Category: Industry News

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

The following is an excerpt from a statement from Genentech. Read the statement in its entirety 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® (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

On September 10th, The New England Journal of Medicine 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

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

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

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 CSL […]

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

On September 1, 2020, HFA and NHF learned that Mylan N.V. is conducting a voluntary nationwide recall of  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

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  reverses a previous ruling by the US District Court.In December 2018, the US District Court issued a patent interpretation judgment […]

Genentech Releases Update on Particles Identified in Hemlibra

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® (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

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 to suspend screening and further enrollment in 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 development, while TAK-748 […]

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

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

Click Below to Translate Page to Spanish. [prisna-google-website-translator]Today BioMarin announced that the U.S. Food and Drug Administration has issued a letter indicating that BioMarin’s 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 […]