Biotech firms Santarus and Pharming Group NV announced yesterday that they have reached agreement with the FDA on a Special Protocol Assessment (SPA) regarding design of their Phase III clinical study of their investigational drug Rhucin. The drug has already gained European approval (as Ruconest) for treatment of acute angioedema episodes in HAE (hereditary angioedema) patients, a genetic disorder.
The Special Protocol Assessment (SPA) process is a streamlined method on the path to approval in which the FDA gives official guidance on the design of proposed drug trials. Or, as one Minyanville.com commentator succinctly put it, in explaining SPAs, “An SPA should eliminate most of the ability of FDA statisticians to nitpick about the design of the trial.” In effect, because FDA has pre-approved the protocol, its number-crunchers have a hard time attacking it later.
However, an SPA agreement is by no means a get-out-of-jail-free card for automatic approval. Final marketing approval still requires efficacious results, a low adverse event profile and a positive evaluation of the risk/benefit factor of treatment.
HAE causes swelling of tissues, and is particularly threatening when it affects airways, causing asphyxiation, potential organ damage, or death. According to the companies’ joint press release, the U.S. Hereditary Angioedema Association estimates that HAE occurs in the general population at the rate of one in 10,000 – 50,000 individuals. The disorder occurs because affected individuals lack a functional plasma protein C1 inhibitor.
Rhucin‘s evaluation is being done via an international, multicenter, randomized, placebo-controlled Phase III study. The study began in February 2011, but the FDA requested the companies to modify the study’s protocols. Principal study changes include increasing the number of patients from 50 to about 75, and allowing use of open-label doses of Rhucin as a rescue medication. The companies continue to expect to complete the study by Q3 2012.
Pharming chief medical officer Dr. Rienk Pijpstra commented, “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.”
Pharming has granted certain exclusive rights to Santarus to market Rhucin in North America for the treatment of HAE and other future uses. Under the license terms, Pharming will receive a $10 million milestone payment from Santarus on successfully reaching the study’s primary endpoint.
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