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FDA Identifies Biomarker for Immune Response to FVIII Products Used to Treat Hemophilia A

September 17, 2019

The following is a press release from the FDA. Read the press release on the FDA website¬†here. Scientists at the U.S. Food and Drug Administration developed a technique that enables them to determine whether immune system cells called dendritic cells appear primed to trigger the production of antibodies against Factor VIII (FVIII) products used to […]

Sigilon Therapeutics Receives Orphan Drug Designation for a Treatment of Hemophilia A

September 6, 2019

The following is a press release from Sigilon Therapeutics. Read the press release on the Sigilon website¬†here. Sigilon Therapeutics¬†has announced it has received Orphan Drug Designation for SIG-001, an investigational therapy for hemophilia A that leverages Sigilon‚Äôs Shielded Living Therapeutics™¬†platform to implant cells engineered to produce stable blood plasma levels of factor VIII, a crucial […]

Genentech Presents Data for Hemlibra at the ISTH 2019 Congress

July 10, 2019

The following is an excerpt from a press release from Genentech. Read the press release in its entirety here. Genentech, a member of the Roche Group, announced today new data for Hemlibra¬ģ (emicizumab-kxwh) across multiple pivotal studies in people with hemophilia A with and without factor VIII inhibitors at the International Society on Thrombosis and […]

Octapharma Presents New Data at the International Society on Thrombosis and Haemostasis 2019 Congress

July 10, 2019

The following is an excerpt from a press release from Octapharma. Read the press release in its entirety here. Octapharma announced that new data on the benefits of Nuwiq¬ģ in patients with hemophilia A were presented during a scientific symposium at the 27th International Society on Thrombosis and Haemostasis Congress in Melbourne, Australia. The symposium, […]

Sangamo and Pfizer Announce Updated Results of Investigational Hemophilia A Gene Therapy

July 9, 2019

The following is an excerpt from a press release from Sangamo. Read the press release in its entirety here. Sangamo Therapeutics Inc., a genomic medicine company, and Pfizer Inc. announced updated results from the Phase 1/2 Alta study evaluating investigational SB-525 gene therapy for severe hemophilia A. The data showed that SB-525 was generally well-tolerated […]

Bayer to present new data at Congress of International Society on Thrombosis and Haemostasis

June 27, 2019

The following is an excerpt of a press release from Bayer. Read the press release in its entirety here. Bayer will present new data highlighting clinical outcomes from its hemophilia A portfolio, which include long-term data from the PROTECT VIII investigational study evaluating use of Jivi¬ģ antihemophilic factor (recombinant) PEGylated-aucl. These data will be presented […]

BioMarin Announces Update to Hemophilia A Clinical Development Program

May 28, 2019

The following is an excerpt from a press release from BioMarin. Read the press release in its entirety here. BioMarin Pharmaceutical Inc. announced its investigational gene therapy, valoctocogene roxaparvovec, for adults with severe hemophilia A achieved pre-specified clinical criteria for regulatory review in the U.S. and Europe. As of May 28, 2019, eight patients in […]

uniQure Announces New Preclinical Data in Hemophilia A and Fabry Disease

May 2, 2019

The following is an excerpt from a press release from uniQure. Read the press release in its entirety here. uniQure N.V., a gene therapy company, presented new preclinical data on its gene therapy candidates for the treatment of hemophilia A and for the treatment of Fabry disease during back-to-back oral presentations at the 22nd¬†American Society […]

Sangamo and Pfizer Announce Phase 1/2 Interim Data for Investigational Hemophilia A Gene Therapy

April 15, 2019

The following is an excerpt from a press release from Sangamo Therapeutics. Read the press release in its entirety here. Sangamo Therapeutics Inc., a genomic medicine company, and Pfizer Inc. announced interim data from the Phase 1/2 Alta study evaluating investigational SB-525 gene therapy for severe hemophilia A. Data indicate that SB-525 was generally well-tolerated […]

Genentech joins the World Federation of Hemophilia Humanitarian Aid Program

February 21, 2019

The following is a press release from Roche. Read the press release in its entirety here. Roche announced it has joined the World Federation of Hemophilia Humanitarian Aid Program, a landmark initiative leading the effort to change the lack of access to care and treatment for people with inherited bleeding disorders in developing countries. Together […]

Novo Nordisk Receives FDA Approval for Hemophilia A Treatment

February 20, 2019

The following is an excerpt from a press release. Read the press release in its entirety here. Denmark-based Novo Nordisk received approval from the U.S. Food and Drug Administration for a new treatment for hemophilia A. However, the newly approved medication will not be available in the United States until 2020. Novo Nordisk said the […]

Genentech’s Hemlibra Provided Sustained Bleed Control in a Study of Children with Hemophilia A with Factor VIII Inhibitors

December 20, 2018

The following is an excerpt from a press release from Genentech. Read the press release in its entirety here. Genentech, a member of the Roche Group announced data from the primary analysis of the Phase III HAVEN 2 study evaluating Hemlibra¬ģ prophylaxis in children younger than 12 years of age with hemophilia A with factor […]

Genentech’s Hemlibra Provided Sustained Bleed Control in the Largest Pivotal Study in Children with Hemophilia A with Inhibitors

December 5, 2018

The following is an excerpt from a press release from Genentech. Read the press release in its entirety here. Genentech, a member of the Roche Group, announced data from the primary analysis of the Phase III HAVEN 2 study evaluating Hemlibra¬ģ¬†prophylaxis in children younger than 12 years of age with hemophilia A with factor VIII […]

Bioverativ Data Presented at ASH Underscore Potential for Once Weekly Dosing with Sustained High Factor Levels in Hemophilia A

December 5, 2018

The following is an excerpt from a press release from Bioverativ. Read the press release in its entirety here. Bioverativ Inc.,¬†a Sanofi company dedicated to transforming the lives of people with rare blood disorders, presented new data from the EXTEN-A Phase 1/2a trial of BIVV001 showing that a single 65 IU/kg dose of BIVV001 extended […]

uniQure Highlights Pipeline Expansion and Advancements in Technology at Research and Development Day

November 28, 2018

The following is a press release from uniQure. uniQure, a gene therapy company advancing transformative therapies for patients with severe medical needs, announced the expansion of its research pipeline with novel AAV gene therapy approaches to treating Hemophilia A, Fabry disease and Spinocerebellar Ataxia Type 3 at the Company‚Äôs Research & Development Day held this […]

FDA Approves Genentech’s Hemlibra

October 4, 2018

The following is an excerpt from a press release from Genentech. Read the entire press release here. Genentech, a member of the Roche Group, announced that the U.S. Food and Drug Administration has approved Hemlibra¬ģ(emicizumab-kxwh) for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children, ages newborn and older, […]

Bayer Receives FDA Approval for Jivi, New Hemophilia A Treatment With Step-Wise Prophylaxis Dosing Regimen

August 31, 2018

The following is an excerpt from a press release from Bayer. Read the press release in its entirety here.¬† Bayer announced that the U.S. Food and Drug Administration has approved Jivi¬ģ for the routine prophylactic treatment of hemophilia A in previously-treated adults and adolescents 12 years of age or older. The initial recommended prophylactic regimen […]

U.S. District Court in Delaware rules against Shire in preliminary injunction

August 8, 2018

On Aug. 7, 2018, the U.S. District Court in Delaware ruled against Shire in its request for a preliminary injunction against Roche AG from creating, transporting and marketing its recently-approved drug Hemlibra in the U.S., which received FDA approval to treat people with hemophilia A with inhibitors in 2017. This ruling means there are no […]

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

January 9, 2018

Below, we‚Äôll 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‚Äôs 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‚Äôs 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‚Äôs 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¬ģ, 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‚ÄďMimetic 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¬†KońĀte¬ģ Double Viral Inactivation (DVI) Antihemophilic Factor (human)¬†with Mix2Vial‚ĄĘ, 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‚Äôs KOVALTRY¬ģ Antihemophilic Factor (Recombinant), an unmodified, full-length factor VIII compound for the treatment of hemophilia A in children and adults. The approval is based […]

Trial for SubQ Hemophilia Inhibitor Treatment Begins

March 14, 2016

Note: The below is an edited press release from Alnylam Pharmaceuticals, Inc. The original release can be read in its entirety¬†here.¬† Alnylam Pharmaceuticals, Inc. announced on Monday, March 14, 2016, that it has initiated dosing of hemophilia patients with inhibitors in Part D of an ongoing Phase 1 clinical trial evaluating a once-monthly subcutaneous dose […]

FDA Gives Orphan Drug Designation for Hemophilia A Gene Therapy

March 1, 2016

Note: The below is an edited version of a press release by BioMarin Pharmaceutical Inc. The original release can be read in its entirety here. BioMarin Pharmaceutical Inc. announced on Tuesday, March 1, that BMN 270, an investigational gene therapy for the treatment of patients with hemophilia A, has been granted orphan drug designation by […]


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