NextCell Pharma

As previously announced, NextCell submitted a proposal in Q4 outlining how ProTrans should be developed for the treatment of children with type-1 diabetes. The agency has returned with comments and recommendations that aiming to ensure that children younger than 7 years old can also receive treatment in the future.

"The PDCO has been very helpful with the development plan and the proposed changes strengthen our proposal.  We will of course implement these before the PIP is finally submitted for approval", says Mathias Svahn, CEO.

A PIP is a development plan aimed at ensuring that all necessary safety and efficacy data are obtained through clinical studies in children, prior to application for authorisation of a medicine for pediatric usage. All applications for marketing authorisation for new medicines have to include the results of studies as described in an agreed PIP.

Only small changes have been requested by EMA, aimed at allowing even younger age groups to be treated after a possible market approval than what is treated in the ongoing pediatric study, ie children younger than 7 years. The changes do not affect the study design of the planned phase 3 study.

In addition to clinical trials, the plan also includes preclinical studies of biodistribution and toxicology. NextCell is currently reviewing the recommendations by the PDCO and amending the proposal prior to submitting for approval.