AUS
NZ
UK
CA
USA
IND
Highlights:
- Mesoblast Limited (ASX:MSB) share price skyrocketed after it updated that FDA had accepted its resubmission.
- If MSB’s resubmission gets FDA approval, remestemcel-L will be the first allogeneic “off-the-shelf” cellular medicine in the US.
- FDA believes resubmission to be a complete answer and has decided on a Prescription Drug User Fee Act target date of 2 August this year.
Mesoblast Limited (ASX:MSB), a world leader in developing allogeneic cellular medicines for treating inflammatory conditions, got a boost on Wednesday, 8 March 2023, as the US drug regulator accepted its resubmission of Biologics License Application (BLA) for remestemcel-L, which is meant for treating children with steroid-refractory acute graft versus host disease (SR-aGVHD).
MSB shares closed 14% higher at AU$1.055 today on the ASX after rising as much as 23.24% to an intraday high of AU$1.14.
Mesoblast said in an ASX filing today that the United States Food and Drug Administration’s (FDA) Office of Therapeutic Products (OTP) has accepted the company’s resubmission of the BLA as mentioned above. The US drug regulator considers the resubmission to be a complete response and has set a Prescription Drug User Fee Act (PDUFA) goal date of 2 August 2023, the company said.
If resubmission gets consent from FDA, remestemcel-L will be the maiden allogeneic “off-the-shelf” cellular medicine in the US. It will also be the first therapy for children under twelve with steroid-refractory acute graft versus host disease.
Survival results have not got better in the last twenty years for the most serious types of SR-aGVHD, which is a life-threatening complication of an allogeneic bone marrow transplant after being treated for blood cancers and other health conditions.
FDA approved remestemcel-L speed-up designation, a procedure to enable the progress and accelerated review of therapies for severe conditions that fill unfulfilled medical requirements, and priority review designation. It is given to drugs that treat an acute condition and considerably improve safety or efficiency over present treatments.
Mesoblast’s remestemcel-L
Remestemcel-L is the company’s lead product candidate, which is an investigational therapy including culture-expanded mesenchymal stromal cells received from the bone marrow of an unrelated donor. It is dispensed to patients in a series of intravenous infusions.
Mesoblast’s resubmission of new points
The company’s resubmission has new points, a few of which are as follows:
New long-duration survival data via a minimum of four years for children enrolled in the phase 3 trial. It was followed by fresh outcome data after remestemcel-L usage in high-risk ailment activity along with survival utilising propensity-matching of children in the phase-3 trial and controls stratified by authenticated biomarkers.
The other point in it is new analyses of prospectively received data pertaining to the authenticated potency assay. It was utilised to release the product for phase 3 clinical trial.
Also, new analyses of clinical data received prospectively relating manufacturing alterations executed during product development before phase 3 to progressive increases in potency as well as to better survival results of children with steroid-refractory acute graft versus host disease being treated with remestemcel-L within expanded access.
