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CSL Behring Update COVID-19

March 13, 2020

CSL Behring has added a statement to their website regarding the measures the company is taking in the wake of the聽2019-nCoV (novel coronavirus) outbreak.

VONVENDI Recall 鈥 Takeda Response

March 6, 2020

As previously reported by HFA and NHF, Takeda announced the recall of two lots of VONVENDI von Willebrand factor (recombinant) 1,300 I.U. vials on Feb. 25, 2020. HFA and NHF recognize that recalls can be very unsettling for many in the bleeding disorders community. On Feb. 27, 2020, the patient organizations announced聽they had submitted a […]

Aptevo Therapeutics Sells IXINITY Hemophilia B Therapy to Medexus Pharmaceuticals

March 6, 2020

The following is an excerpt from a press release from Aptevo Therapeutics. Read the press release in its entirety here. Aptevo Therapeutics Inc. , a biotechnology company focused on developing novel immuno-oncology therapeutics based on its proprietary ADAPTIR™ bispecific technology platform, announced it has completed the sale of its marketed recombinant factor IX therapeutic, IXINITY庐 […]

Bayer Statement on Availability of Factor VIII Products Amid 2019-nCoV (Novel Coronavirus)

March 5, 2020

Bayer has issued the following statement regarding the supply and availability of Bayer’s Factor VIII/Hemophilia A products in the wake of the聽2019-nCoV (novel coronavirus) outbreak:

Genentech Statement on Hemlibra Supply Amid 2019-nCoV (Novel Coronavirus)

February 28, 2020

Genentech has issued the following statement regarding the supply and availability of Hemlibra in the wake of the聽2019-nCoV (novel coronavirus) outbreak: Please note that HFA does not recommend, endorse or make any representation about the efficacy, appropriateness or suitability of any specific products, treatments, or opinions. If you have any questions or concerns about your […]

JOINT STATEMENT ON RECALL OF VONVENDI LOTS

February 27, 2020

Takeda announced the recall of two lots of VONVENDI vonWillebrand factor (recombinant) 1,300 I.U. vials on February 25, 2020. HFA and NHF recognize that recalls can be very unsettling for many in the bleeding disorders community. We are in communications with Takeda to obtain additional detailed and timely information regarding the events that led up […]

Takeda Issues Pharmacy Level Recall of VONVENDI

February 26, 2020

Late yesterday (February 25, 2020), Takeda announced a pharmacy-level recall of two lots of VONVENDI von Willebrand factor (recombinant) 1,300 IU vials. Takeda鈥檚 announcement stated that 鈥淎lthough both lots met all acceptance criteria, Takeda is issuing a voluntary recall out of an abundance of caution. Takeda believes that despite this voluntary recall that [sic] there […]

Novo Nordisk launches ESPEROCT庐 in the U.S. for the treatment of people with hemophilia A

February 11, 2020

The following is an excerpt from a press release from Novo Nordisk. Read the press release in its entirety here. Novo Nordisk announced ESPEROCT庐 [antihemophilic factor (recombinant), glycopegylated-exei] is now available in聽the U.S.聽for the treatment of adults and children with hemophilia A. ESPEROCT庐 is a recombinant extended half-life factor VIII replacement therapy used to prevent […]

BioMarin Announces Gene Therapy Program Updates

January 23, 2020

The following is an announcement from BioMarin. BioMarin announced an update to the community regarding our ongoing gene therapy聽clinical trial program in hemophilia A. BioMarin鈥檚 investigational gene therapy for聽hemophilia A has not been approved for use; it is in ongoing clinical trials evaluating聽its safety and efficacy. Clinical Trial Overview BioMarin鈥檚 investigational gene therapy valoctocogene roxaparvovec […]

Sangamo and Pfizer Announce Updated Results in Gene Therapy Treatment

December 18, 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 follow-up results from the Phase 1/2 Alta study evaluating investigational SB-525 gene therapy in patients with severe hemophilia A. The data showed that SB-525 […]

First Patient Dosed in Phase 3 of Study in Patients with Severe Hemophilia A

December 12, 2019

The following is an excerpt from a press release from Sanofi Genzyme. Read the press release in its entirety here. Sanofi and SobiTM聽announced the first patient has been dosed in the Phase 3, open-label, interventional study of BIVV001 (rFVIIIFc-VWF-XTEN), in patients with severe hemophilia A (XTEND-1 study; NCT04161495). Sobi and Sanofi are development partners for […]

BioMarin Hemophilia A Clinical Development Program Update

November 21, 2019

The following is a press release from BioMarin. 聽BioMarin is pleased to update the community regarding our gene therapy clinical trial program in hemophilia A.聽 Clinical Trial Update聽 BioMarin鈥檚 investigational gene therapy valoctocogene roxaparvovec, is currently being studied in adults with severe hemophilia A.聽 The first Phase 1/2 study was initiated in 2015 and consists […]

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聽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鈥檚 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鈥檚 statement, and see images of the product box,聽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鈥檚 recent announcement regarding reports of particulate matter in vials of Hemlibra. You can read comments on the聽statement from NHF鈥檚 Medical and Scientific Advisory Council (MASAC). Today HFA and NHF sent Genentech a letter with additional questions, which you […]

FDA Approves Octapharma鈥檚 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庐 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鈥檚 statement below, and can read comments on the statement from NHF鈥檚 Medical and Scientific Advisory Council (MASAC). The events of the past several months, including today鈥檚 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.聽 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聽and pharmacovigilance summary聽to HFA and NHF. Bayer submitted these materials in response to a joint HFA-NHF letter, part of the ongoing dialog over Bayer鈥檚 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聽letter聽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聽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庐 FS Lots

September 23, 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 […]

Genentech Patient Foundation/Medvantx Announce Ancillary Safety Issue

September 21, 2019

HFA has received the聽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 […]


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