The USA’s Osiris Therapeutics has reached an agreement with regulators in the USA which could result in the firm’s Prochymal becoming the first stem cell therapy to be given full approval.

Osiris says it has held “a successful pre-Biologics License Application meeting” with the US Food and Drug Administration for Prochymal used in the treatment of graft-versus-host disease. Chief executive Randal Mills said that “agreement on the timing and content of the BLA marks an important milestone in our quest to make Prochymal the world’s first fully-approved stem cell therapy”.

He thanked the FDA for its “significant efforts over the past several years as we have forged a highly productive partnership in this new and exciting area of medicine”. The talks centred around the type and extent of safety and efficacy data that would be included in the BLA, manufacturing and quality data, plus the process and format of the submission itself. By doing this, “we have created a clear understanding of the regulatory path to market approval,” Mr Mills added.

Osiris and the FDA also discussed the need for an expanded access programme (EAP) to make Prochymal available to critically ill steroid-refractory adult GvHD patients until a final regulatory decision is reached. Osiris currently has an EAP in place for paediatric GvHD patients in the USA and Canada.

In November last year, Osiris and Genzyme Corp signed a development and commercialisation pact for Prochymal.