Hemophilia Federation of America is a national nonprofit organization that assists, educates and advocates for the bleeding disorders community.
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 […]
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 […]
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 […]
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, […]
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 […]
The following is an excerpt from a press release from Spark Therapeutics. Spark Therapeutics, a member of the Roche Group and a fully integrated, commercial gene therapy company dedicated to challenging the inevitability of genetic disease, announced updated data from three dose cohorts of the ongoing Phase 1/2 clinical trial of investigational SPK-8011 in hemophilia […]
The following is an excerpt from a press release from uniQure. Read the press release in its entirety here. uniQure N.V., a gene therapy company advancing transformative therapies for patients with severe medical needs, announced that uniQure and CSL Behring have entered into a licensing agreement providing CSL Behring with exclusive global rights to etranacogene […]
The following is an excerpt from a press release from Pfizer. Read the press release in its entirety here. Pfizer Inc. and Sangamo Therapeutics Inc. 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. All five patients with […]
The following is an excerpt from a press release from Sanofi. Read the press release in its entirety here. Sanofi and Sobi™, together with the World Federation of Hemophilia (WFH) and WFH USA, announced an extension of their support of the WFH Humanitarian Aid Program with an additional donation of up to 500 million IUs […]
The following is an excerpt from a press release from BioMarin. Read the press release in its entirety here. BioMarin Pharmaceutical Inc. announced an update to its previously reported results of an open-label Phase 1/2 study of valoctocogene roxaparvovec, an investigational gene therapy treatment for adults with severe hemophilia A. The data have been submitted […]
The following is from a press release from Dr. Reddy’s Laboratories. Read the press release in its entirety here. Dr. Reddy鈥檚 Laboratories Ltd., along with its subsidiaries together referred to as 鈥淒r. Reddy鈥檚鈥 today announced the launch of Desmopressin Acetate Injection USP, 4 mcg/mL Single-dose Ampules, a therapeutic equivalent generic version of DDAVP (desmopressin acetate) […]
Novo Nordisk recently distributed a notice relevant to its product Rebinyn庐,聽raising awareness that one-stage clotting assay results can be significantly affected by the type of activated partial thromboplastin time (aPTT) reagent used, which can result in over or under-estimation of Factor IX activity. The notice which was addressed to HTCs, physicians and other healthcare providers, […]
The following is an excerpt from a press release from uniQure. Read the press release in its entirety here. uniQure N.V., a gene therapy company advancing transformative therapies for patients with severe medical needs, presented new preclinical data on its gene therapy candidates AMT-150 for Spinocerebellar Ataxia type 3, AMT-190 for Fabry disease, and AMT-180 […]
The following is a press release from Pfizer. Read it in its entirety here. Pfizer Inc. and BioNTech SE announced the first participants have been dosed in the U.S. in the Phase 1/2 clinical trial for the BNT162 vaccine program to prevent COVID-19. The trial is part of a global development program, and the dosing […]
The following is an excerpt from a press release from uniQure. Read the release in its entirety here. uniQure N.V., a gene therapy company advancing transformative therapies for patients with severe medical needs, reported recent progress across its business. 鈥淒espite the ongoing global crisis caused by the coronavirus pandemic, we ended the first quarter of […]
The following information is provided by BioMarin to provide general information about its ongoing clinical trials in the context of the COVID-19 pandemic.聽 “As more cases of COVID-19 are confirmed, we recognize the increased challenges and concerns faced by participants in the BioMarin Gene Therapy clinical studies. The safety and well-being of patients, healthcare providers, […]
Below is a letter from Takeda. “To our valued patients, customers and other stakeholders,聽 As the COVID-19 crisis continues to unfold, Takeda鈥檚 Patient Assistance Response team is working around the clock to help ensure that the people who need our medicines and treatments can continue to access them safely and without interruption.聽 Consistent with our […]
The following is an update on the Spark and Roche gene therapy program. “Together, Spark and Roche remain fully committed in our objective to meet the needs of the hemophilia community by developing gene therapies for hemophilia A that, with the lowest effective dose and optimal regimen to manage the immune system response, demonstrate safety, […]
Genentech recently distributed a notice relevant to its product Hemlibra庐, reminding health care providers that it is known to interfere with certain laboratory assays used to measure blood coagulation. These tests are often used for patients with bleeding disorders such as hemophilia, and to diagnose and monitor individuals suffering from other conditions that affect coagulation […]
The following is a copy of a letter from Bayer. “For more than 30 years, Bayer has been providing FVIII therapies and support programs to people living with hemophilia A. As we all face the current coronavirus (COVID-19) outbreak, the health and safety of our patients continues to be a priority. We realize you may […]
The following is from a press release featuring various industry partners. See the press release here. Partnership brings together world-leading plasma companies to focus on developing and delivering a hyperimmune immunoglobulin in the global fight against COVID-19 Biotest, BPL, LFB, and Octapharma have joined an alliance formed by CSL Behring and Takeda Pharmaceutical Company Limited […]
The following is a press release from HEMA Biologics. HEMA BiologicsTM Announces FDA Approval of SEVENFACT庐 [coagulation factor VIIa (recombinant)-jncw] for Treatment and Control of Bleeding Episodes Occurring in Adult and Adolescent Hemophilia A and B Patients with Inhibitors Louisville, KY 鈥 April 6, 2020 HEMA Biologics, LLC, (鈥淗EMA Biologics鈥) today announced that the U.S. […]
The following is information shared with the bleeding disorders community by Takeda. “Takeda has been closely monitoring the news surrounding COVID-19. Our highest priority is the health and safety of our community during this uncertain time. We are taking steps to help ensure the community鈥檚 safety including postponing upcoming educational events such as our HELLO […]
The following is information shared by Pfizer Inc. “To avoid adding to the demands on the healthcare system during this unprecedented crisis with COVID-19, we are pausing for three weeks the recruitment portion of our new and ongoing global interventional clinical studies. We are taking this action in the interests of public health, so that […]
The following is a press release from uniQure. uniQure N.V., a leading gene therapy company advancing transformative therapies for patients with severe unmet medical needs, announced it has achieved the targeted dosing of patients in the HOPE-B pivotal trial of etranacogene dezaparvovec (AMT-061), an investigational AAV5-based gene therapy incorporating the patent-protected FIX-Padua variant for the […]
The following is a letter provided by Spark Therapeutics. “In the unprecedented time of the COVID-19 (novel coronavirus) pandemic, Spark Therapeutics has closely monitored this evolving situation and taken steps to protect the well-being and safety of both our workforce and the patients and families we serve. In reference to our ongoing gene therapy clinical […]
The following is a letter provided by Octapharma. “As we all manage the challenges presented by the Coronavirus pandemic, Octapharma USA would like to remind you of our commitment to providing our customers life-saving therapies, manufactured to the highest standards of viral clearance and safety.聽 Our staff continues to focus on maintaining production of all […]
The following is a press release from Genentech. “The World Health Organization has declared the COVID-19 (coronavirus) outbreak a global pandemic. We extend our heartfelt sympathies to everyone who has lost loved ones, to patients and healthcare professionals battling this devastating outbreak. With multiple countries implementing measures to slow the spread of the virus, based […]
The following is a press release from Pfizer. Pfizer Confirms BeneFIX庐 and XYNTHA庐 Remain Available Pfizer recognizes the public concern in relation to the COVID-19 pandemic, which continues to evolve. We are actively monitoring the situation across the globe, assessing the implications to our patients, colleagues and company, and taking actions to support continued health […]
The following is a letter from Takeda. 聽“To the bleeding disorders community,聽 As a global pharmaceutical leader, our top priority during the ongoing COVID-19 outbreak is to do all we can to make sure our medicines and services continue to reach the individuals who rely on them, while we protect the health of our employees, […]
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