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Genentech to Present New Data at the American Society of Hematology 2018 Annual Meeting

November 7, 2018

  The following is an excerpt from a press release from Genentech. Read the press release in its entirety here. Genentech has announced new data that will be presented at this year’s annual American Society of Hematology meeting held in San Diego from Dec. 1-4. Ten Genentech medicines will be featured in more than 70 abstracts, including […]

FDA Approves Genentech’s Hemlibra

October 4, 2018

The following is an excerpt from a press release from Genentech. Read the entire press release here. Genentech, a member of the Roche Group, announced that the U.S. Food and Drug Administration has approved Hemlibra®(emicizumab-kxwh) for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children, ages newborn and older, […]

LA FDA APRUEBA HEMLIBRA DE GENENTECH

October 4, 2018

El siguiente es un comunicado de prensa proporcionado por Genentech.  LA FDA APRUEBA HEMLIBRA® DE GENENTECH (EMICIZUMAB-KXWH) PARA LA HEMOFILIA A SIN INHIBIDORES DE FACTOR VIII – El primer medicamento que reduce considerablemente la hemorragia tratada en comparación con la previa profilaxis de factor VIII basada en una comparación intrapaciente – – Único medicamento que puede […]

Genentech Announces the Launch of New Safety Website

September 3, 2018

Genentech has announced a new web portal for patients and caregivers intended to provide timely and accurate information on targeted serious adverse events of interest for HEMLIBRA. Visit the safety website: http://www.emipatientinfo.com/. Earlier this year, Genentech launched a similar site specifically for health care providers. In announcing the launch, Genentech stated “This website is meant to […]

Phase III Results for Genentech’s Hemlibra Published in New England Journal of Medicine

August 31, 2018

The following is an excerpt from a press release from Genentech. Read the press release in its entirety here.  Genentech, a member of the Roche Group, announced that pivotal data from the Phase III HAVEN 3 study, which evaluated HEMLIBRA® prophylaxis administered every week or every two weeks in adults and adolescents aged 12 years or […]

Bayer Receives FDA Approval for Jivi, New Hemophilia A Treatment With Step-Wise Prophylaxis Dosing Regimen

August 31, 2018

The following is an excerpt from a press release from Bayer. Read the press release in its entirety here.  Bayer announced that the U.S. Food and Drug Administration has approved Jivi® for the routine prophylactic treatment of hemophilia A in previously-treated adults and adolescents 12 years of age or older. The initial recommended prophylactic regimen […]

uniQure Announces First Patient Treated in Dose-Confirmation Study in Patients with Hemophilia B

August 24, 2018

The following is an excerpt from a press release from uniQure. Read the press release in its entirety here.  uniQure, a leading gene therapy company advancing transformative therapies for patients with severe unmet medical needs, announced it has treated the first patient in its Phase IIb dose-confirmation study of AMT-061, an investigational AAV5-based gene therapy […]

U.S. District Court in Delaware rules against Shire in preliminary injunction

August 8, 2018

On Aug. 7, 2018, the U.S. District Court in Delaware ruled against Shire in its request for a preliminary injunction against Roche AG from creating, transporting and marketing its recently-approved drug Hemlibra in the U.S., which received FDA approval to treat people with hemophilia A with inhibitors in 2017. This ruling means there are no […]

Spark Therapeutics investigational trail moves into Phase 3

August 7, 2018

The following is an excerpt from a press release from Spark Therapeutics. Read the press release in its entirety here. Investigational SPK-8011 moving to Phase 3 As of the July 13 data cutoff, 12 participants in the Phase 1/2 trial have received a single administration of investigational SPK-8011, including two at a dose of 5×1011 […]

Catalyst Biosciences releases update on trial of Factor IX treatment of severe hemophilia B

June 19, 2018

The following is a summary of a press release from Catalyst Biosciences by Dr. Robert Sidonio, a trusted medical advisor. Read the full press release here. On June 18, Catalyst Biosciences provided an update of the progress from its Phase ½ trial investigating the use of FIX variant product, called CB 2679d/ISU304, which is a […]

FDA GRANTS PRIORITY REVIEW TO GENENTECH’S HEMLIBRA FOR PEOPLE WITH HEMOPHILIA A WITHOUT FACTOR VIII INHIBITORS

June 5, 2018

The following is an excerpt from a press release from Genentech. Read the entire press release here. Genentech, a member of the Roche Group, announced the U.S. Food and Drug Administration has accepted the company’s supplemental Biologics License Application and granted Priority Review for HEMLIBRA  for adults and children with hemophilia A without factor VIII […]

BioMarin Presented an Update on the Phase 1/2 Study of Valoctocogene Roxaparvovec at WFH’s 2018 World Congress in Scotland

May 22, 2018

The following is an excerpt from a press release from BioMarin. Read the entire press release here. BioMarin Pharmaceutical Inc. announced an update to its previously reported results of an open-label Phase 1/2 study of valoctocogene roxaparvovec (formerly BMN 270), an investigational gene therapy treatment for severe hemophilia A. The updated results were presented during […]

Novo Nordisk Announced New Data at WFH’s 2018 World Congress in Scotland

May 22, 2018

The following is an excerpt from a press release from Novo Nordisk. Read the full press release here. Adults with haemophilia B who received a single dose Refixia® (nonacog beta pegol; N9-GP) achieved greater total factor IX exposure than those treated with rFIXFc (recombinant factor IX-Fc fusion protein). The head-to-head paradigm7 trial also observed a […]

uniQure Presents New Data Demonstrating Clinical Benefits at American Society of Gene and Cell Therapy Annual Meeting in Chicago

May 22, 2018

The following is an excerpt from a press release from uniQure. Read the full press release here. uniQure N.V., a leading gene therapy company advancing transformative therapies for patients with severe medical needs, presented data showing successful liver transduction with the AAV5 vector in both non-human primates and humans with pre-existing anti-AAV5 neutralizing antibodies (NABs). […]

Bioverativ Presents Preliminary Phase 1/2a Data at WFH’s 2018 World Congress in Scotland

May 22, 2018

The following is a press release from Bioverativ. Read the full press release here. Bioverativ Inc., a Sanofi company dedicated to transforming the lives of people with rare blood disorders, presented initial clinical data for BIVV001, a novel and investigational von Willebrand factor (VWF)-independent factor VIII therapy for people with hemophilia A. Preliminary safety and […]

Spark Therapeutics and Pfizer Announced Data at WFH’s 2018 World Congress in Scotland

May 22, 2018

The following is an excerpt from a press release from Spark Therapeutics. Read the full press release here. Spark Therapeutics, a fully integrated gene therapy company dedicated to challenging the inevitability of genetic disease, and Pfizer, announced, with a cumulative follow-up of more than 18 patient years of observation, all 15 participants in the ongoing […]

Genentech Announced Full Results of Phase III Haven 3 Study at WFH’s 2018 World Congress in Scotland

May 21, 2018

The following is an excerpt from the press release from Genentech. Read the full press release here. Genentech, a member of the Roche Group, announced the full results from the Phase III HAVEN 3 study evaluating HEMLIBRA® (emicizumab-kxwh) prophylaxis administered every week or every two weeks in people with hemophilia A without factor VIII inhibitors […]

Genentech Releases Statement on Hemlibra Patient

April 24, 2018

The following is an excerpt from a statement from Genentech. Read the entire statement  HERE. _________________________________________________________ Genentech has recently learned that a patient in the Phase III HAVEN 2 clinical trial developed a neutralizing anti-drug antibody. For this patient, the anti-drug antibody resulted in reduced efficacy of HEMLIBRA.

Bioverativ Highlights the Impact of Humanitarian Aid in Hemophilia

April 17, 2018

The following is an excerpt from a press release from Bioverativ. Read the entire press release here.  More than 15,000 people with hemophilia in 40 developing countries have already been treated following Bioverativ and Sobi’s unprecedented donation of factor therapy to the WFH Humanitarian Aid Program Bioverativ Inc., a Sanofi company, joins the global hemophilia […]

FDA Grants Breakthrough Therapy Designation for Genentech’s Hemlibra

April 17, 2018

The following is an excerpt from a press release from Genentech. Read the full press release here.  Genentech, a member of the Roche Group, announced today the U.S. Food and Drug Administration has granted Breakthrough Therapy Designation to HEMLIBRA for people with hemophilia A without factor VIII inhibitors. Breakthrough Therapy Designation is designed to accelerate the […]

Final marketplace rule will revive discrimination and raise out-of-pocket costs for Americans with pre-existing conditions

April 13, 2018

The following is from a press release from the American Heart Association. Read the press release here.  A group of 21 patient and consumer groups issued the following statement today on the 2019 Notice of Benefit and Payment Parameters final rule:  This final rule will severely weaken key benefits that underpin core consumer protections for […]

CVS Health Announces $260,000 in New Support for Bleeding Disorder Programs

March 30, 2018

The following is an excerpt from a press release from CVS. Read the entire press release here.  Company to provide funding to National Hemophilia Foundation, Hemophilia Federation of America and local organizations across U.S. CVS Health announced more than $260,000 in charitable commitments to local and national hemophilia and bleeding disorder programs across the country. The announcement […]

Genentech Offers Hemlibra Update

March 28, 2018

Note: The following is an excerpt from a press release from Genentech. Read the full press release here. ______________________________________________________________________________________ Given the recent dialogue, we are reaching out to clarify the facts surrounding five people with hemophilia A with inhibitors to factor VIII who have passed away while receiving Hemlibra® (emicizumab-kxwh). Since 2016, five adults with hemophilia A […]

Genentech Medical Communications Line Available to Answer Questions About Hemlibra 

March 27, 2018

HFA was informed by Genentech on March 26, 2018, that a total of five patient deaths have occurred while the patients were using Hemlibra (emicizumab-kxwh). Genentech has little information that they can presently share about the circumstances surrounding the most recent patient deaths; however, Genentech could confirm that the patients had received Hemlibra as part of compassionate use and expanded patient access. Genentech has a Medical Communications line at 1(800)-821-8590 for patients, concerned community members, and healthcare providers who seek further information. […]

Reposting: HFA & NHF Issue Joint Statement Regarding Shire Lawsuit Against Genentech/Roche

March 21, 2018

[Reposted from January 16, 2018] In the past few weeks, we have received many inquiries regarding the scope of the injunction Shire is seeking in its lawsuit against Genentech/Roche. Certain information has become public during this period that enables NHF and HFA to provide some limited guidance as to the scope of the injunction that […]

FDA Advises CSL Behring Their Promotional Materials are Misleading

March 6, 2018

Note: The following is a summary of a letter dated Feb. 27, 2018, from the US Food and Drug Administration (FDA) to CSL Behring. You can read the original letter here. The FDA has advised CSL Behring that the company’s promotional materials make misleading claims about the effectiveness of its Idelvion (Factor IX) product. “Such […]

Novo Nordisk Launches Rebinyn in the United States for People with Hemophilia B

February 21, 2018

Note: The following is an excerpt from a press release from Novo Nordisk. Read the full press release here.  Novo Nordisk, a global healthcare company, today announced that Rebinyn, Coagulation Factor IX (Recombinant), GlycoPEGylated, is now available in the United States for the treatment of hemophilia B. Rebinyn is an extended half-life injectable medicine used to […]

Breakthrough in Hemophilia Gene Therapy

January 31, 2018

The following is an excerpt from an article in Medical News Bulletin. Read the full article here. Researchers at Spark Therapeutics and Pfizer tested the safety and efficacy of a new hemophilia gene therapy vector for the treatment of hemophilia B. The body’s ability to stop bleeding and repair damaged blood vessels depends heavily upon […]

Sanofi to Acquire Bioverativ for $11.6 Billion

January 22, 2018

Note: The following is edited from a press release from Sanofi. Read the full press release in its entirety here. Sanofi and Bioverativ Inc., a biopharmaceutical company focused on therapies for hemophilia and other rare blood disorders, have entered into a definitive agreement under which Sanofi will acquire all of the outstanding shares of Bioverativ for $105 […]

Third Circuit Says Health Care Company’s Donations Didn’t Violate Anti-Kickback Statute

January 20, 2018

Note: The following information comes from an article published in the New Jersey Law Journal. Read the full article here. The U.S. Court of Appeals for the Third Circuit has cleared Medco Health Solutions of violating the Anti-Kickback Statute, finding in a precedential ruling that a whistleblower failed to show a connection between alleged kickbacks […]


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