FDA defies advisory panel recommendation, rejecting Mesoblast’s Ryoncil in paediatric steroid-refractory acute graft versus host disease
pharmafile | October 5, 2020 | News story | Sales and Marketing | FDA, Mesoblast, Ryoncil
The FDA has issued a surprising rejection of Mesoblast’s Ryoncil (remestemcel-L) as a treatment for paediatric steroid-refractory acute graft versus host disease (SR-aGVHD), it has been reported.
The ruling runs contrary to an almost unanimous recommendation from the regulator’s Oncologic Drugs Advisory Committee (ODAC) in August earlier this year, which voted nine-to one in favour of authorising the drug.
While the FDA is under no obligation to follow the advice of these expert committees when it comes to reaching a final approval decision, it commonly acts in line with their recommendations.
In its Complete Response Letter rejecting the drug, the FDA has asked for Mesoblast to conduct an additional randomized clinical trial to generate further efficacy data in adult and/or paediatric patients in this indication.
The decision is particularly problematic as there are currently no approved treatments in the US for patients under 12 with the condition. For this reason, Mesoblast has said it will “urgently” request a Type A meeting with the regulator to “discuss a potential accelerated approval with a post-approval condition for an additional study”.
The news sent the company’s shares falling by 44%, while CEO Silviu Itescu said that the decision would delay the planned launch of Ryoncil “somewhere in the order of three to five months”.
Matt Fellows
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