Donate

Industry News

Sanofi presents amended protocols in fitusiran clinical studies

February 24, 2021

The following is an excerpt from a press release from Sanofi. Read the full press release here. The amended protocol being implemented for all ongoing adult and adolescent fitusiran clinical studies was presented at the 14th Annual Congress of the European Association for Hemophilia and Allied Disorders (EAHAD). Fitusiran is an investigational, subcutaneously administered small […]

Octapharma Announces Final Data from Study on the Immunogenicity in Previously Untreated Patients with Severe Hemophilia A

February 18, 2021

The following is an excerpt from a press release from Octapharma. Read the full press release here. Octapharma announced the final results from the NuProtect study on the immunogenicity of Nuwiq庐 in previously untreated patients with severe hamophilia A have been published in the medical journal Thrombosis and Haemostasis (Liesner RJ et al. 鈥淪imoctocog Alfa […]

Update Regarding Availability of Stimate Nasal Spray

February 4, 2021

Ferring Pharmaceuticals has provided an update to HFA and NHF regarding the availability of Stimate庐 (desmopressin acetate, 1.5 mg/1 mL) Nasal Spray (鈥淪timate鈥).聽 The letter addresses the investigation completed by Ferring; the investigation determined that the “underlying聽cause for the out-of-specification results as an issue with the tightness of the seal of the bottle.” Furthermore, the […]

RECALL NOTICE: VOLUNTARY RECALL MONONINE COAGULATION FACTOR IX (HUMAN)

January 21, 2021

CSL Behring has announced a voluntary聽pharmacy level聽product recall of聽one batch of Mononine Coagulation Factor IX (Human), a plasma-derived product used to treat hemophilia B. CSL Behring鈥檚 notification states, 鈥淧atients can continue to use product they may have. Although the potential for safety risk to patients is considered low, it cannot be fully excluded.鈥 The affected […]

Baxject II Device Currently Out of Stock

January 13, 2021

HFA has received the following information from Takeda regarding the Baxject II Device: The Baxject II device, that comes in a 5 unit blister pack and may be ordered separately from Takeda products, is currently out of stock due to circumstances that are not safety related. This does not impact BaxJect II devices that are […]

uniQure Announces Clinical Hold on Hemophilia B Gene Therapy Program

December 21, 2020

The following is an excerpt from a press release from uniQure. Read the update in its entirety聽here. Today uniQure announced a clinical hold by the U.S. Food and Drug Administration (FDA) in its聽phase III HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients. Per uniQure鈥檚 statement: 鈥淭he clinical hold was initiated following […]

Sanofi to Resume Dosing in Fitusiran Clinical Studies in the U.S.

December 11, 2020

The following is a press release from Sanofi. Read the press release in its entirety here. Sanofi will resume fitusiran dosing in ongoing U.S. adolescent and adult clinical studies. Fitusiran is an investigational, small interference RNA therapy in development for the treatment of people with hemophilia A or B, with or without inhibitors. Sanofi鈥檚 first […]

Pfizer and Sangamo Announce Updated Results in Hemophilia A Gene Therapy

December 10, 2020

The following is an excerpt from a press release from Pfizer. Read the full press release here. Pfizer Inc. and Sangamo Therapeutics Inc., a genomic medicines company, announced updated follow-up data from the Phase 1/2 Alta study of giroctocogene fitelparvovec (SB-525 or PF-07055480), an investigational gene therapy for patients with severe hemophilia A. These data […]

uniQure Presents Data from HOPE-B Pivotal Gene Therapy Trial in Patients with Hemophilia B at ASH Annual Meeting

December 10, 2020

The following is an excerpt from a press release from uniQure. Read the full press release here. uniQure,聽a gene therapy company advancing transformative therapies for patients with severe medical needs, today announced the late-breaking presentation of initial data from its pivotal, Phase III HOPE-B gene therapy trial of etranacogene dezaparvovec, an investigational adeno-associated virus five […]

Pfizer Doses First Participant In Phase 3 Study for People With Severe Hemophilia A and B with or Without Inhibitors

November 25, 2020

The following is from a press release from Pfizer. Read it in its entirety here. Pfizer Inc. announced that the first participant has been dosed in the Phase 3 BASIS study of marstacimab (PF-06741086), an anti-tissue factor pathway inhibitor (anti-TFPI) being evaluated for the treatment of people with severe hemophilia A or B, with or […]

uniQure Announces Positive Data from the HOPE-B Pivotal Trial Gene Therapy in Patients with Hemophilia B

November 25, 2020

The following is an excerpt from a press release from uniQure. Read the press release in its entirety here. uniQure N.V.,聽a gene therapy company advancing transformative therapies for patients with severe medical needs, announced positive top-line data from its pivotal, Phase III HOPE-B gene therapy trial of etranacogene dezaparvovec, an investigational adeno-associated virus five (AAV5)-based […]

Octapharma announces FDA approval of updated NUWIQ庐 Prescribing Information to include immunogenicity data in previously untreated patients

October 23, 2020

The following is an excerpt from a statement from聽Octapharma. Read the statement in its entirety聽here. LACHEN, Switzerland鈥斅燨ctapharma is pleased to announce that the U.S. Food and Drug Administration (FDA) has approved an updated Prescribing Information (PI) for NUWIQ庐, Octapharma鈥檚 human cell line-derived recombinant factor VIII (FVIII). NUWIQ聽庐聽is approved for the prevention and treatment of bleeding […]

Pfizer and Sangamo Dose First Participant in Phase 3 Study Evaluating Hemophilia A Gene Therapy Treatment

October 7, 2020

The following is an excerpt from a statement from Pfizer. Read the statement in its entirety聽here. AFFINE is a global Phase 3, open-label, multicenter, single arm study that will evaluate the efficacy and safety of giroctocogene fitelparvovec in patients with moderately severe to severe hemophilia A. The primary endpoint is impact on annual bleed rate […]

CSL Behring Provides Update on Stimate Nasal Spray Availability

October 2, 2020

The following is an excerpt from an update from CSL Behring regarding the availability of Stimate Nasal Spray. Read the update in its entirety here. Ferring Pharmaceuticals Inc. initiated a voluntary, consumer-level recall of all batches of Stimate庐 Nasal Spray (desmopressin acetate) 1.5 mg/mL, which is distributed and sold by CSL Behring LLC.聽 At this […]

An update from Genentech on Hemlibra庐 post-approval data communication process and site update

September 24, 2020

The following is an excerpt from a statement from Genentech. Read the statement in its entirety聽here. To the hemophilia community, At Genentech, we recognize and appreciate the ongoing conversation within the hemophilia community around the long-term efficacy and safety of Hemlibra庐 (emicizumab-kxwh). We understand that the subject of long-term safety for hemophilia treatments is an […]

New England Journal of Medicine publishes positive final results from Phase 1/2a study of BIVV001 in people with severe hemophilia A

September 17, 2020

On September 10th, The New England Journal of Medicine聽published positive final results from the Phase 1/2a trial evaluating the safety, tolerability and pharmacokinetics of BIVV001 (rFVIIIFc-VWF-XTEN) in adult patients with severe hemophilia A. BIVV001 is an investigational factor VIII therapy designed to provide higher bleed protection in a once-weekly prophylactic treatment regimen. Sanofi and Sobi™ […]

CSL Behring Announces Discontinuation of Mononine Coagulation Factor IX

September 15, 2020

CSL Behring issued the following statement to patient advocacy groups: “CSL Behring is focused on delivering breakthrough therapies to improve patients鈥 lives and meet the changing needs of our patients. Since CSL Behring鈥檚 introduction of MONONINE庐 Coagulation Factor IX (Human) in 1992, advancements have been made for the treatment of Hemophilia B.聽 Over time, patients […]

HFA, NHF, Hemophilia Alliance Request Additional Information Regarding Stimate Recall

September 14, 2020

Hemophilia Federation of America and the National Hemophilia Foundation continue to follow up on issues arising from the recent recall of Stimate (desmopressin) nasal spray, which is manufactured by Ferring Pharmaceuticals and distributed in the United States by CSL Behring. On September 3, HFA, NHF and the Hemophilia Alliance sent a new joint letter to […]

Recall Notice: Mylan Initiates Voluntary Nationwide Recall of Four Lots of Tranexamic Acid Injection, USP Due to Carton Label Mix-Up

September 2, 2020

On September 1, 2020, HFA and NHF learned that Mylan N.V. is conducting a voluntary nationwide recall of聽 four lots of Tranexamic Acid Injection, USP 1000 mg/10 mL, packaged in cartons of 10 single-dose 10 mL vials. Tranexamic acid injection is indicated in patients with hemophilia for short term use to reduce or prevent hemorrhage […]

Genentech Provides Update to US District Court Decision

August 31, 2020

The following is an excerpt from an update from Genentech. Read the update in its entirety here. Genentech has provided a statement surrounding an August 27, 2020 US Federal Circuit Court of Appeals ruling which 聽reverses a previous ruling by the US District Court. In December 2018, the US District Court issued a patent interpretation […]

Genentech Releases Update on Particles Identified in Hemlibra

August 31, 2020

The following is an excerpt from a statement from Genentech. Read the statement in its entirety here. Genentech has announced an update on measures that are being taken in relation to the translucent particles that were identified in Hemlibra庐 (emicizumab-kxwh) in 2019 during a routine examination of product batches as part of quality assurance processes. […]

Takeda Suspends Screening & Further Enrollment in TAK-754, TAK-748 Clinical Studies

August 21, 2020

The following is an excerpt from an update from Takeda. Read the update in its entirety here. Takeda recently informed HFA of it’s decision聽to suspend screening and further enrollment in聽the TAK-754 and TAK-748 clinical studies, effective immediately. TAK-754 is an AAV8 gene therapy for the treatment of hemophilia A currently in Phase 1/2 clinical聽development, while […]

Novo Nordisk Resumes Clinical Trials Investigating Concizumab in Hemophilia A and B With or Without Inhibitors

August 20, 2020

The following is an excerpt from a press release from Novo Nordisk. Read the press release in its entirety here. Novo Nordisk announced the clinical trials in the concizumab phase 3 programme (explorer 6, 7 and 8) are being resumed. The clinical trials are investigating subcutaneous concizumab prophylaxis treatment in hemophilia A and B patients […]

HFA Responds to News of FDA Action on BioMarin Gene Therapy Application

August 19, 2020

Click Below to Translate Page to Spanish. Today BioMarin announced that the U.S. Food and Drug Administration has issued a letter indicating that BioMarin鈥檚 investigational gene therapy for severe hemophilia A is not ready for approval in its present form. BioMarin says the FDA has asked for two years of follow-up data showing evidence of […]

BioMarin Receives Complete Response Letter (CRL) from FDA for Gene Therapy for Severe Hemophilia A

August 19, 2020

Click Below to Translate Page to Spanish. BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that the U.S. Food and Drug Administration (FDA) issued a Complete Response Letter (CRL) to the Company’s Biologics License Application (BLA) for valoctocogene roxaparvovec gene therapy for severe hemophilia A on聽August 18, 2020. 聽The FDA issues a CRL to indicate that […]

Stimate Recall – CSL Behring & Ferring Pharmaceuticals Announces Extension of Stimate Recall to Consumer Level

August 13, 2020

Ferring Pharmaceuticals announced it was extending the recall of Stimate (desmopressin) to the consumer/user level. Stimate is manufactured by Ferring Pharmaceuticals Inc. and is distributed and sold in the United States by CSL Behring. This marks an expansion of Ferring鈥檚 initial July 2020 plans to conduct a narrower pharmacy-level recall. Under a consumer-level recall, people […]

Stimate Recall – CSL Behring & Ferring Pharmaceuticals Response to HFA-NHF Letter

August 7, 2020

  Click Below to Translate Page to Spanish. On July 21, 2020, Ferring Pharmaceuticals issued a voluntary recall of Stimate (desmopressin) nasal spray due to 鈥渟uperpotency鈥 鈥 in other words, amounts of desmopressin being higher than specified. Stimate is used to treat von Willebrand disease (VWD) and mild hemophilia A. It is licensed and distributed […]

Joint Statement on Recall of Stimate

July 22, 2020

Click Below to Translate Page to Spanish. On July 21, CSL Behring and Ferring Pharmaceuticals announced the recall of multiple lots of Stimate (desmopressin)聽in the United States, as part of a global recall. This聽troubling聽announcement has raised many questions on the part of national and international patient organizations and community members. Today, HFA and NHF have聽submitted […]

RECALL NOTICE: Voluntary Recall of Stimate (Desmopressin Acetate)

July 21, 2020

Click Below to Translate Page to Spanish. Late today (July 21, 2020), HFA and NHF learned of a聽pharmacy level聽product recall of聽Stimate (desmopressin)聽nasal spray manufactured by Ferring Pharmaceuticals,聽and聽distributed in the U.S. by CSL Behring. Ferring has prepared a Health Hazard Safety Evaluation, warning that 鈥渢here is a reasonable probability that the use of, or exposure to, […]

New Data From Study Reinforces Safety Profile of Genentech’s Hemlibra庐 in People with Hemophilia A

July 16, 2020

The following is a press release from Genentech. Genentech, a member of the Roche Group announced results from the second interim analysis of the Phase IIIb STASEY study, which reinforce the safety profile of Hemlibra庐 (emicizumab-kxwh) characterized in the Phase III HAVEN clinical program. In the STASEY study, Hemlibra was effective with no new safety […]


Sign up for E-mails, Dateline Magazine, and other ways to stay connected.