Mesoblast Fails to Secure FDA Approval for Pediatric Cell Therapy; No Safety Concerns Noted

Mesoblast Limited (Nasdaq: MESO) has failed in its bid to secure FDA approval for its biologics license application (BLA) for a cell therapy for steroid-refractory acute graft versus host disease (SR-aGVHD) in children. The FDA has provided a complete response to Mesoblast’s BLA resubmission, and requires more data to support marketing approval.

To obtain the data required, Mesoblast will conduct a targeted, controlled study in the highest-risk adults with the greatest mortality. Adults comprise 80% of the SR-aGVHD market.\

Mesoblast Chief Executive, Silviu Itescu, said, “We remain steadfast in making remestemcel-L available to both children and adults suffering from this devastating disease.”

Highlights

The FDA’s inspection of Mesoblast’s manufacturing process resulted in no observed concerns, no safety issues across more than 1300 patients who have received remestemcel-L to date, and acknowledged improvements to the company’s potency assay.

Mesoblast intends to enroll adult patients at highest mortality risk with SR-aGVHD where existing therapy has not improved outcomes and 90-day survival remains as low as 20-30%.

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

About Mesoblast

Mesoblast is a world leader in developing allogeneic (off-the-shelf) cellular medicines for the treatment of severe and life-threatening inflammatory conditions. The Company has a strong and extensive global intellectual property portfolio with protection extending through to at least 2041 in all major markets. Mesoblast’s proprietary manufacturing processes yield industrial-scale, cryopreserved, off-the-shelf, cellular medicines. These cell therapies, with defined pharmaceutical release criteria, are planned to be readily available to patients worldwide.

Mesoblast is developing product candidates for distinct indications based on its remestemcel-L and rexlemestrocel-L allogeneic stromal cell technology platforms. 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. Rexlemestrocel-L is in development for advanced chronic heart failure and chronic low back pain. Two products have 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.

Mesoblast has locations in Australia, the United States and Singapore and is listed on the Australian Securities Exchange (MSB) and on the Nasdaq (MESO). For more information, please see www.mesoblast.com.

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Mesoblast Fails to Secure FDA Approval for Pediatric Cell Therapy; No Safety Concerns Noted

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