Fit Factor: Inspiring Fitness Stories to Get You Going in 2018

January 9, 2018

Below, we’ll introduce you to three such people: Trevor Dunn, a young man with von Willebrand Disease, Dawn Evans, an adult woman who is asymptomatic carrier of Hemophilia A, and Barry Haarde, an adult male with severe Hemophilia A.  All of them have discovered a form of physical activity that is enjoyable and adaptable to […]

Catalyst Biosciences Initiates Phase 2 of Potential Hemophilia A and B Therapy

January 5, 2018

Note: The following is an excerpt from an article by Rare Disease Report. Read the full article in its entirety here. On Jan. 4, Catalyst Biosciences announced the initiation and open enrollment of the Phase 2 part of its Phase 2/3 program of marzeptacog alfa (activated) (MarzAA), a highly potent, subcutaneously administered Factor VIIa therapy in development for the treatment […]

BioMarin’s Investigational Gene Therapy for Hemophilia A Maintains Average Factor VIII Levels within Normal Range for over One Year

July 11, 2017

Note: The following is an edited version of a press release from BioMarin. Read the press release from BioMarin in it’s entirety here. BioMarin Pharmaceutical Inc. announced an update to its previously reported interim results of an open-label Phase 1/2 study of BMN 270, an investigational gene therapy treatment for severe hemophilia A. The updated results were presented […]

FDA Accepts Bioverativ’s Application for Investigational Hemophilia A Drug

June 12, 2017

Bioverativ announced on June 12, 2017 that the U.S. Food and Drug Administration (FDA) has accepted the company’s Investigational New Drug (IND) application for BIVV001 (also known as rFVIIIFc-VWF-XTEN), a novel, investigational factor VIII therapy designed to potentially extend protection from bleeds with prophylaxis dosing of once weekly or longer for people with hemophilia A. […]

Shire’s Adynovate Receives Two New FDA Approvals for Treatment of Hemophilia A

December 29, 2016

Note: The following is edited from a press release from Shire. Read the full press release in it’s entirety here. Shire plc recently announced that the U.S. Food and Drug Administration (FDA) has approved Adynovate, an extended circulating half-life recombinant Factor VIII treatment for hemophilia A, in pediatric patients under 12 years of age. The FDA […]

Octapharma Announces Two Hemophilia A Clinical Trials Utilizing Wilate

November 16, 2016

The following is excerpted from a press release from Octapharma USA. Read the press release in it’s entirety here.  Octapharma USA has announced that WILATE®, von Willebrand Factor/Coagulation Factor VIII Complex (Human) Lyophilized Powder for Solution for Intravenous Injection will be utilized for two important clinical trials for hemophilia A launching in December. Octapharma USA […]

FDA Approves First Single Chain Product for Hemophilia A

May 27, 2016

Note: The below is an edited version of a press release by CSL Behring. The original release can be read in it’s entirety here. KING OF PRUSSIA, Pa., May 26, 2016 — CSL Behring announced today that the U.S. Food and Drug Administration (FDA) has approved AFSTYLA [Antihemophilic Factor (Recombinant), Single Chain], its novel long-lasting recombinant factor […]

Emicizumab Study Results Released in New England Journal of Medicine

May 26, 2016

Note: The New England Journal of Medicine released an article outlining the results of a study of a new drug, Emicizumab (ACE910, Chugai Pharmaceuticals), that shows reduced bleeding in patients with severe hemophilia A. Read the full article, Factor VIII–Mimetic Function of Humanized Bispecific Antibody in Hemophilia A, in the New England Journal of Medicine. This video […]

FDA Approves Needle-Free Transfer Device for Hemophilia A Product

March 31, 2016

Note: The following in an edited press release from Kedrion Biopharma. The original release can be read in its entirety here. Kedrion Biopharma has gained approval from the U.S. Food and Drug Administration to package Koāte® Double Viral Inactivation (DVI) Antihemophilic Factor (human) with Mix2Vial™, a needle-free transfer device. The new packaging is designed to offer hemophilia […]

FDA Approves New Recombinant Factor Following LEOPOLD Trials

March 17, 2016

Note: The following is an edited version of a press release from Bayer. The original press release can be found here.  The U.S. Food and Drug Administration (FDA) has approved Bayer’s KOVALTRY® Antihemophilic Factor (Recombinant), an unmodified, full-length factor VIII compound for the treatment of hemophilia A in children and adults. The approval is based […]

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