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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 陆 trial investigating the use of FIX variant product, called CB 2679d/ISU304, which is a […]

FDA GRANTS PRIORITY REVIEW TO GENENTECH鈥橲 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鈥檚 supplemental Biologics License Application and granted Priority Review for HEMLIBRA 聽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庐 (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庐 (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 聽HERE. _________________________________________________________ Genentech has recently learned that a patient in the Phase III HAVEN 2 clinical trial developed a neutralizing anti-drug聽antibody. For聽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.聽 More than 15,000 people with hemophilia in 40 developing countries have already been treated following Bioverativ and Sobi鈥檚 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.聽 Genentech, a member of the Roche Group, announced today the U.S. Food and Drug Administration has granted Breakthrough Therapy Designation to HEMLIBRA聽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.聽 A group of 21 patient and consumer groups issued the following statement today on the 2019 Notice of Benefit and Payment Parameters final rule:聽 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聽excerpt from a press release from CVS. Read the entire press release here.聽 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聽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聽hemophilia A with inhibitors to factor VIII who have passed away while receiving Hemlibra庐聽(emicizumab-kxwh). Since 2016, five adults with hemophilia A […]

Genentech Medical Communications Line Available to Answer Questions About Hemlibra聽

March 27, 2018

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

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.聽 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聽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鈥檚 ability to stop bleeding and repair damaged blood vessels depends heavily upon […]

Sanofi to Acquire Bioverativ for $11.6 Billion

January 22, 2018

Note:聽The following is edited from a press release from Sanofi. Read the full press release in its entirety聽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 […]

Consolidated Postings Regarding Shire’s Lawsuit Against Genentech/Roche

January 16, 2018

In an effort to provide the hemophilia community a centralized location to find the most up-to-date statements and press releases regarding a recent injunction Shire is seeking in its lawsuit against Genentech/Roche, HFA will publish links on this page. As a reminder,聽HFA will NOT engage in the dialogue around patent ownership and will not be […]

Shire Provides Statement Regarding Lawsuit

January 16, 2018

Shire has published a聽statement regarding the ongoing lawsuit Shire has filed against Genentech/Roche. Read the full statement聽in its entirety聽here.    

HFA and NHF Issue Joint Statement Regarding Shire Lawsuit Against Genentech/Roche

January 12, 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.聽 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 Shire has requested from the […]

Genentech Provides Statement Regarding Patient Access to Hemlibra

January 11, 2018

Genentech has published a聽statement regarding patient access to Hemlibra (Emicizumab-kkwh). Read the full statement聽in its entirety聽here.  

PSI Files Lawsuit Against U.S. Department of Health and Human Services

January 9, 2018

On January 9th, Patient Services, Inc. (PSI) filed a lawsuit against the Federal Government alleging that a 2017 Modified Advisory Opinion from the Office of the Inspector General (OIG) violates their First Amendment rights. For more information, please see the press release from PSI.

Alnylam and Sanofi Enter into Strategic Restructuring of RNAi Therapeutics Rare Disease Alliance

January 9, 2018

Note:聽The following is edited from a press release from Alnylam. Read the full press release in its entirety聽here. Alnylam Pharmaceuticals, Inc.,聽an RNAi therapeutics company, and Sanofi announced today a strategic restructuring of their RNAi therapeutics alliance to streamline and optimize development, and commercialization of certain products for the treatment of rare genetic diseases. Specifically, Alnylam […]

Catalyst Biosciences Initiates Phase 2 of Potential Hemophilia A and B Therapy

January 5, 2018

Note:聽The following is聽an excerpt聽from an article by Rare Disease Report. Read the full聽article in its entirety聽here. On Jan. 4, Catalyst Biosciences announced the initiation and open enrollment of the Phase 2 part of its Phase 2/3 program of marzeptacog alfa (activated) (MarzAA), a highly potent, subcutaneously administered Factor VIIa therapy in development for the treatment […]

uniQure Announces Updated, Long-Term Clinical Data from Ongoing Phase I/II Trial of AMT-060 In Patients with Severe Hemophilia B

December 15, 2017

  Note: The following is edited from a press release from Spark Therapeutics. Read the full press release in its entirety here. uniQure N.V.聽a leading gene therapy company advancing transformative therapies for patients with severe medical needs, announced on Dec. 11, 2017, updated results聽from its ongoing, dose-ranging Phase I/II trial of AMT-060, its investigational gene […]

BioMarin Provides 1.5 years of Clinical Data for Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A

December 15, 2017

Note: The following is edited from a press release from Spark Therapeutics. Read the full 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 (formerly BMN 270), an investigational gene therapy treatment for severe hemophilia A. The data […]


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