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Sangamo to Announce Gene Therapy and Ex Vivo Gene-Edited Cell Therapy Data at ASH

November 7, 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, announced that hemophilia A gene therapy clinical data and hemoglobinopathies ex vivo gene-edited cell therapy data will be featured in poster presentations at the 61st Annual Meeting of the […]

Sigilon Therapeutics to Present Preclinical Data on Rare Blood Disorders at ASH

November 7, 2019

The following is a press release from Sigilon Therapeutics. Sigilon Therapeutics¬†announced it will present data at the American Society of Hematology Annual Meeting demonstrating that its novel Shielded Living TherapeuticsTM platform for rare bleeding disorders remains viable in animal models for at least six months. The data also demonstrate dose-responsive in vivoexpression of human coagulation […]

Genentech Response to HFA-NHF Letter Regarding Particulate Matter in Hemlibra

October 17, 2019

On October 16, 2019, Genentech sent the following letter to HFA and NHF. Genentech‚Äôs letter responds to questions posed in the joint HFA-NHF letter of October 11, regarding reports of particulate matter in vials of Hemlibra. HFA and NHF will keep the bleeding disorders community informed as further information develops. Please note that HFA and […]

CSL Behring Announces Printing Misalignment for Humate-P

October 15, 2019

CSL Behring has advised HFA and NHF of a printing misalignment on the label of its Humate P product for von Willebrand disease. You can read CSL Behring‚Äôs statement, and see images of the product box,¬†below. While the current Humate P notification is prompted by a printing error on the outside of the box, NHF […]

Joint Letter from HFA-NHF to Genentech Regarding Particulate Matter in Hemlibra

October 11, 2019

Hemophilia Federation of America and National Hemophilia Foundation continue to follow up on issues arising from Genentech‚Äôs recent announcement regarding reports of particulate matter in vials of Hemlibra. You can read comments on the¬†statement from NHF‚Äôs Medical and Scientific Advisory Council (MASAC). Today HFA and NHF sent Genentech a letter with additional questions, which you […]

FDA Approves Octapharma’s Wilate for Hemophilia A in Adult and Adolescent Patients

October 9, 2019

The following is a press release from Octapharma. Read the press release in its entirety here. Octapharma USA today announced the U.S. Food and Drug Administration (FDA) has approved WILATE¬ģ for treatment of adults and adolescents with hemophilia A for routine prophylaxis to reduce the frequency of bleeding episodes and on demand treatment and control […]

Genentech Issues Statement on Hemlibra Particles; MASAC Responds

October 7, 2019

Today, Genentech provided the following statement regarding reports of particulate matter in vials of Hemlibra. You can read Genentech‚Äôs statement below, and can read comments on the statement from NHF‚Äôs Medical and Scientific Advisory Council (MASAC). The events of the past several months, including today‚Äôs announcement by Genentech, underscore the importance of the joint Safety […]

HFA & NHF Discuss Bayer Recall with FDA

October 7, 2019

HFA and NHF are pleased to share a brief update with the community concerning a recent meeting we had with the FDA as well as a number of additional resources that the FDA provided after the call.¬† HFA and NHF will continue to update the bleeding disorders community as any further information develops. Please note […]

Bayer Responds to HFA-NHF Letter

September 27, 2019

On Sept. 27, 2019, Bayer sent the following letter¬†and pharmacovigilance summary¬†to HFA and NHF. Bayer submitted these materials in response to a joint HFA-NHF letter, part of the ongoing dialog over Bayer‚Äôs distribution and subsequent recall of two lots of improperly labeled, expired clotting factor. Please note that NHF and HFA do not recommend, endorse […]

Genentech Patient Foundation Responds to HFA-NHF Letter

September 27, 2019

On September 24, 2019, HFA and NHF sent a¬†letter¬†to Genentech and MedVantx regarding the shipment of incorrect length injection needles to patients who receive Hemlibra through the Genentech Patient Foundation. The Genentech Patient Foundation responded to our inquiries by letter dated September 25, 2019. You can read their response¬†here. HFA and NHF will update the […]

Joint Statement #1 on Genentech Patient Foundation/Medvantx Needle Error

September 24, 2019

Late in the day on Friday, September 20, 2019, Genentech informed HFA and NHF that a contracted specialty pharmacy, Medvantx, had shipped incorrect length injection needles to patients who receive Hemlibra through the Genentech Patient Foundation. HFA and NHF posted that notice on September 21st. Today we followed up with a letter to Genentech and […]

Joint Statement #5 on Recall of Bayer Kogenate¬ģ FS Lots

September 23, 2019

Bayer announced the recall of two lots of Kogenate¬ģ¬†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 […]

Genentech Patient Foundation/Medvantx Announce Ancillary Safety Issue

September 21, 2019

HFA has received the¬†following notice from Genentech regarding the shipment of incorrect length injection needles to patients who receive Hemlibra through the Genentech Patient Foundation: “We were recently informed that ancillary supplies shipped by a specialty pharmacy, Medvantx, to a specific group of patients receiving Hemlibra through the Genentech Patient Foundation contained incorrect length injection […]

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 […]

Joint Statement #4 on Recall of Bayer Kogenate¬ģ FS Lots

August 22, 2019

Hemophilia Federation of America and National Hemophilia Foundation continue to follow up on issues arising from Bayer‚Äôs 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¬ģ 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‚Äôs […]

Joint Statement #2 on Recall of Bayer Kogenate¬ģ FS Lots

July 31, 2019

Bayer announced the recall of two lots of Kogenate¬ģ¬†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¬ģ¬†FS Lots

July 26, 2019

Last week, Bayer announced the recall of two lots of Kogenate¬ģ¬†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¬ģ 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 ‚ÄĒ 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.¬† 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¬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 […]

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‚Äôs 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¬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 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¬†announced updated clinical data in patients treated in the company‚Äôs 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¬†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 […]


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