You do have to wonder if MSB had only taken one application and gone with a purely AU focused trial and regulatory pathway if it would not have done better? Leveraged any financial, IP and regulatory success therein. Built on organic domestic success internationally. The Fin Review, so long a hard critic of MSB, at least concedes it leads the world in stem cell research. What a tragedy not just for patients, but for Australia if such a coveted collection of experts and clinical research, ultimately Australian based, does not prevail.
In April 2023 the FDA approved Gamida's Omisirge
https://www.fda.gov/vaccines-blood-biologics/omisirgeIt is described as a nicotinamide modified, allogeneic hematopoietic progenitor stem cell therapy. All the other hematopoietic blood cancer treatments approved in the US tending to be autologous bone marrow based. Many more better 'scientists' than I, will argue the fairness of it being described the first full allogeneic stem cell treatment approved by the FDA ahead of MSB, as it often is by the media - it is an umbilical cord blood derived human allogeneic stem cell product - but its approval history interesting.
Gamida is a 25-year-old Israel-based biotech listed in the US, that I have been invested in during the past. The company has suffered like MSB really:
Sorry couldn't resist.
If has struggled for years with the FDA, most recently in 2021 when the FDA requested more information on Gamida’s manufacturing facility (sounding familiar?) for the treatment after its successful phase 3 trial used drug supplies made from three different sites. Another delay in 2022 when the FDA said it needed an additional three months to decide on Gamida’s application after the company submitted additional data the regulator considered “a major amendment” to the submission.
Guess which other dodgy big pharma company (and they have been very dodgy at times) has been involved in Gamida? Oh the irony. You have to laugh or you'd cry:
https://www.labiotech.eu/trends-news/novartis-declines-gamidas-buyout-deal-second-time/#:~:text=Novartis%20has%20rejected%20the%20Israeli,milestones%20of%20the%20bilateral%20agreement.
It will be a tragic shame if MSB does not succeed. For all the shouting in forums, MSB is convinced it can disprove the FDA's homogeneity concerns and if so it really does feel like they will get approval via one final trial, with lower costs and and more authenticity being conducted by the Blood and Marrow Transplant Clinical Trials Network (BMT CTN), a body after all, responsible for approximately 80% of all US transplants. Its just whether another bigger player or investor can be willed into partnering/investing.
(20min delay)