FDA rejects Rhucin BLA and asks for additional clinical data
Leiden/San Diego – Bad news for the Dutch Pharming Group and its partner Santarus Inc, who wants to market the heriditary angioedema treatment Rhucin (INN conestat alfa) in North America – the FDA said on Monday, that the companies’ BLA submitted in December was not sufficiently complete to enable a critical medical review of the EU approved recombinant human C1 inhibitor that is produced in milk of transgenic rabbits. In its „refusal to file” letter, the FDA said that the studies submitted had a insufficient number of subjects to support the proposed dose and lacked prospective validation of the visual analog scale used in measuring the clinical effects of the drug. The FDA asked to include the data of an ongoing Phase IIIb clinical study in any future BLA submission for Rhucin. Under the terms of the partnership, Pharming will be responsible for conducting and paying for the current Phase IIIb clinical study with Rhucin (Ruconest),. The drug was recommended in Europe after doubts about its efficacy. Rhucin is also under development for follow-on indications, i.e. antibody-mediated rejection (AMR) and delayed graft function (DGF) following kidney transplantation.