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Mesoblast scores FDA committee's backing for cell therapy despite some early worries
by
Kyle Blankenship |
Aug 14, 2020 8:36am

The FDA planned some tough question around Mesoblast's data and manufacturing for cell therapy Ryoncil. (FDA)
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Cell and gene therapy makers have to contend with a laundry list of concerns on the road to approval, including a complicated manufacturing process and often, some regulatory pushback. Facing what was expected to be an FDA firing line over its cell therapy, Mesoblast is instead looking at a clear path to market.


Despite some pushback over its clinical data and manufacturing process, Mesoblast received a near-unanimous thumbs up Thursday for cell therapy Ryoncil from an FDA advisory committee.

The committee voted 9-1 to recommend the FDA's approval of Ryoncil (remestemcel-L) in pediatric acute graft vs. host disease (GVHD), an indication with no approved therapies. The drug faces a full FDA review by Sept. 30.

While advisory committees' votes are non-binding, the FDA often follows its advisors' recommendations.

In a statement, Mesoblast said it was "very encouraged" by the vote and pledged to continue working with the FDA toward a full approval.

Cantor analyst Louise Chen called the vote "clearly good news" for Mesoblast in a note to clients, and shareholders will likely agree. The drugmaker halted trading early Thursday prior to the committee's vote.



The panel's positive review was no sure thing after early briefing documents forecast some hard questions around Ryoncil's data and manufacturing.

In documents posted online Tuesday, the FDA argued that Mesoblast's pivotal phase 3 data for Ryoncil did not demonstrate that the therapy's proposed mechanism of action was actually tied to positive clinical outcomes. Ryoncil is composed of culture-expanded mesenchymal stromal cells (MSCs) that Mesoblast links to the reduction of pathogenic inflammation in GVHD patients.

The FDA also questioned Mesoblast's manufacturing of the therapy, saying other MSC-based therapies show "significant functional heterogeneity" between patients and that the biotech's expansion of MSCs in culture through adult donor banks could dilute doses over time.

RELATED: Mesoblast preps first filings for GVHD drug after phase 3 win

With the FDA's full approval, Mesoblast could escape the fate of other cell and gene therapy makers that have run into regulatory roadblocks over their manufacturing, a notoriously expensive and labor-intensive process.

In February 2018, Mesoblast posted pivotal phase 3 data in pediatric GVHD showing 69% of Ryoncil patients had a complete or partial response to the treatment after 28 days. The therapy is already approved as Temcell in Japan, where it is licensed to JCR Pharma.

Mesoblast is also pursuing clinical trials for Ryoncil in adult and chronic GVHD and to treat acute respiratory distress syndrome in COVID-19 patients, among other indications.