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FDA Approves New Novo Nordisk Treatment for Patients with Hemophilia B

June 1, 2017

  Novo Nordisk recently announced that the U.S. Food and Drug Administration (FDA) has approved the Biologics License Application for REBINYN庐 (Coagulation Factor IX (Recombinant), GlycoPEGylated) for the treatment of adults and children with hemophilia B.聽Novo Nordisk expects to launch REBINYN庐 in the U.S. in the first half of 2018. For more info on REBINYN, […]

Dimension Therapeutics Discontinues DTX101, a Investigational Gene Therapy Product

May 12, 2017

Dimension Therapeutics聽announced it will聽discontinue the development of DTX101, an investigational AAVrh10-based gene therapy product in development for the treatment of moderate/severe-to-severe hemophilia B. The decision followed the review of the emerging DTX101 Phase 1/2 clinical study data, including the data as of the beginning of聽May 2017, and the observation that the data would not meet […]

Sangamo Therapeutics And Pfizer Announce Collaboration For Hemophilia A Gene Therapy

May 12, 2017

Sangamo Therapeutics, Inc. and Pfizer Inc. recently聽announced an exclusive, global collaboration and license agreement for the development and commercialization of gene therapy programs for Hemophilia A, including SB-525, one of Sangamo’s four lead product candidates, which Sangamo expects will enter the clinic this quarter. Click here to read the full press release from Sangamo Therapeutics.

uniQure Announces Successful Gene Therapy Delivery in Animal Model

May 12, 2017

uniQure recently presented data at the American Society of Gene and Cell Therapy (ASGCT) 20th聽Annual Meeting in Washington, D.C.聽on successful repeated hepatic gene delivery with their聽AAV5 vector following a proprietary immunoadsorption procedure in non-human primates (NHPs). The results described successful sequential transduction with the two reporter transgenes and highlighted the progress made in readministration protocols […]

Kedrion Announces Return to Normal Supply Levels of Ko膩te

May 12, 2017

Kedrion Biopharma announced a return to normal supply levels of Ko膩te in the 1000 vial size and a series of large-scale improvements to their聽overall聽operations following a temporary period of limited availability of product. Click here to read the full contents of聽letter provided by Kedrion Biopharma regarding this matter.  

uniQure Receives EU PRIME Designation For Hemophilia B Product

May 2, 2017

聽uniQure recently received the designation of a “priority medicine” from the European Union for a potential gene therapy treatment for Hemophilia B. From the press release: uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that AMT-060, its proprietary, investigational gene therapy聽in patients with severe […]

Pharmaceutical Companies Recognize World Hemophilia Day

April 17, 2017

Today, on World Hemophilia Day, many pharmaceutical companies joined in the celebration by recognizing and supporting the hemophilia community. Linked below are statements from a wide group of companies on today’s celebration of World Hemophilia Day: Grifols Press Release Bioverativ Press Release Novo Nordisk on supporting women with bleeding disorders Shire Press Release CSL Behring […]

Novo Nordisk Recognizes World Hemophilia Day

April 17, 2017

Drug manufacturer, Novo Nordisk recognized World Hemophilia Day 2017 by celebrating the theme of the year; Hear Their Voices, by showing support for women with bleeding disorders. To learn more, read Novo Nordisk’s press release in it’s entirety.聽here.

Shire Recognizes World Hemophilia Day

April 17, 2017

Clotting factor manufacturer, Shire, is celebrating World Hemophilia Day in part by hosting an awareness rally in Chicago, Illinois. The rally is aimed mainly to educate the public on the dramatic estimate of a hemophilia bleeding episode occurring worldwide every 3-15 seconds. See more on their website. Read a full statement from聽Shire recognizing World Hemophilia […]

Grifols Donates 140 Million Units of Factor on World Hemophilia Day

April 17, 2017

As part of聽World Hemophilia Day celebrations, Grifols has announced a large donation of clotting factor to the World Federation of Hemophilia (WFH) Humanitarian Aid Program.聽This announcement is a continuation of the company鈥檚 three-year commitment from 2014, bringing the total humanitarian aid commitment to more than 200M IU of Factor VIII over eight years Read the […]

Catalyst Biosciences Completes New Hemophilia B Drug Toxicology Study

April 14, 2017

On April 11, Catalyst Biosciences, a clinical-stage biopharmaceutical company focused on developing novel medicines to address hematology indications, reached a key milestone towards starting human trials with聽completion of the CB 2679d/ISU304 toxicology studies. Their press release can be read in full聽here.

Aptevo’s Ixinity Found To Be Safe In Children Under 12 With Hemophilia B

April 14, 2017

Aptevo’s Ixinity, previously approved for use in people over the age of 12, just received approval for use in children under 12, per their ongoing phase 3 trial. Read the full press release here.

Spark Therapeutics Presents Updated Preliminary Data from Hemophilia B Phase 1/2 Trial

April 14, 2017

Spark Therapeutics issued a press release on updated preliminary data from 10 infused participants in the ongoing Phase 1/2 clinical trial of investigational SPK-9001 for hemophilia B. The data is聽being presented at Hemostastis &聽Thrombosis Research Society (HTRS) this week. The press release can be read in full here.

Spark Therapeutics Announces Phase 1/2 Gene Therapy Trial for Hemophilia A

February 23, 2017

Note: The following is an edited version of a press release from Spark Therapeutics.聽Read the press release from Spark Therapeutics聽in it鈥檚 entirety聽here. Spark Therapeutics has announced that its Phase 1/2 trial for the investigation of gene therapy for hemophilia A (factor聽VIII deficiency) has begun. This trial will initially be enrolling participants at the Children鈥檚 Hospital […]

Bioverativ Officially Spins-Off of Biogen鈥檚 Hemophilia Business

February 1, 2017

Note: The following is an edited version of a press release from Bioverativ.聽Read the press release from聽Bioverativ聽in it’s entirety聽here. Today, February 1, 2017, Bioverativ聽launched as an independent, global biotechnology company focused on the discovery, development, and commercialization of innovative therapies for hemophilia and other rare blood disorders. Bioverativ is formed by a spin-off of Biogen鈥檚 […]

UniQure Receives FDA Breakthrough Designation of AMT-060 Investigational Gene Therapy

January 30, 2017

______________________________________________________________________________ Note: The following is edited from a press release from UniQure. Read the full press release in it鈥檚 entirety聽here. UniQure announced that AMT-060, 聽its proprietary, investigational gene therapy聽in patients with severe hemophilia B, has received Breakthrough Therapy designation by the U.S. Food and Drug Administration (FDA).聽 This designation is based on results from the […]

Shire’s Adynovate Receives Two New FDA Approvals for Treatment of Hemophilia A

December 29, 2016

Note: The following is edited from a press release from Shire. Read the full press release in it鈥檚 entirety聽here. Shire plc recently announced that the U.S. Food and Drug Administration (FDA) has approved Adynovate, an extended circulating half-life recombinant Factor VIII treatment for hemophilia A, in pediatric patients under 12 years of age. The FDA […]

Genentech Meets Primary Endpoint for Phase III Emicizumab Study

December 22, 2016

______________________________________________________________________________ Note: The following is edited from a press release from Genentech. Read the full press release in it鈥檚 entirety聽here. Genentech, a member of the Roche Group, announced that the primary endpoint has been met for the Phase III HAVEN 1 study evaluating emicizumab prophylaxis in people 12 years of age or older with hemophilia […]

UniQure Announces New Data in Phase I/II Clinical Trial

December 12, 2016

______________________________________________________________________________ Note: The following is edited from a press release from UniQure. Read the full press release in it鈥檚 entirety here. UniQure announced new and updated results during the American Society of Hematology (ASH) meeting聽in relation to its ongoing, dose-ranging Phase I/II trial of AMT-060. AMT-060 is UniQure鈥檚 proprietary, investigational gene therapy聽for patients with severe […]

Sangamo Biosciences Announces Plans to File for New Investigational Drug

December 12, 2016

______________________________________________________________________________ Note: The following is edited from a press release from Sangamo Biosciences. Read the full press release in its entirety here. Sangamo Biosciences presented data during the recent American Society of Hematology (ASH) Annual Meeting regarding preclinical and manufacturing data of SB-525, its gene therapy program for hemophilia A. “We have developed an improved […]

Spark Therapeutics Shares Updated Phase I/II Clinical Trial Data

December 12, 2016

______________________________________________________________________________ Note: The following is edited from a press release from Spark Therapeutics. Read the full press release in it鈥檚 entirety here.. Spark Therapeutics and Pfizer announced updated results from the Phase 1/2 study of SPK-9001, an investigational recombinant gene transfer product in hemophilia B, at the 58th American Society of Hematology (ASH) Annual Meeting, […]

Alnylam Presents Phase I Clinical Drug Trial Data at ASH 2016

December 12, 2016

聽 Note: The following is an excerpt from a press release from Alnylam Pharmaceuticals, Inc. regarding news from their recent presentation at the American Society of Hematology (ASH) Annual Meeting. The original article can be read in its entirety here. ______________________________________________________________________________ Alnylam Pharmaceuticals, Inc. presented data from the phase 1 study of fitusiran at the […]

Octapharma Announces Two Hemophilia A Clinical Trials Utilizing Wilate

November 16, 2016

The following is excerpted from a press release from Octapharma USA. Read the press release in it’s entirety here.聽 Octapharma USA has announced that WILATE庐, von Willebrand Factor/Coagulation Factor VIII Complex (Human) Lyophilized Powder for Solution for Intravenous Injection will be utilized for two important clinical trials for hemophilia A launching in December. Octapharma USA […]

Prescription Benefit Program Changes for Recombinant Clotting Factor Product

October 18, 2016

  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聽patients using CVS/Caremark effective January 1, 2017. Patients currently using Helixate FS who have the “Advanced Control Formulary” plan with聽CVS/Caremark will have the […]

PPTA Releases Updated Statement on Zika

September 13, 2016

The following is a summary of a statement released by the Plasma Protein Therapeutics Association (PPTA).聽To read the PPTA statement in full, click聽HERE. On September 8, 2016, PPTA聽issued an updated聽news release聽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 […]

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

September 9, 2016

The following is excerpted from a press release from PRNewswire. Read the press release in it’s entirety聽here. Sangamo BioSciences, Inc.聽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 […]

Baxalta Sponsors 63 Students in 2016-17

August 22, 2016

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 […]

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

August 9, 2016

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 […]

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

August 9, 2016

The following is an excerpt from FierceBiotech.com To read the article in full, click聽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 […]

Shire Discontinues Hemophilia Gene Therapy Drug Development

August 4, 2016

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


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