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.
