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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聽here. Sigilon Therapeutics聽has announced it has received Orphan Drug Designation for SIG-001, an investigational therapy for hemophilia A that leverages Sigilon鈥檚 Shielded Living Therapeutics™聽platform to implant cells engineered to produce stable blood plasma levels of factor VIII, a crucial […]

Joint Statement #4 on Recall of Bayer Kogenate庐 FS Lots

August 22, 2019

Hemophilia Federation of America and National Hemophilia Foundation continue to follow up on issues arising from Bayer鈥檚 distribution of mislabeled, expired clotting factor and subsequent recall of that product. The most recent set of joint HFA-NHF inquiries to Bayer focused on the short- and long-term medical consequences for affected patients. Bayer today released this response […]

Joint Statement #3 on Recall of Bayer Kogenate庐 FS Lots

August 12, 2019

We recognize the recent recall from Bayer has caused deep concern and has been unsettling for many in the bleeding disorders community. As part of our ongoing discussion with Bayer, Hemophilia Federation of America and National Hemophilia Foundation have received the following letter and update regarding the recall of Kogenate FS. This letter includes Bayer鈥檚 […]

Joint Statement #2 on Recall of Bayer Kogenate庐 FS Lots

July 31, 2019

Bayer announced the recall of two lots of Kogenate庐聽FS antihemophilic factor (recombinant) 2000 I.U. vials on July 19, 2019. Bayer announced that 3000 I.U. of one product (Jivi), past its expiration date, had been mislabeled as another product (2000 I.U. of Kogenate FS) and distributed to the public for six months. This is a deeply […]

Joint Statement on Recall of Bayer Kogenate庐聽FS Lots

July 26, 2019

Last week, Bayer announced the recall of two lots of Kogenate庐聽FS antihemophilic factor (recombinant) 2000 IU vials.That highly concerning announcement has raised many questions on the part of both national organizations and community members. The fact that one product (Jivi), past its expiration date, was mislabeled as another product (Kogenate FS) and distributed to the […]

RECALL NOTICE: Bayer Voluntarily Recalling Factor 8 Product

July 19, 2019

The following is a press release from Bayer regarding a voluntary recall. PATIENTS SHOULD NOT USE THIS PRODUCT! Bayer is voluntarily recalling two lots of Kogenate庐 FS antihemophilic factor (recombinant) 2000 IU vials in the United States to the patient level. Certain vials from these two lots that were labeled as Kogenate FS actually contain […]

SPECIAL PRESIDENT AND CEO ANNOUNCEMENT

July 16, 2019

Dear Bleeding Disorders Community 鈥 I want to let you know that I will be stepping down from HFA as your President and CEO at the end of August. Leaving HFA has been an incredibly difficult decision I have been wrestling with for about a year.聽 I love HFA but ultimately this is just the […]

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庐 (emicizumab-kxwh) across multiple pivotal studies in people with hemophilia A with and without factor VIII inhibitors at the International Society on Thrombosis and […]

uniQure Announces Follow-Up Data from Study in Patients with Hemophilia B

July 10, 2019

The following is an excerpt from a press release from uniQure. Read the press release in its entirety here. uniQure, a gene therapy company advancing transformative therapies for patients with severe medical needs, announced updated clinical data on the three patients treated in the company鈥檚 ongoing Phase IIb study of AMT-061, an investigational AAV5-based gene […]

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庐 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庐 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 Presents Updated Clinical Data on Gene Therapy for Hemophilia B

May 10, 2019

The following is an excerpt from a press release from uniQure. Read the full press release here. uniQure聽announced updated clinical data in patients treated in the company鈥檚 ongoing Phase IIb study of AMT-061, an investigational AAV5-based gene therapy containing a patent-protected FIX-Padua variant, for the treatment of patients with severe and moderately severe hemophilia B. […]

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聽American Society […]

Recall Notice: Fentanyl Transdermal Pain Patches Recalled Due to Product Mislabeling

April 23, 2019

The following is from the FDA MedWatch. Read the recall notice in its entirety here. Alvogen Inc. is voluntarily recalling two lots of Fentanyl Transdermal System 12 mcg/h transdermal patches to the consumer level. A small number of cartons labeled 12 mcg/h Fentanyl Transdermal System patches contained 50 mcg/h patches. The 50 mcg/h patches that […]

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 […]

Spark Therapeutics Enters into Merger Agreement with Roche

February 25, 2019

The following is an excerpt from a press release from Spark Therapeutics. Read the press release in its entirety here. Spark Therapeutics , a fully integrated, commercial gene therapy company dedicated to challenging the inevitability of genetic disease, announced today that it has entered into a definitive merger agreement for Roche to fully acquire Spark […]

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 […]

uniQure Announces Updated Clinical Data from Phase IIb Study in Patients with Hemophilia B

February 8, 2019

The following is an excerpt from a press release from uniQure. Read the full press release here. uniQure,聽a leading gene therapy company advancing transformative therapies for patients with severe unmet medical needs, announced updated clinical data in patients treated in the company鈥檚 ongoing Phase IIb study of AMT-061, an investigational AAV5-based gene therapy containing a […]

Roche joins the World Federation of Hemophilia Humanitarian Aid Program

February 7, 2019

The following is an excerpt from a press release from Roche. Read it in its entirety here. Roche today announced that 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 […]

uniQure Announces First Patient Treated in HOPE-B Pivotal Trial of AMT-061 in Patients with Hemophilia B

February 6, 2019

The following is an excerpt from a press release from uniQure. Read the full press release here. uniQure, a leading gene therapy company advancing transformative therapies for patients with severe unmet medical needs, today announced that it treated the first patient in its HOPE-B pivotal trial of AMT-061, an investigational AAV5-based gene therapy incorporating the […]

Takeda Completes Acquisition of Shire, Becoming a Global, Values-based, Biopharmaceutical Leader

January 23, 2019

The following is a press release from Takeda, formerly Shire. Find the original press release here. Takeda Pharmaceutical Company Limited announced the completion of its acquisition of Shire, becoming a global, values-based, R&D-driven biopharmaceutical leader headquartered in Japan. Takeda now has an attractive, expanded geographic footprint and leading position in Japan and the U.S., bringing […]

Save One Life Inc. Welcomes Chris Bombardier as New Executive Director

January 16, 2019

The following is an excerpt from a press release from Save One Life Inc. Read the press release in its entirety here.聽 Save One Life Inc. announces a new executive director. Save One Life is an international nonprofit that assists people with hemophilia in developing countries. Christopher G. Bombardier, famous for being the first person […]

Plasma Protein Therapeutics Association Welcomes New President and CEO

January 16, 2019

  The following is an excerpt from the Plasma Protein Therapeutics Association. Read the full press release here. The Board of Directors of the Plasma Protein Therapeutics Association announced Amy Efantis will be its next President and Chief Executive Officer effective Jan. 16, 2019. Having served most recently as Vice President, Global Public Policy & […]

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庐 prophylaxis in children younger than 12 years of age with hemophilia A with factor […]

Sangamo Announces Treatment Of First Patient In Clinical Trial Of In Vivo Genome Editing Therapy For Hemophilia B

December 20, 2018

The following is an excerpt from a press release from Sangamo. Read the press release in its entirety here. Hemophilia B is a rare, genetic bleeding disorder caused by a lack of the factor IX (FIX) protein, which is necessary for normal blood clotting. Current hemophilia B treatments typically require frequent intravenous infusions of clotting […]

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庐聽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.,聽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 […]


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