konijnenmelkertbaan schreef op 26 september 2012 12:43:
De acceptatie van het BLA-dossier door de FDA zal niet zo lang duren als wat hier genoemd wordt, want er is een versnelde procedure afgesproken, waarbij Pharming tussentijds heeft afgestemd met de FDA. Weten we het nog?
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 US$10 million milestone is payable to Pharming upon successful achievement of the primary endpoint of the Phase III clinical study.
About the Special Protocol Assessment Process
The Special Protocol Assessment (SPA) process is a procedure by which the FDA provides official evaluation and written guidance on the design of proposed protocols that are intended to form the basis for a BLA or New Drug Application (NDA). Final marketing approval depends on the results of efficacy, the adverse event profile and an evaluation of the benefit/risk of treatment demonstrated in all the data contained in the BLA or NDA submission.