Mesoblast

The FDA approved remestemcel-L (Ryoncil) for pediatric steroid-refractory acute graft-vs-host disease (SR-aGVHD), supported by phase 3 GVHD001/002 trial data. Remestemcel-L showed higher day 28 overall response (70.4%) and day 100 survival (87%) compared to best available therapy. A 4-year survival study indicated durable benefits, with 49% survival at 4 years. The approval followed a resubmission addressing FDA concerns.

Mesoblast's Ryoncil, a mesenchymal stromal cell therapy, received FDA approval for treating children with steroid-refractory acute graft-versus-host disease (SR-aGvHD). The therapy, derived from bone marrow, is the first of its kind in the US market. Ryoncil is now available as an intravenous infusion at US transplant centers. Mesoblast aims to expand Ryoncil's indications and pursue approval for other late-stage products. The company will also debut on the Nasdaq Biotechnology Index on December 23.

Mesoblast's RYONCIL® (remestemcel-L) is the first FDA-approved mesenchymal stromal cell (MSC) therapy in the U.S., specifically for children aged 2 months and older with steroid-refractory acute graft versus host disease (SR-aGvHD). Clinical trials showed a 70% overall response rate by Day 28 in SR-aGvHD patients, addressing a significant medical need as 50% of U.S. allogeneic bone marrow transplant patients develop aGvHD, with nearly half not responding to first-line steroid treatment.

The FDA approved remestemcel-L (Ryoncil) for pediatric steroid-refractory acute graft-vs-host disease (SR-aGVHD), supported by a 70% overall response rate in a phase 3 trial. The approval process faced multiple CRLs requiring additional data, culminating in a third BLA acceptance in 2024.

The FDA approved Mesoblast's remestemcel-L, the first allogeneic MSC therapy, for steroid-refractory acute GvHD in children. Ryoncil, marketed under this approval, marks a milestone in cell-based therapies for life-threatening diseases. The decision was based on sufficient data from a phase 3 trial, showing 30% complete and 41% partial responses in treated children. Common adverse reactions included infections and fever, with precautions for hypersensitivity and infusion reactions.

FDA approves Mesoblast's remestemcel-L-rknd for steroid-refractory acute graft-versus-host disease in pediatric patients, marking the first FDA-approved mesenchymal stromal cell therapy. The approval is based on a study showing 50% survival through 4 years in remestemcel-L-treated patients, compared to less than 20% expected. The therapy demonstrated 70.4% day 28 overall response and 87% day 100 survival in pediatric patients.

Mesoblast (Nasdaq:MESO; ASX:MSB) announced its upcoming addition to the Nasdaq Biotechnology Index (NBI), effective after the U.S. market opens on December 23, 2024. The NBI includes Nasdaq-listed companies classified as Biotechnology or Pharmaceuticals, meeting specific eligibility requirements like minimum market capitalization and average daily trading volume. The index operates under a modified capitalization-weighted methodology, with annual constituent selection in December.

Mesoblast shares rose on the ASX after being added to the Nasdaq biotechnology index, up 1.42% to $1.99 by 2pm AEDT, having surged over 500% since March. The Melbourne-based company will join the index on 23 December, which includes Nasdaq-listed biotech and pharmaceutical companies meeting specific criteria.

Mesoblast announced positive Phase III trial results for Revascor, an allogeneic mesenchymal precursor cell therapy, showing improved survival and reduced major morbidity in high-risk ischemic heart failure patients. The treatment, delivered via minimally invasive cardiac catheterisation, demonstrated sustained reduction in cardiovascular mortality and major adverse events, offering a promising regenerative approach for cardiac damage repair.