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- Mesoblast (MSB) has submitted its Biologics Licence Application (BLA) to the U.S. Food and Drug Administration (US FDA) for Ryoncil
- Ryoncil is the company’s lead cell therapy used to treat children with steroid-refractory acute graft versus host disease
- Mesoblast requested the application be given Priority Review as there currently isn’t any FDA-approved treatments for children with this disease
- If this goes well, Ryoncil is expected to be launched in the U.S. some time this year
- Mesoblast is down a slight 0.67 per cent with shares trading at $2.98 each
Mesoblast (MSB) has submitted its Biologics Licence Application (BLA) to the United States Food and Drug Administration (US FDA) for Ryoncil.
Ryoncil (remestemcel-L) is Mesoblast’s lead allogeneic cell therapy which is used to treat children with steroid-refractory acute graft versus host disease (SR-aGVHD).
The regenerative medicine company filed the final module for the application with the FDA on January 31. It has requested Priority Review of the BLA by the FDA and, if approved, Ryoncil is expected to be launched in the U.S. this year.
“This is a major corporate milestone for Mesoblast,” CEO Dr Silviu Itescu said.
Acute GVHD occurs in about 50 per cent of patients who go through an allogenic bone marrow transplant.
In patients with the most severe form of the disease, mortality is as high as 90 per cent. Unfortunately however, there isn’t any FDA-approved treatments in the U.S. for children with SR-aGVHD.
Ryoncil has been used on 309 children with SR-aGVHD across three separate studies. It was used as a salvage therapy in an expanded access program in 241 children who failed institutional standard of care.
The lead drug candidate was also used as first-line therapy in Mesoblast’s Phase 3 trial in 55 children with SR-aGVHD, 89 per cent of whom has a severe form of the disease.
“We look forward to working closely with the FDA to potentially bring Ryoncil to market and providing our innovative biologic therapy to the many children with this life-threatening condition,” Dr Silviu added.
Mesoblast is down a slight 0.67 per cent with shares trading at $2.98 each at 12:11 pm AEDT.
