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Pharming announces positive results from North American randomized trial with rhucin

18 Jun 2008 - Pharming Group N.V. announced positive safety and efficacy results from its North American trial with RhucinŽ (recombinant human C1 inhibitor) for the acute treatment of Hereditary Angioedema (HAE). The positive results with both primary and secondary endpoints of study allow Pharming to move forward with regulatory filings for RhucinŽ.
 
The randomized double-blind placebo-controlled study with RhucinŽ was conducted at several sites in the US and Canada. On having an acute HAE attack, 39 patients were randomized to one of two doses of RhucinŽ (100 U/kg, 50 U/kg) or placebo. For the primary endpoint, the intent-to-treat analysis demonstrates that patients who received the 100 U/kg dose of RhucinŽ reported median first symptom relief at approximately 68 minutes, those that received the 50 U/kg dose reported relief at approximately 100 minutes, and those that received placebo reported symptom relief at approximately 258 minutes. The time to minimal clinical symptoms, the secondary endpoint, was reported by patients in the 100 U/kg, 50 U/kg, placebo groups as approximately 245, 247, and 1098 minutes, respectively. The primary and secondary endpoint results with both doses of RhucinŽ were clinically meaningful and statistically significant relative to placebo with p-values < 0.01. There was no statistically significant difference observed between patients treated with 100 U/kg or 50 U/kg dose of RhucinŽ.
 
The North American randomized results with RhucinŽ are in line with the positive results from the European randomized placebo-controlled Phase III clinical trial reported previously. The safety experience from the trial indicates that RhucinŽ continues to be very well tolerated and no relevant, treatment-related adverse events were reported in any of the three treatment groups. Preliminary data from the ongoing open-label phase of the study, including treatment of over 100 attacks, further demonstrates that RhucinŽ is safe and effective when treating patients for HAE attacks on a repeated basis. Data on treating repeat attacks represents an important addition to the database and will strengthen upcoming filings. The full results of the open label studies in the US and EU will be reported separately.
 
Based on these results, Pharming intends to file regulatory submissions for RhucinŽ in several early launch markets in the second half of 2008 and will meet with EMEA and FDA to accelerate regulatory filings in Europe and the USA.