ReNeuron Group plc has announced that the US Food and Drug Administration (FDA) has granted Fast Track designation to ReNeuron’s human Retinal Progenitor Cell (hRPC) therapy candidate for retinitis pigmentosa (RP). RP is a group of hereditary diseases of the eye that lead to progressive loss of sight due to cells in the retina becoming damaged and eventually dying.
The granting of Fast Track status follows the Company’s announcement on 5 May 2015 that it had received regulatory approval from the FDA to commence a Phase I/II clinical trial in the US with its stem cell therapy candidate for RP.
Fast Track designation is an FDA programme intended to expedite the development and review of new drugs or biological products targeting unmet medical need where the diseases concerned are serious or life threatening. Developers of products with Fast Track designation benefit from more frequent interactions with the FDA during clinical development as well as the potential for priority review and an accelerated market approval process for the product if supported by appropriate clinical data.
ReNeuron’s cell therapy candidate for RP has already been granted Orphan Drug Designation in both Europe and the US by the European Commission and the FDA, respectively. Products with Orphan Drug Designation benefit from potential market exclusivity post-approval for up to 7 years in the US and up to 10 years in Europe.
The Phase I/II clinical trial with ReNeuron’s cell therapy candidate for RP will be conducted at Massachusetts Eye and Ear, Boston, a world-renowned clinical centre for the treatment of retinal diseases. The trial design is an open-label, dose escalation study to evaluate the safety, tolerability and preliminary efficacy of the hRPC stem cell therapy candidate in up to 15 patients with advanced RP. Preparations for the study have commenced and ReNeuron expects the study to begin in the second half of this year.
Olav Hellebø, Chief Executive Officer of ReNeuron, commented: “The granting of Fast Track designation from the FDA for our hRPC stem cell therapy candidate for RP is a very significant positive development for the Company. In considering the pre-clinical data we have presented to them and granting the designation, the FDA has recognized the potential of this treatment candidate to address RP as a serious unmet medical need. This, together with the Orphan Drug Designation already granted for the programme in both the US and Europe, provides accelerated clinical development and marketing authorization processes for our RP treatment candidate as well as the potential for a significant period of market exclusivity once approved in these major territories.”