December 28, 2010 08:00 AM Eastern Time
Santarus and Pharming Announce Submission of RHUCIN Biologics License Application to FDA
SAN DIEGO & LEIDEN, Netherlands--(BUSINESS WIRE)--Specialty biopharmaceutical company Santarus, Inc. (NASDAQ: SNTS) and biotech company Pharming Group NV (NYSE Euronext: PHARM) today announced the submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) to obtain marketing approval for RHUCIN® (recombinant human C1 inhibitor) for the treatment of acute angioedema attacks in patients with Hereditary Angioedema (HAE).
The safety and efficacy of RHUCIN for the treatment of HAE attacks were evaluated in two randomized placebo-controlled studies and are supported by four open label treatment studies. Both placebo-controlled clinical studies showed statistically significant and clinically relevant improvement in the primary endpoint of time to beginning of relief of symptoms at RHUCIN dosage strengths of 50 U/kg and 100 U/kg compared to placebo. In October 2010, Pharming received Marketing Authorization for RUCONEST™ (RHUCIN in non-European countries) for the treatment of acute HAE in the European Union. Pharming has updated the clinical dataset reviewed and approved by the European Medicines Agency with additional patient data and analyses. In total, the BLA dossier includes nine clinical studies covering 714 administrations in 190 subjects.
Santarus has licensed certain exclusive rights from Pharming to commercialize RHUCIN in North America for the treatment of acute attacks of HAE and other future indications. Under the terms of the license agreement, a $5 million milestone is payable to Pharming upon FDA acceptance for review of the BLA for RHUCIN.
About RHUCIN (RUCONEST in European countries) and Hereditary Angioedema
RHUCIN (INN conestat alfa) is a recombinant version of the human protein C1 inhibitor (C1INH). RHUCIN is produced through Pharming’s proprietary technology in milk of transgenic rabbits and in Europe is approved under the name RUCONEST for treatment of acute angioedema attacks in patients with HAE. The FDA has granted Orphan Drug and Fast Track Status to RHUCIN for the treatment of acute attacks of HAE, a genetic disorder in which the patient is deficient in or lacks a functional plasma protein C1 inhibitor, resulting in unpredictable and debilitating episodes of intense swelling of the extremities, face, trunk, genitals, abdomen and upper airway. The frequency and severity of HAE attacks vary and are most serious when they involve laryngeal edema, which can close the upper airway and cause death by asphyxiation. According to the U.S. Hereditary Angioedema Association, epidemiological estimates for HAE range from one in 10,000 to one in 50,000 individuals. Based on prior discussions with the FDA, Pharming is planning to initiate an additional randomized placebo-controlled, clinical study with RHUCIN in approximately 50 patients to provide additional data in support of the 50 U/kg dose. Data from the placebo-controlled study will also be used to provide additional validation of the visual analog scale used in measuring the clinical effects of RHUCIN.