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Blood Disorder Web News

ELOCTATE Will Be Available by Prescription in Mid July 2014

You’ll be among the first to know about product availability and updates. ELOCTATE is now FDA approved based on the results from a clinical trial of 164 previously-treated patients. ELOCTATE is a recombinant-DNA-derived, antihemophilic factor indicated in adults and children with Hemophilia A for:

  • Control and prevention of bleeding episodes
  • Perioperative management (surgical prophylaxis)
  • Routine prophylaxis to prevent or reduce the frequency of bleeding episodes.
  • ELOCTATE is not indicated for the treatment of von Willebrand disease.

FDA Approves Bayer’s Kogenate® FS Antihemophilic Factor VIII (recombinant) for Routine Prophylaxis in Adults with Hemophilia A

May 12, 2014 – Bayer HealthCare announced today that the U.S. Food and Drug Administration (FDA) has approved a new indication for Bayer’s Kogenate® FS antihemophilic factor VIII (recombinant), for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults with hemophilia A. The approval is based on data from the SPINART study, in which 84 patients ages 15 to 50 were randomized to either prophylaxis (25 IU/kg three times per week) or on-demand treatment.

Baxter Hemophilia Co-Pay/Co-Insurance Assistance Program

Many members of the Hemophilia community are struggling with the high costs of healthcare. Baxter remains committed to the community and recognizes our responsibility to develop new approaches to help improve access to therapies. The Baxter Hemophilia Co-Pay/Co-Insurance Assistance Program is a financial needs-based initiative which serves the hemophilia community by providing eligible patients with financial support toward the purchase of Baxter factor or inhibitor products.

People who are enrolled in the program will receive support with their insurance expenses specific to co-payment/co-insurance for Baxter Hemophilia treatment for up to 12 months.

Patients are eligible to apply for the program if

They have mild, moderate, or severe Hemophilia A or B, or have an inhibitor

They currently have private health insurance

Patients are not eligible to apply if

  • They are presently on public insurance including, but not limited to, Medicare, Medicaid, TriCare, DOD, and PCIP

New CSL Behring Biotechnology Manufacturing Facility

King of Prussia, PA — 9 May 2014 — CSL Limited (ASX:CSL), parent company of CSL Behring which is based in King of Prussia, PA, today opened the CSL Behring Biotechnology Manufacturing Facility in Melbourne, Australia. The new facility, located adjacent to the site’s manufacturing plant for plasma products, is the centerpiece of CSL’s $250 million expansion at its Broadmeadows site and will play an increasingly important role in the company’s global operations, particularly in the late-stage development of new types of hemophilia products. It is one of the largest and most advanced facilities of its kind in the world and will produce novel recombinant therapies on a large scale for international clinical trials.

  • Leading-edge science at the core of $250 million expansion to drive long-term growth in promising bleeding disorders portfolio
  • CSL Behring to commercialize therapies produced in part at the facility, pending required approvals
  • Opening taking place in advance of World Federation of Hemophilia 2014 Congress in Melbourne, Australia

Baxter Announces Phase III Data Evaluating Prophylaxis Treatment of FEIBA NF for Hemophilia Patients with Inhibitors

Pivotal study to form basis for upcoming biologic license application to the FDA

DEERFIELD, Ill., JANUARY 8, 2013 — Baxter International Inc. (NYSE:BAX) today announced pivotal Phase III study results evaluating the efficacy and safety of routine prophylaxis compared to on-demand treatment of FEIBA NF [Anti-Inhibitor Coagulant Complex], Nanofiltered and Vapor Heated, in patients with hemophilia A or B and inhibitors. Top-line results from the study showed a reduced median annual bleed rate (ABR) from 28.7 during FEIBA NF on-demand treatment to 7.9 during FEIBA NF prophylactic treatment (a 72.5% reduction). The Phase III study will form the basis of a biologics license application (BLA) to be filed with the U.S. Food and Drug Administration (FDA) in the first quarter of 2013.

Read the complete press release >

Bayer Leadership Program

December 28, 2012 — Apply to be an intern through the Bayer Hemophilia Leadership Development Program (BHLDP) and experience a unique opportunity to build foundational leadership skills AND deepen your connection to the hemophilia community!

Bayer is looking for students enrolled full-time in college who are touched by hemophilia and have a strong interest in and commitment to becoming a future leader in the hemophilia community. Interns will travel to Bayer’s U.S. headquarters in New Jersey, where they will spend eight weeks participating in activities that aim to help them grow personally and professionally.

Through the program, selected interns will have the opportunity to:

  • Participate in formal training on communication skills, effective problem solving, leadership and compliance
  • Spend time working with mentors within Bayer to gain insights into the inner workings of the various corporate departments–emphasizing the importance of collaboration
  • Work with hemophilia organizations in the area to learn about the work done to support the hemophilia community and how business professionals can support these efforts
  • Engage in hands on projects that will build on the skills developed during the program

The program will also include a meeting with healthcare public policy professionals in Washington D.C. where interns will see first-hand how effective advocacy relations impacts legislative decisions.

This is an eight-week, paid internship program (June through August 2013). All housing, travel and related activities will be organized and underwritten by Bayer.

Interns will be selected based on an application form and interview process. Applications are due no later than Friday, February 8, 2013, at 11:59 pm ET.

Read the complete article >

Baxter Presents Phase I/III Data Evaluating Safety and Efficacy of BAX 326, An Investigational Recombinant Factor IX for Hemophilia B, Showing More Than 40 Percent of Patients Experienced No Bleeds

BAX 326 Granted Orphan-drug Designation by U.S. FDA

DEERFIELD, Ill., December 10, 2012 — Baxter International Inc. (NYSE:BAX) today announced pivotal Phase I/III study results evaluating the safety and efficacy of BAX 326, an investigational recombinant factor IX (rFIX) protein, for the treatment and prophylaxis of bleeding episodes for patients with hemophilia B over 12 years of age. The data were presented at the 54th Annual Meeting and Exposition of the American Society of Hematology (ASH) in Atlanta. BAX 326 was recently granted orphan-drug designation by the U.S. Food and Drug Administration (FDA), a special status given to a product that when approved, will address an unmet need for people with a rare disease or condition. Data supporting the prophylaxis indication, which is the basis for the orphan drug designation, was included in the biologics license application (BLA).

Read the complete press release >

Novo Nordisk Announces U.S. Results
of Largest Multinational Psychosocial Study of Living with Hemophilia

November 7, 2012 — Orlando, Fla. — U.S. patient and caregiver results derived from the largest multinational psychosocial study of the hemophilia community are being presented at the National Hemophilia Foundation (NHF) 64th Annual Meeting. In total, four posters were accepted from the Hemophilia Experiences, Results and Opportunities (HERO) study, a comprehensive analysis of the experience of living with hemophilia. One of the posters will be featured in an oral presentation at the meeting.

HERO examined the disorder’s effect on interpersonal relationships, careers, access to care and quality of life. The HERO initiative aims to improve outcomes in hemophilia by calling for and enabling enhanced psychosocial support based on increased understanding and awareness of the issues.

“We worked with key physicians and advocacy groups to develop a comprehensive study that, for the first time, examines the impact of living with hemophilia on all aspects of a family’s life, including interpersonal relationships, access to care, employment, and the quality of life,” said Robert Gut, MD, PhD, VP Clinical Development & Medical Affairs Biopharmaceuticals, Novo Nordisk.

In the United States, 189 adults with hemophilia and 190 parents of children with hemophilia participated in the quantitative arm of the HERO research.

Read the complete press release >

Biogen Idec & Sobi Announce Study Results
Positive Top-Line Results From Phase 3 Study Investigating Long-Lasting Recombinant Factor VIII Fc Fusion Protein In Hemophilia A

  • Individualized and weekly prophylactic regimens resulted in low single-digit median annualized bleeding rates
  • 98% of bleeding episodes were controlled with one or two injections of rFVIIIFc
  • No patients developed inhibitors to rFVIIIFc
  • The primary efficacy and safety objectives were met and Biogen Idec plans to submit an application to US FDA in first half 2013

October 31, 2012 — Weston, Mass. & Stockholm, Sweden — Biogen Idec and (Sobi) today announced positive results from A-LONG, a clinical study that evaluated a new long-lasting clotting factor candidate in people with hemophilia A. Hemophilia A is a rare inherited disorder that impairs blood coagulation. Top-line results from A-LONG, a global, multi-center, Phase 3 clinical study of the companies’ long-lasting recombinant Factor VIII Fc fusion protein (rFVIIIFc), showed that rFVIIIFc was effective in the control and prevention of bleeding, routine prophylaxis and perioperative management. Recombinant FVIIIFc was generally well-tolerated. Additional analyses of the A-LONG study are ongoing, and the companies anticipate presenting detailed results at a future scientific meeting.

Biogen Idec plans to submit a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) in the first half of 2013. Consistent with guidelines published by theEuropean Medicines Agency (EMA) that require a study in children less than 12 years of age prior to filing, Biogen Idec and Sobi expect to file a Marketing Authorization Application with the EMA upon completion of the ongoing Kids A-LONG study.

Read the complete press release >

Biogen Idec & Sobi Announce Positive Top-Line Results for Hemophilia B

Biogen Idec and Sobi Announce Positive Top-Line Results from Phase 3 Study Investigating Long-Lasting Recombinant Factor IX Fc Fusion Protein in Hemophilia B

September 26, 2012 — Prophylactic regimens resulted in low single-digit annualized bleeding rates:

  • Median dosing interval was 14 days in the individualized interval prophylaxis arm during the last 6 months on study
  • Greater than 90% of bleeding episodes were controlled by a single injection of rFIXFc
  • No patients developed inhibitors to rFIXFc
  • The primary efficacy and safety objectives were met and Biogen Idec plans to submit a BLA to US FDA in first half 2013

Read the complete press release .

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