Keep Factually independent
Whether you agree or disagree with our analysis, these conversations matter for democracy. We don't take money from political groups - even a $5 donation helps us keep it that way.
Has Memoblast received regulatory approval from the FDA, EMA, or other agencies?
Executive summary
Mesoblast’s remestemcel‑L (branded Ryoncil) has received U.S. FDA approval for pediatric steroid‑refractory acute graft‑versus‑host disease (SR‑aGvHD), according to multiple reports citing a December 2024 FDA decision and company announcements [1] [2]. Available sources also note prior and separate approvals or marketing in other countries (for similar MSC products or different brand names), and the literature frames this FDA nod as a milestone for mesenchymal stromal cell (MSC) therapies [3] [4].
1. What regulators have approved Memoblast / remestemcel‑L — the headline
Mesoblast’s product remestemcel‑L, marketed as Ryoncil, was approved by the U.S. Food and Drug Administration for treating steroid‑refractory acute graft‑versus‑host disease in children (patients aged 2 months and up), with coverage in BioSpace, FiercePharma and company releases describing a December 2024 FDA sign‑off [1] [4] [2]. Company materials likewise list “December 2024 RYONCIL FDA Approval” in corporate updates [5].
2. What the FDA approval covers and why it matters
The FDA approval is for pediatric SR‑aGvHD and—per reporting—was based on a Phase III single‑arm trial showing a 70% overall response rate at day 28 in pediatric patients, supporting the product label and the agency’s safety monitoring recommendations for infusion‑related reactions [1]. Industry analysis and the International Society for Cell & Gene Therapy statement frame Ryoncil as the first FDA‑approved mesenchymal stromal cell (MSC) therapy in the U.S., a regulatory landmark after prior setbacks for the program [2] [3].
3. Regulatory history and earlier rejections — context on the pathway
Mesoblast’s remestemcel‑L endured a long, iterative regulatory path: earlier BLA attempts, a 9–1 advisory committee vote in favor years ago, and at least two FDA rejections (including a 2020 rejection and an August 2023 Complete Response Letter) before an accepted resubmission and eventual approval [6] [4] [7]. Reports note the FDA assigned a PDUFA goal date of January 7, 2025, for a resubmitted BLA in mid‑2024, reflecting the multi‑stage review and back‑and‑forth on data and manufacturing controls [8] [7] [9].
4. Other jurisdictions — what’s approved elsewhere and what sources say
While the FDA approval for Ryoncil in December 2024 is the clearest U.S. regulatory milestone in these materials, sources also point to approvals of related MSC products in other countries: Japan, Canada and New Zealand have marketed versions of the same or similar MSC therapies under different names (for example, Temcell or Prochymal), and China’s NMPA provided a conditional approval for a cord‑derived MSC product for acute GVHD in older patients [7] [3]. The EMA has previously approved an adipose‑derived MSC product (Alofisel) for a different indication (complex anal fistulas in Crohn’s disease), showing that Europe has approved MSC products in other niches [3]. Specific EMA approval of remestemcel‑L for SR‑aGvHD is not mentioned in the available reporting (not found in current reporting).
5. Competing viewpoints and unresolved questions
Coverage emphasizes the approval as a breakthrough for MSC therapeutics after repeated FDA rejections, but the historical record also shows agency caution—requests for randomized data and manufacturing clarity delayed approvals for years [6] [7]. Some press pieces highlight safety considerations (infusion reactions, potential infectious risk, ectopic tissue formation) that the FDA recommends monitoring [1]. Available sources do not mention an EMA approval for remestemcel‑L specifically and do not provide details about European regulatory submission status for this product (not found in current reporting).
6. What this means for patients, investors and the field
Journalists and analysts framed the FDA nod as likely to revive investor and R&D interest in MSC programs and to provide a new option for children with SR‑aGvHD who lack effective therapies, while also underscoring that the approval followed a protracted, evidence‑intensive process that included prior regulatory setbacks [2] [3] [4]. Company statements and market coverage reported a sharp share‑price reaction to the approval, reflecting commercial as well as clinical implications [4].
Sources cited in this piece: BioSpace, Mesoblast corporate materials, CGTlive, FiercePharma, Pharmaceutical Technology, ISCT statement (cited inline as [1], [5], [8], [4], [7], [3], [2], p1_s5).