Pharming, Santarus Get Refusal-to-File Letter for Rhucin

The FDA has declined to review Pharming and Santarus’ BLA for their hereditary angioedema treatment Rhucin. The companies submitted the BLA in late December, using data from two randomized, placebo-controlled studies demonstrating safety and efficacy of the drug in doses of 50 units/kg and 100 units/kg. The FDA says in its letter the trials did not provide data from a sufficient number of patients to support the 50 U/kg dose strength. The BLA also lacked prospective validation of the visual analog scale measuring the clinical effects of Rhucin (recombinant human C1 inhibitor).
Drug Industry Daily