Mesoblast seeks FDA fast-track for GVHD treatment

Mesoblast (ASX:MSB) will soon meet with the US FDA to discuss potential pathways for accelerated approval for allogenic stem cell product Prochymal in acute graft versus host disease (GVHD).

Mesoblast acquired Prochymal as part of its purchase of the culture-expanded mesenchymal stem cell business of Osiris Therapeutics, in a deal announced in October last year. The purchase price may reach up to US$100 million ($107.7 million) after clinical development and milestone payments.

A key rationale for the acquisition was the fact that Prochymal has both Orphan Drug and Fast Track designation from the US FDA.

GVHD is a potentially life-threatening complication of hematopoetic stem cell transplants (HSCT), which are used for treating disorders including some forms of anaemia and immunological deficiencies. It occurs when immune cells in the donated cell population attack the recipient cells.

Prochymal already has conditional approval in Canada and New Zealand for the treatment of children with acute GVHD.

Mesoblast is also developing Prochymal for Crohn’s disease, a chronic inflammatory disorder of the gastrointestinal tract.

The company said an internal review has reinforced its intention to complete an ongoing phase III trial in adult patients with Crohn’s disease refractory to steroids, immunosuppressives and biologics.

Mesoblast expects to know by the end of the year whether the primary endpoint - day 28 remission in biologic-refractory patients - has been achieved, and whether there is evidence of efficacy in high-risk groups. Patients are currently being recruited for a follow-on re-treatment trial.

Mesoblast (ASX:MSB) shares ended Wednesday’s trading 0.21% lower at $4.66