean any other treatment that references MSC’s will be breaching Mesoblast-held patents. They mean that other bone-marrow derived MSC therapies may infringe in certain areas.
Result: Cynata use iPSC derived MSC’s. There have been numerous studies that demonstrate different donor tissue-derived MSCs have significantly different and overall inferior properties to Cynata’s iPSC derived MSC’s. They latest study information is summarised here: https://cdn-api.markitdigital.com/apiman-gateway/CommSec/commsec-node-api/1.0/event/document/1410-02909649-444F9DKPON2RSGOKHRACR0GN4H/pdf?access_token=0007dRXpUoLOWrVxU8shOSpxkVtF
If you are still worried about the Mesoblast announcement, note that the original aGvHD treatment, Ruxolitinib, has Orphan Drug Designation in both adults and children aged 12 and older until May 2026: https://www.accessdata.fda.gov/scripts/opdlisting/oopd/detailedIndex.cfm?cfgridkey=542716
How many times have Mesoblast brought that up as an issue?
Honestly, the Mesoblast announcement screams insecurity. I have no doubt that they are referring to Cynata with the specific references to ODD preventing MSC products, despite ODD covering any competing product. A company with a value of $2200 million is scared of a competitor valued at $40 million.
That should tell you everything.
(20min delay)