Specialty biopharmaceutical company Santarus, Inc. and biotech company Pharming Group NV announced that Pharming has begun an international, multicenter, randomized, placebo-controlled phase III b clinical study evaluating the investigational drug RHUCIN (recombinant human C1 inhibitor) for the treatment of acute attacks of angioedema in patients with Hereditary Angioedema (HAE).

Pharming expects to enroll approximately 50 patients in the study, which may provide additional data, if required by the US Food and Drug Administration (FDA), in support of an approval for RHUCIN at the 50 U/kg dose. Data from the study will also be used to provide additional validation of the visual analogue scale used in measuring the clinical effects of RHUCIN. The study is expected to be completed in 12 to 18 months.

The safety and efficacy of RHUCIN for the treatment of HAE attacks were previously evaluated in two randomized placebo-controlled studies and 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 in the European Union for RUCONEST (RHUCIN in non-European territories) for the treatment of acute angioedema attacks in patients with HAE. Pharming submitted a Biologics License Application (BLA) for RHUCIN to the FDA in late December 2010. In total, the BLA dossier included 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, Pharming is responsible for conducting and paying for the current clinical study with RHUCIN in the treatment of acute attacks of angioedema in patients with HAE.

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 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 US Hereditary Angioedema Association, epidemiological estimates for HAE range from one in 10,000 to one in 50,000 individuals.

Pharming Group NV is developing innovative products for the treatment of unmet medical needs. RUCONEST (RHUCIN in non-European territories) is a recombinant human C1 inhibitor approved for the treatment of angioedema attacks in patients with HAE in all 27 EU countries plus Norway, Iceland and Liechtenstein.

The product is also under development for follow-on indications, i.e. Antibody-Mediated Rejection (AMR) and Delayed Graft Function (DGF) following kidney transplantation. Pharming's advanced technologies include innovative platforms for the production of protein therapeutics, technology and processes for the purification and formulation of these products.

Santarus, Inc. is a specialty biopharmaceutical company focused on acquiring, developing and commercializing proprietary products that address the needs of patients treated by physician specialists.