OBI-1 Developed By Ipsen And Inspiration Has Obtained A Positive Opinion For The Orphan Drug Status In Europe

Ipsen (Euronext : FR0010259150; IPN) and Inspiration Biopharmaceuticals, Inc. (Inspiration) announced that the Committee for Orphan Medicinal Products of the European Medicines Agency has issued a positive opinion on the granting of orphan drug status for OBI-1 for the treatment of hemophilia. Final adoption of the opinion is expected from the European Commission later this year and subject to it being finally granted, the orphan drug status would trigger a 10-year market exclusivity to OBI-1 in the European Union after its marketing approval. The FDA also issued an Orphan Drug Designation for OBI-1 in March 2004.

Jean-Luc Bélingard, Chairman and Chief Executive Officer of Ipsen said: “Our transaction with Inspiration in late January of this year expresses Ipsen’s long term strategy to create a world leading hemophilia franchise. We are honored that the Committee for Orphan Medicinal Products of the European Medicines Agency shares our view of the medical benefit provided by OBI-1 to the hemophilia community.”

John Taylor, Co-Founder and Chairman of Inspiration added: “We are pleased with the continued progress of OBI-1 as a new, innovative therapy in the treatment of unmet medical needs in hemophilia.”

About Hemophilia

Hemophilia, congenital or acquired, is a bleeding disorder caused by low levels or absence of a protein called a coagulation factor, essential for blood clotting. The two most common forms of hemophilia are types A and B. Hemophilia A is caused by a factor VIII deficiency and occurs in ~1 out of every 5,000 male births. Hemophilia B is caused by factor IX deficiency and occurs in ~1 out of every 30,000 male births. Approximately 60% of persons with hemophilia have a severe condition, which results in frequent spontaneous bleeding episodes in addition to serious bleeding after injuries. The market for hemophilia treatment is 7.5 billion dollars annually.

About OBI-1

About a third of patients with congenital hemophilia A and patients with acquired hemophilia develop an immune reaction to human forms of FVIII (hFVIII) and can no longer respond to human Factor VIII. Since OBI-1 possesses low cross reactivity to anti-hFVIII antibodies, it is expected that OBI-1 can provide therapeutic benefits to patients who are not able to use hFVIII.

OBI-1, a recombinant B-domain deleted FVIII bioengineered for low cross reactivity to anti-human FVIII inhibitors based on the porcine amino acid sequence, has recently been tested in a Phase II trial. OBI-1 was administered to patients with congenital hemophilia A complicated by the presence of human FVIII inhibitors experiencing a non-life/non-limb threatening bleed. A total of 25 bleeding episodes in 9 patients were treated with OBI-1, and all were successfully controlled. One subject had a mild infusion reaction and when re-treated for a subsequent bleed the subject did not report any adverse event. Eight out of nine (89%) subjects developed anti-pFVIII antibodies following exposure to OBI-1 and in subjects receiving repeated OBI-1 treatment higher anti-pFVIII titres did not affect efficacy or safety. The study demonstrated that OBI-1 is well-tolerated and can be given as a short infusion. OBI-1 is expected to enter phase III in 2010.

Inspiration Biopharmaceuticals

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