Sigilon Therapeutics Announces Clinical Hold on SIG-001 Phase 1/2 Study in Hemophilia A
The following is sourced from a press release from Sigilon Therapeutics. Read the update in its entirety here. The U.S. Food and Drug Administration (FDA) has placed a clinical hold on Sigilon Therapeutics’ 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 amounts […]
BioMarin: Clinical Development Program Update
Update for Hemophilia Associations BioMarin is pleased to update the community regarding our 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 Update For Patient Associations View PDF | Download PDF […]
CSL Behring Announces Closing of Global Commercialization and License Agreement with uniQure for etranacogene dezaparvovec
Read the full press release from CSL Behring here. Global biotherapeutics leader CSL Behring 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 to result in near-normal […]
uniQure Announces Closing of Commercialization and License Agreement with CSL Behring
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
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’s hemophilia B gene therapy program after determining the Company satisfactorily addressed all issues identied by the FDA related to […]
uniQure Releases Findings in Hemophilia B Gene Therapy Program
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 […]
Sanofi Presents Amended Protocols in Fitusiran Clinical Studies
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
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® in previously untreated patients with severe hamophilia A have been published in the medical journal Thrombosis and Haemostasis (Liesner RJ et al. “Simoctocog Alfa […]
Update Regarding Availability of Stimate Nasal Spray
Ferring Pharmaceuticals has provided an update to HFA and NHF regarding the availability of Stimate® (desmopressin acetate, 1.5 mg/1 mL) Nasal Spray (“Stimate”). The letter addresses the investigation completed by Ferring; the investigation determined that the “underlying cause for the out-of-specification results as an issue with the tightness of the seal of the bottle.” Furthermore, the letter […]
Recall Notice: Voluntary Recall Mononine Coagulation Factor IX (Human)
CSL Behring has announced a voluntary pharmacy level product recall of one batch of Mononine Coagulation Factor IX (Human), a plasma-derived product used to treat hemophilia B. CSL Behring’s notification states, “Patients can continue to use product they may have. Although the potential for safety risk to patients is considered low, it cannot be fully excluded.” The affected […]
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