Pharming: US approval in sight
09.11.2012 - Dutch Pharming and its US-partner Santaris Inc. have reported positive Phase III results for Ruconest, their treatment of hereditary angioedema.
The development of Pharming's C1 inhibitor Rhucin, also called Ruconest outside Europe, seemed to be a never ending story. But after the Dutch company and its US-partner Santaris Inc. reported positive top-line data for the protein to treat hereditary angioedema, the story could come to a happy ending.
Rhucin met the primary endpoint of reducing median time to beginning of symptom relief vs. placebo in the Phase III Study 1310 trial to treat acute attacks of hereditary angioedema (HAE). The positive data trigger a US$10m milestone payment to Pharming from Santarus, which has exclusive commercialisation rights to Rhucin in North America. "We are very pleased with these pivotal study results and look forward to working with our colleagues at Santarus to prepare and submit the Biologics License Application (BLA) for Ruconest to the FDA in the first half of 2013," said Bruno Giannetti, COO of Pharming. "We anticipate that additional data from this Phase III study will be presented at an appropriate medical meeting in 2013." Last year, FDA refused a similar application. Pharming subsequently amended the protocol to address issues raised by FDA. The recombinant human complement 1 (C1) esterase inhibitor is approved as Rhucin in the EU to treat acute attacks of HAE and is co-marketed with Swedish Orphan Biovitrum (SOBI). Originally, Pharming had begun the first Phase III for Rhucin in April 2004.
Rhucin has exactly the same amino acid sequence as endogenous human C1 inhibitor protein. It is the second recombinant protein produced in transgenic animals to get to market in Europe, following the approval of ATryn, a recombinant antithrombin, extracted from transgenic goat milk, in 2006. In Europe, there is significant competition in the HAE space. Aside from a C1 inhibitor protein derived from human plasma, the main competition for Rhucin on the continent is Shire’s approved Firazyr, a bradykinin B2 receptor antagonist originally developed by German Jerini AG, which delivered sales of about US$10 million in 2010. Firazyr was the first new treatment for HAE in Europe in 30 years.