USA— The United States Food and Drug Administration (US FDA), has requested for more data to support marketing approval for Mesoblast’s remestemcel-L, a regenerative medicine to counter graft versus host disease in children under 12 years.

Subsequently, the US FDA has rejected the Mesoblast’s Biologics License Application (BLA) for remestemcel-L for the treatment of pediatric steroid-refractory acute graft versus host disease (SR-aGVHD).

The company announced in a statement, that the US FDA had provided a complete response and “requires more data to support marketing approval”.

The company continued that to obtain the data required, it would conduct a targeted, controlled study in the highest-risk groups with the greatest mortality.

“This study is in line with our overall commercial strategy, which envisioned a sequenced progression from pediatric to adult SR-aGVHD indications,” the statement from Mesoblast noted.

Mesoblast Chief Executive Silviu Itescu said that the company remained steadfast in making remestemcel-L available to both children and adults suffering from this devastating disease and has received substantial clarity on how to bring this much-needed product to these patients.

Correspondingly, Itescu added that in line with the overall commercial strategy to expand into the adult SR-aGVHD indication and the pharmaceutical manufacturer had already been working with leading investigators at various US centers of excellence to establish the adult follow-on study protocol.

The company will seek alignment with FDA on the trial design for the adult study at a type A meeting within 45 days.

Prior to the resubmission, FDA has guided Mesoblast to resolve outstanding chemistry, manufacturing, and controls (CMC) issues before initiating any additional clinical trial.

The news has sent the regenerative medicine manufacturer’s shares tumbling 58% downwards by the close of trading on Friday at the Australian Stocks Exchange.

Reuters reported that Mesoblast shares slumped as much as 58.3% when they opened in Sydney, before marginally paring some losses to trade down 54.8% at A$0.49 by 0416 GMT.

Mesoblast is a world leader in developing allogeneic cellular medicines for the treatment of severe and life-threatening inflammatory conditions.

The pharmaceutical company has leveraged its proprietary mesenchymal lineage cell therapy technology platform to establish a broad portfolio of late-stage product candidates which respond to severe inflammation.

Remestemcel-L is being developed for inflammatory diseases in children and adults including steroid-refractory acute graft versus host disease, biologic-resistant inflammatory bowel disease, and acute respiratory distress syndrome.

The product has been commercialized in Japan and Europe by Mesoblast’s licensees, and the company has established commercial partnerships in Europe and China for certain Phase 3 assets.

About Steroid-Refractory Acute Graft Versus Host Disease (GVHD)

According to the National Institute of Health (NIH), Acute GVHD occurs in approximately 50% of patients who receive an allogeneic bone marrow transplant (BMT).

Moreover, over 30,000 patients worldwide undergo an allogeneic BMT annually, primarily during treatment for blood cancers, including about 20% in pediatric patients.

First-line treatment involves systemic corticosteroids, and a significant proportion of patients have a severe rejection of steroids.

The SR-aGVHD is associated with mortality as high as 90% and significant extended hospital stay costs.

There are no approved therapies in children for the condition in which the donor’s bone marrow or stem cells attack the recipient.

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