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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 […]

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, 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 statement regarding the ongoing lawsuit Shire has filed against Genentech/Roche. Read the full statement in its entirety 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.  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 statement regarding patient access to Hemlibra (Emicizumab-kkwh). Read the full statement in its entirety 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: The following is edited from a press release from Alnylam. Read the full press release in its entirety here. Alnylam Pharmaceuticals, Inc., 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: The following is an excerpt from an article by Rare Disease Report. Read the full article in its entirety 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. a leading gene therapy company advancing transformative therapies for patients with severe medical needs, announced on Dec. 11, 2017, updated results 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 […]

Spark Therapeutics and Pfizer Announce Longer-term Preliminary Data Showing Consistent and Sustained Factor IX Levels in 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. Spark Therapeutics and Pfizer Inc. announced on Dec. 11, 2017, with a cumulative follow-up of more than 13 patient years of observation, all 11 participants in the ongoing Phase 1/2 clinical trial of investigational SPK-9001 for […]

Spark Therapeutics Presents Preliminary Data on Investigational SPK-8011 Phase 1/2 Dose-escalation Clinical Trial in 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. Spark Therapeutics announced on Dec. 11, 2017, it has dosed seven participants in the Phase 1/2 clinical trial of SPK-8011 in hemophilia A. The first four participants, who have been followed at least 12 weeks post […]

Bioverativ Announces Results of Eloctate Study Showing High Bleed Protection and Target Joint Resolution

December 15, 2017

Note: The following is edited from a press release from Bioverativ. Read the full press release in its entirety here. Bioverativ Inc. and Swedish Orphan Biovitrum AB announced the results of a new, post-hoc, longitudinal analysis of the pivotal Phase 3 A-LONG study and ASPIRE long-term extension study, showing that weekly prophylactic dosing with its […]

Bioverativ Announces Findings From Novel Imaging Study Using Alprolix

December 15, 2017

Note: The following is edited from a press release from Bioverativ. Read the full press release in its entirety here. Bioverativ Inc., a global biopharmaceutical company dedicated to transforming the lives of people with rare blood disorders, today announced findings from a novel imaging study investigating extravascular distribution of factor IX therapies, including its leading extended […]

FDA Lifts Clinical Hold on Fitusiran

December 15, 2017

Note: The following is edited from a press release from Alnylam Pharmaceuticals. Read the full press release in its entirety here. Alnylam Pharmaceuticals Inc., a RNAi therapeutics company, and Sanofi Genzyme, the specialty care global business unit of Sanofi, announced Dec. 15, 2017, the U.S. Food and Drug Administration (FDA) has lifted the hold on clinical studies with fitusiran, including […]

Genentech Presents New Data on Hemlibra at ASH 2017

December 15, 2017

Note: The following is edited from a press release from Genentech. Read the full press release in its entirety here. Genentech, a member of the Roche Group, announced on Dec. 9, 2017, new data from the ongoing HEMLIBRA® (emicizumab-kxwh) clinical development program were presented at the 59th American Society of Hematology (ASH) Annual Meeting. These data […]

Shire Files Preliminary Injunction Against Genentech/Roche

December 14, 2017

Note: The following is edited from a press release from Shire. Read the full press release in its entirety here. On December 14, 2017, Shire filed a motion for preliminary injunction against Roche subsidiaries Genentech Inc. and Chugai Pharmaceutical Co., Ltd., as part of an ongoing U.S. patent infringement lawsuit surrounding Genentech/Roche’s emicizumab, currently being marketed as […]

Genentech Announces Interim Result from Phase III HAVEN 4 Study

December 8, 2017

Note: The following is an edited excerpt from a press release from Genentech. To read the press release in it’s entirety, click HERE. Genentech, a member of the Roche Group, announced today positive interim results from the Phase III HAVEN 4 study evaluating HEMLIBRA® (emicizumab-kxwh) prophylaxis dosed once every four weeks in adults and adolescents […]

Genentech Releases Phase III HAVEN 3 Results

November 20, 2017

The following is an edited excerpt from a press release from Genentech. To read the press release in full, click here. Genentech, a member of the Roche Group, announced positive results from the Phase III HAVEN 3 study evaluating Hemlibra (emicizumab-kxwh) in adults and adolescents (aged 12 years or older) with hemophilia A without inhibitors to […]

FDA Approves Genentech’s HEMLIBRA, (Emicizumab-KXWH) for Hemophilia A with Inhibitors

November 16, 2017

Note: The following is an edited excerpt from a press release from Genentech. To read the press release in it’s entirety, click HERE. Genentech, a member of the Roche Group, announced today that the U.S. Food and Drug Administration (FDA) has approved HEMLIBRA® (emicizumab-kxwh) for routine prophylaxis to prevent or reduce the frequency of bleeding episodes […]

Alnylam Announces Successful Outcome Following FDA Meeting to Discuss Fitusiran Program

November 9, 2017

The following is an edited version of a press release from Alnylam Pharmaceuticals. To read the press in full, click here. Alnylam completed a successful meeting with the U.S. Food and Drug Administration (FDA), reaching alignment on safety measures and a risk mitigation strategy for clinical studies with fitusiran.  These measures include protocol-specified guidelines along […]

uniQure Announces Hemophilia B Gene Therapy Program To Enter Pivotal Study

October 19, 2017

Note: The followed is excerpted from a press release from uniQure. Read uniQure’s full press release HERE.  uniQure announced today plans to advance their development program for hemophilia B gene therapy into a pivotal study using an enhanced version of their product candidate called AMT-061. AMT-061 is identical to AMT-060 in every way apart from a change from […]

Bayer Issues Voluntary Recall of Kogenate FS

October 18, 2017

Note: The following is the full text of an email communication received by HFA from Bayer HealthCare Pharmaceuticals: Bayer in consultation with the FDA has voluntarily recalled approximately 300 vials (part of one lot) of Kogenate® FS, Antihemophilic Factor (Recombinant) in the U.S. as an excipient, used to stabilize the product during manufacturing, may not conform […]


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