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Hemophilia Bleeding Disorder Industry News
Industry News

Prescription Benefit Program Changes for Recombinant Clotting Factor Product

  It has come to our attention that Helixate FS, a recombinant clotting factor product sold and distributed by CSL Behring, is being removed from prescription benefit programs for a limited number of patients using CVS/Caremark effective January 1, 2017. Patients currently using Helixate FS who have the “Advanced Control Formulary” plan with CVS/Caremark will have the […]

Industry News

PPTA Releases Updated Statement on Zika

The following is a summary of a statement released by the Plasma Protein Therapeutics Association (PPTA). To read the PPTA statement in full, click HERE. On September 8, 2016, PPTA issued an updated news release regarding the Zika virus (ZIKV) and plasma protein therapies. In the release, PPTA notes that: ZIKV is in a family of (lipid-enveloped) viruses that is […]

Industry News

Sangamo BioSciences Receives Orphan Drug Designation From The FDA For SB-FIX

The following is excerpted from a press release from PRNewswire. Read the press release in it’s entirety here. Sangamo BioSciences, Inc. announced earlier this week that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to SB-FIX, the company’s zinc finger nuclease (ZFN)-mediated genome editing product candidate for the treatment of hemophilia B. Sangamo […]

Industry News

Baxalta Sponsors 63 Students in 2016-17

Baxalta, now part of Shire, has awarded scholarships to 63 students from 13 states, further adding to the list of recipients of its Education Advantage scholarship program. For the seventh consecutive year, Baxalta US Inc. is helping eligible students with a bleeding disorder offset the costs of higher education, advance their careers, and pursue their […]

Industry News

First FDA-Approved Recombinant von Willebrand Factor, Vonvendi, Now Available for von Willebrand Disease

The following is excerpted from a press release from Baxalta, now a part of Shire. Read the press release in it’s entirety here. Shire has announced that Vonvendi [von Willebrand factor (Recombinant)], indicated for the on-demand treatment and control of bleeding episodes in adults (age 18 and older) diagnosed with von Willebrand disease, is now […]

Industry News

Biogen Names New Spin Off, Bioverativ; Aiming for Early 2017 Launch

The following is an excerpt from FierceBiotech.com To read the article in full, click here. Biogen previously announced its plan to spin out its blood disorder products and candidates into a separate publicly traded company. The new company will be known as Bioverativ and is slated to launch in early 2017 and trade on the Nasdaq […]

Industry News

Shire Discontinues Hemophilia Gene Therapy Drug Development

The following is an excerpt from xconomy.com. To read the article in full, click here. On a conference call Tuesday, Shire CEO Flemming Ornskov said that the Dublin and Lexington, MA, company has discontinued development of BAX 335, an experimental gene therapy for hemophilia B. Prior to the merger of Baxalta and Shire, Baxalta had indicated plans […]

Industry News

Genentech Presents Latest Data on Emicizumab (ACE910)

Note: The below is an edited version of an article originally published on BusinessWire.  The original article can be read in their entirety here. The latest data from an ongoing Japanese Phase I/II study of Emicizumab were presented earlier this week at the World Federation of Hemophilia 2016 World Congress.  Emicizumab (a product being developed by […]

Industry News

BioMarin Releases Results of Phase 1/2 Study of Gene Therapy Treatment

Note: The below is an edited version of a press release issued by BioMarin Pharmaceutical Inc. The original release can be read in its entirety here. BioMarin Pharmaceutical announced today positive interim results of an open-label Phase 1/2 study of BMN 270, an investigational gene therapy treatment for severe hemophilia A. Nine patients with severe hemophilia […]

Industry News

Sangamo BioSciences Announces New Gene Therapy Clinical Development Program

Note: The below is an edited version of an article originally published on PR Newswire. The original release can be read in its entirety here. Sangamo BioSciences, Inc. announced the presentation of preclinical data that supports the clinical development of its new proprietary gene therapy for the treatment of hemophilia A. This new therapeutic comprises an adeno-associated […]

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