Pharming, Santarus reach SPA agreement with FDA for RHUCIN Phase III trial

Aug 4 2011

Specialty biopharmaceutical company Santarus, Inc. (NASDAQ: SNTS) and biotech company Pharming Group NV (NYSE Euronext: PHARM) today announced that they have reached agreement with the U.S. Food and Drug Administration (FDA) on the design of a Phase III clinical study with the investigational drug RHUCIN^® (recombinant human C1 inhibitor) under the Special Protocol Assessment (SPA) process that is intended to support the submission of a Biologics License Application (BLA).

RHUCIN is being evaluated for the treatment of acute attacks of angioedema in patients with Hereditary Angioedema (HAE) in an international, multicenter, randomized, placebo-controlled Phase III study at a dosage strength of 50 U/kg with a primary endpoint of time to beginning of relief of symptoms. The Phase III study was initiated in February 2011, but the FDA subsequently requested modifications to the study protocol.

Following discussions with the FDA and implementation of the Agency's recommended changes to the study protocol, the FDA has confirmed that Pharming's proposed trial design, clinical endpoints and statistical analyses are acceptable to the FDA. As a result of the discussions with the FDA, the changes to the study design include, as previously announced, a modification to the way the primary endpoint will be assessed and an increase in the number of patients from 50 to approximately 75. The protocol will also be changed to allow the introduction of open-label doses of RHUCIN as a rescue medication. The study is still expected to be completed by the third quarter of 2012.

"We are pleased to have reached agreement with the FDA under an SPA on the protocol for the Phase III clinical study to support a BLA for RHUCIN in the U.S. Over the past months we have continued to open additional investigational sites and to screen patients for eligibility who can now be randomized into the amended trial," said Rienk Pijpstra, MD, MBA, Chief Medical Officer at Pharming.

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 $10 million milestone is payable to Pharming upon successful achievement of the primary endpoint of the Phase III clinical study.

Source: Santarus, Inc.