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Biomarin: Clinical Development Program Update

July 20, 2021

The following is sourced from BioMarin. Read the update in its entirety “BioMarin Hemophilia A Clinical聽Development Program: An Update for the Hemophilia Community” View PDF | Download PDF BioMarin Hemophilia A Clinical Development Program: An Update for the Hemophilia Community BioMarin is pleased to update the community regarding our ongoing gene therapy clinical trial program […]

Sigilon Therapeutics Announces Clinical Hold on SIG-001 Phase 1/2 Study in Hemophilia A

July 12, 2021

The following is sourced from a press release from Sigilon Therapeutics. Read the update in its entirety聽here. The U.S. Food and Drug Administration (FDA) has placed a clinical hold on Sigilon Therapeutics鈥 phase 1/2 study of SIG-001 in patients with severe or moderately severe hemophilia A. SIG-001 is a cell-based therapy, designed to carry significant […]

BioMarin: Clinical Development Program Update

July 2, 2021

BioMarin is pleased to update the community regarding our ongoing gene therapy clinical trial program in hemophilia A. BioMarin’s investigational gene therapy for hemophilia A has not been approved for use; it is in ongoing clinical trials evaluating its safety and efficacy. BioMarin Hemophilia A Clinical聽Development Program: An Update for the Hemophilia Community View PDF […]

BioMarin: Clinical Development Program Update

May 21, 2021

Update for Hemophilia Associations BioMarin is pleased to update the community regarding our gene therapy clinical trial program in hemophilia A. BioMarin鈥檚 investigational gene therapy for hemophilia A has not been approved for use; it is in ongoing clinical trials evaluating its safety and efficacy. BioMarin Hemophilia Update For Patient Associations View PDF | Download […]

CSL Behring Announces Closing of Global Commercialization and License Agreement with uniQure for etranacogene dezaparvovec

May 7, 2021

Read the full press release from CSL Behring here. Global biotherapeutics leader CSL Behring 聽announced the closing of its global Commercialization and License agreement with uniQure for etranacogene dezaparvovec, a novel gene therapy for the treatment of hemophilia B. Etranacogene dezaparvovec is currently in Phase 3 clinical trials and has been shown聽 to result in […]

uniQure Announces Closing of Commercialization and License Agreement with CSL Behring

May 6, 2021

Read the original press release here uniQure, a leading gene therapy company advancing transformative therapies for patients with severe medical needs, announced that the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976 for the global licensing agreement with CSL Behring for etranacogene dezaparvovec, an investigational gene therapy for patients with hemophilia B, expired […]

uniQure Announces FDA Removes Clinical Hold on Hemophilia B Gene Therapy Program

April 26, 2021

Read the full release here. uniQure, a leading gene therapy company advancing transformative therapies for patients with severe medical needs, announced that the U.S. Food and Drug Administration (FDA) has removed the clinical hold on the Company鈥檚 hemophilia B gene therapy program after determining the Company satisfactorily addressed all issues identied by the FDA related […]

uniQure Releases Findings in Hemophilia B Gene Therapy Program

March 29, 2021

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 medical needs, announced the results of a comprehensive investigation into the case of hepatocellular carcinoma (HCC) diagnosed in one patient in the HOPE-B pivotal trial […]

BioMarin Provides Update to Gene Therapy Clinical Trial Program in Hemophilia A

March 16, 2021

BioMarin is pleased to update the community regarding our gene therapy clinical trial program in hemophilia A. BioMarin鈥檚 investigational gene therapy for hemophilia A has not been approved for use; it is in ongoing clinical trials evaluating its safety and efficacy. Clinical Trial Overview BioMarin鈥檚 valoctocogene roxaparvovec, is currently being studied in adults with severe […]

Genentech Issues Update to Warning and Precautions Section of Hemlibra Label

March 15, 2021

In Genentech鈥檚 ongoing efforts to transparently communicate with the hemophilia community, they have shared two updates to the Hemlibra label that were requested by the U.S. Food and Drug Administration. These changes, which were accepted and implemented on March 10, 2021, impact the Warnings and Precautions (sections 5.1 and 5.2) and Clinical Pharmacology (section 12.3) […]

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 […]

Genentech Launches New Hemophilia Website

September 25, 2020

The following information has been provided by Genentech. “Genentech recently launched a new聽website聽GenentechHemophilia.com, dedicated to supporting the Hemophilia community. Here you can watch videos, read articles and learn how Genentech is committed to advancing all aspects of life with hemophilia A. Genentech supports the Hemophilia community through a variety of ways including a web series […]

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 […]


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