Mesoblast’s Ryoncil Wins FDA Approval for Rare Children’s Disease

Mesoblast, a biotech company, secured US Food and Drug Administration (FDA) approval for its first-ever treatment for children with graft versus host disease (GVHD). The stock surged over 35% to $16.61 in afternoon trades, with analysts predicting strong growth and potential revenue of up to $500 million.

GVHD is a common complication following bone-marrow transplants, affecting half of the roughly 10,000 patients who undergo such procedures each year. Current steroid treatments only work for about half of these cases. The new treatment, Ryoncil, targets pediatric patients with GVHD that doesn’t respond to steroids and has shown promising results.

Analysts praise Mesoblast’s achievement, saying it validates the FDA’s growing support for cell therapy. Ryoncil is the first FDA-approved treatment using mesenchymal stromal cells derived from bone marrow, which can differentiate into various cell types. The company’s shares have nearly doubled since breaking out of a cup base in October, and analysts expect continued growth.

Mesoblast is also working on treatments for heart failure and chronic low back pain. The approval of Ryoncil marks a significant milestone for the biotech industry and positions Mesoblast for future success.

Source: https://www.investors.com/news/technology/mesoblast-biotech-stock-graft-versus-host-disease-ryoncil-approval