Inspiration Biopharmaceuticals' OBI-1 Receives Orphan Drug Designation in Europe
10/20/2010 7:45:18 AM
LAGUNA NIGUEL, Calif., Oct. 19 /PRNewswire/ -- Inspiration Biopharmaceuticals, Inc. (Inspiration) announced today that the European Commission has granted orphan drug status for OBI-1 for the treatment of hemophilia. Expected to enter pivotal clinical trials before the end of this year, OBI-1 is designed to treat individuals with hemophilia who have developed inhibitors against human Factor VIII (hFVIII). The orphan drug status would trigger a 10-year market exclusivity for OBI-1 in the European Union after its marketing approval. The U.S. Food & Drug Administration (FDA) issued an Orphan Drug Designation for OBI-1 in March 2004.
Michael Griffith, Ph.D., President and CEO of Inspiration, commented, "OBI-1 provides a unique, alternative approach to treating individuals with hemophilia who develop an immune reaction to human FVIII. The orphan drug designation in Europe is another sign of the overall progress of our program to develop OBI-1 and address a clear, unmet medical need among individuals with hemophilia." According to a partnership agreement signed with Ipsen SA (Euronext: IPN; ADR: IPSEY) in January 2010, Inspiration licensed OBI-1 from Ipsen and is responsible for the OBI-1 clinical trial development and commercialization.
Hemophilia 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 annual market for hemophilia treatments is $7.5 billion worldwide.
Approximately one-third of individuals with hemophilia A develop an immune reaction (inhibitors) to hFVIII and can no longer respond to treatment with the coagulation factor. Current therapies, specifically FVIIa and FEIBA, work by bypassing the natural hemostatic pathway and forcing coagulation with much higher levels of FVIIa than normal. In contrast, OBI-1, a recombinant form of porcine FVIII that typically possesses low cross reactivity to anti-hFVIII antibodies, is a replacement therapy, activating the natural hemostatic pathway. This would allow clinicians to correlate activity and efficacy with a biomarker and therefore guide dosing and better predict treatment outcomes. OBI-1 presents a unique and alternative approach to address the needs of individuals who have developed inhibitors to FVIII and is highly desired by the medical and patient communities.
OBI-1 has been evaluated in a Phase 2 study in patients with congenital hemophilia A who have developed inhibitors to FVIII and who presented with a non life/non limb threatening bleed. The Phase 2 study demonstrated OBI-1 was well-tolerated and can be given over a short infusion time. Inspiration expects to initiate a pivotal trial of OBI-1 in individuals with acquired hemophilia in the fourth quarter of 2010 and a separate pivotal trial in individuals with congenital hemophilia who have developed inhibitors against hFVIII in the first half of 2011.
About Inspiration Biopharmaceuticals
Inspiration Biopharmaceuticals is dedicated exclusively to developing treatments for hemophilia, with a primary mission to broaden access to care, including prophylactic therapy, and improve the treatment of individuals with inhibitor complications. Inspiration has a broad development portfolio, which includes advanced, clinical-stage product candidates for the treatment of hemophilia A and hemophilia B.
Inspiration's lead product candidates are IB1001, an intravenous recombinant factor IX (FIX) for the acute and preventative treatment of bleeding in individuals with hemophilia B, and OBI-1, an intravenous porcine recombinant factor VIII (FVIII) for the treatment of individuals who have developed inhibitors against human FVIII and for individuals with acquired hemophilia A. IB1001 currently is in Phase 3 clinical development, and OBI-1 is expected to begin the first of two pivotal trials later this year. Earlier-stage preclinical programs at Inspiration are focused on human recombinant factor VIIa (FVIIa) for individuals with either hemophilia A or hemophilia B who have developed inhibitors and individuals with factor VII deficiency, and human recombinant FVIII to treat individuals with hemophilia A.
Inspiration's team has extensive expertise and background in hemophilia product development, biologics manufacturing, regulatory approval and global commercialization; the Company's senior leadership was directly responsible for the development and commercialization of the majority of hemophilia products currently on the market. In addition, in January 2010, Inspiration entered into a strategic partnership with Ipsen SA, leveraging the combined expertise and resources of the two companies. For further information on Inspiration, please visit http://www.inspirationbio.com.
SOURCE Inspiration Biopharmaceuticals, Inc.