FDA Provides Seven Years of Orphan-Drug Exclusive Approval for Ryoncil®

Core Insights - Mesoblast has received seven years of orphan-drug exclusive approval from the FDA for Ryoncil (remestemcel-L) to treat steroid-refractory acute graft versus host disease (SR-aGvHD) in pediatric patients aged 2 months and older [1][5] - The FDA's approval grants Mesoblast statutory exclusivity, preventing the approval of other mesenchymal stromal or stem cell products for this indication during the exclusivity period [2] - Mesoblast holds biologic exclusivity that prevents other sponsors from referencing the Ryoncil biologic license application until December 2036, providing a barrier against biosimilar market entry [2] - The company has a strong U.S. intellectual property position on MSC composition, manufacturing, and indications, providing commercial protection against competitors through 2044 [3][7] Company Overview - Mesoblast is a leader in developing allogeneic cellular medicines for severe inflammatory conditions, utilizing proprietary mesenchymal lineage cell therapy technology [4] - Ryoncil is the first FDA-approved MSC therapy for SR-aGvHD in pediatric patients [5] - The company is also developing additional cell therapies for other inflammatory diseases and has established commercial partnerships in Japan, Europe, and China [6] Intellectual Property and Manufacturing - Mesoblast has over 1,000 granted patents or patent applications covering various aspects of MSC technology, expected to provide commercial protection through at least 2044 [7] - The company's proprietary manufacturing processes yield industrial-scale, cryopreserved, off-the-shelf cellular medicines, ensuring availability to patients worldwide [8]