Specialty pharmaceutical company Santarus, Inc. and biotech company Pharming Group NV announced receipt of a refusal to file letter from the Food and Drug Administration for Rhucin Biologics License Application submitted by pharming. In the letter the FDA indicated that the BLA was not sufficiently complete to enable a critical examination.Rhucin is a recombinant version of the protein C1 inhibitor . Rhucin has obtained orphan drug designation for the acute treatment of HAE is a genetic disease where the patient is deficient or lack of a functional protein of plasma C1 inhibitor, resulting in unpredictable episodes and debilitating swelling of the extremities, face, trunk , genitals, abdomen and upper airways. The frequency and severity of HAE attacks vary and are more severe when they involve the larynx, which can close the airway and cause death by asphyxiation. According to the American Association hereditary angioedema, epidemiological estimates for the range of 10,000 to 50,000 people HAE in one.
About Rhucin and hereditary angioedema
Next, the researchers plan to compare the results of children treated at trauma centers vs. non-trauma centers, Dr.