Mesoblast scores FDA committee's backing for cell therapy despite some early worries
The FDA planned some tough question around Mesoblast's data and manufacturing for cell therapy Ryoncil. (FDA)
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.