FDA Approves Advate to Treat Hemophilia A

The U.S. Food and Drug Administration (FDA) approved Baxter International Inc.’s Advate (antihemophilic factor [recombinant] plasma/albumin free method) for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in patients with hemophilia A. Advate is the only antihemophilic factor approved in the U.S. for prophylactic use in both adults and children.

The approval is based on a Phase IV prophylaxis study that demonstrated a statistically significant reduction in the median annual bleeding rate. Patients receiving on-demand treatment experienced 44 bleeds (per patient per year) compared with one bleed (per patient per year) while on either of the prophylactic regimens evaluated (a 98 percent reduction in annual bleed rate). Forty-two percent of study patients experienced zero bleeds during one year on prophylaxis. And, of the two prophylactic regimens approved for use, the dosing schedule of every three days (a pharmacokinetic-driven regimen based on patients’ clinical response) offered some patients the option of fewer infusions over one year of treatment.

Hemophiliacs Eligible for College Scholarships

College students with hemophilia or a related bleeding disorder and their family members are eligible to apply for the Eric Dostie Memorial College Scholarship. The scholarship will award $1,000 each to 10 students who can best demonstrate scholastic achievement, community service and financial need. Applicants must submit an essay describing how his or her education will be used to serve humankind and to encourage self-improvement and enrichment.

The annual scholarship is made possible by FFF Enterprises Inc., Temecula, Calif., the nation’s leading distributor of critical-care plasma products, antihemophilic factors and preventive vaccines, and was created to honor the memory of Eric Steven Dostie of Easthampton, Massachusetts, a five-year-old boy with hemophilia, was tragically murdered on August 27, 1994. Eric’s brief life forever touched many others with joy, humor and unending love. Eric used to tell his parents and grandparents that he might grow up to be a scientist and invent a cure for hemophilia in the form of a “chocolate pill.” Although Eric’s dream will never be realized, it has the chance to live on in the recipients of the award, who pursue a college degree to broaden their education and career opportunities.

To be eligible to apply, students must be citizens of the United States and enrolled full-time in an accredited two- or four-year college program. The scholarship application deadline is March 1, 2011. An application can be accessed at www.NuFACTOR.com.

Gene Therapy Tested for Hemophilia B

Amsterdam Molecular Therapeutics has begun a Phase I/II exploratory clinical trial with a gene therapy product for hemophilia B. The trial is an open-label dose-escalation study using a vector-gene combination developed at St. Jude Children’s Research Hospital. The hemophilia B gene therapy, administered once, will introduce the functional gene for the Factor IX protein into the patient’s liver cells with the goal to restore blood clotting functionality long-term. In pre-clinical studies, Factor IX gene therapy resulted in long-term production of Factor IX protein at a therapeutically significant level after a single administration.

If this approach is successful, the long-term efficacy of one-time administered hemophilia B gene therapy is expected to be perceived as a significant advance over the current regular dosing of recombinant Factor IX. In addition, the efficacy profile of this gene therapy is anticipated to exceed that of current therapy, as the gene therapy will lead to stable Factor IX levels, whereas recombinant protein treatment causes peaks and troughs.

Coagulation Factor R&D Report Published

A new report, titled Coagulation Factors 2009: Target Pipeline and Corporate Benchmark Analysis, analyzes and assesses the target pipeline for each of the coagulation factors used for systemic and topical administration. Companies active in the therapeutic coagulation business are evaluated, and the strengths, weaknesses, opportunities and threats in their R&D pipeline are benchmarked in the respective peer group. Technologies used for creation of next generation coagulation factors also are discussed. More information about the report is available from Research and Markets at www.researchandmarkets.com.