Octapharma USA Announces FDA Approval of NUWIQ New Product Strengths
Note: The following is an edited version of a press release from Octapharma USA. Read the press release from Octapharma USA in it’s entirety here. Octapharma USA today announced the U.S. Food and Drug Administration (FDA) has approved new product strengths for NUWIQ. The agency approved new single dose NUWIQ vial strengths of 2500, 3000 and 4000 International […]
Spark Therapeutics Releases Preliminary Data for SPK-8011
Note: The following is an edited version of a press release from Spark Therapeutics. Read the press release from Spark Therapeutics in it’s entirety here. The company has disclosed preliminary initial data from the Phase 1/2 dose-escalation clinical trial for SPK-8011, a novel bio-engineered adeno-associated viral (AAV) vector utilizing the Spark200 capsid and containing a codon-optimized human factor […]
Spark Therapeutics Presents Updated Interim Hemophilia B Data Supporting Consistent & Sustained Response at ISTH 2017
Note: The following is an edited version of a press release from Spark Therapeutics. Read the press release from Spark Therapeutics in it’s entirety here. Spark Therapeutics recently announced that 10 participants in its ongoing Phase 1/2 clinical trial of SPK-9001 for hemophilia B, as of the June 5, 2017 data cut off, had their AIR reduced approximately […]
BioMarin's Investigational Gene Therapy for Hemophilia A Maintains Average Factor VIII Levels within Normal Range for over One Year
Note: The following is an edited version of a press release from BioMarin. Read the press release from BioMarin in it’s entirety here. BioMarin Pharmaceutical Inc. announced an update to its previously reported interim results of an open-label Phase 1/2 study of BMN 270, an investigational gene therapy treatment for severe hemophilia A. The updated results were presented […]
uniQure Announces Updated, Long-Term Clinical Data from Ongoing Phase I/II Trial of AMT-060 In Patients with Severe Hemophilia B
Note: The following is an edited version of a press release from uniQure. Read the press release from uniQure in it’s entirety here. uniQure N.V. has announced updated results from its ongoing, dose-ranging Phase I/II trial of AMT-060, its proprietary, investigational gene therapy in patients with severe hemophilia B. The data includes up to 18 months of follow-up from the low-dose […]
Bioverativ Files Complaints Against CSL Behring's Idelvion
Note: This story has been sourced from the website, www.thepharmaletter.com and The Australian Business Review. On Friday, July 7, 2017, Bioverativ filed complaints in the U.S. District Court for the District of Delaware and with the U.S. International Trade Commission alleging that Idelvion, a novel factor IX albumin fusion protein developed by CSL Behring, infringes upon […]
Alnylam & Sanofi Genzyme Report Positive Results from Ongoing Phase 2 Study with Investigational RNAi Therapeutic Fitusiran in Patients with Hemophilia A and B With or Without Inhibitors
Note: The following is an edited version of a press release from Alnylam. Read the press release from Alnylam in it’s entirety here. Alnylam Pharmaceuticals, Inc. and Sanofi Genzyme, announced today new positive results from the ongoing Phase 2 open-label extension study with fitusiran in patients with hemophilia A and B, with or without inhibitors. These results were presented today […]
Shire Gets Injunction Against Roche Over Emicizumab
Note: The following is an excerpt from a Reuters news article. Read the article in full here. Pharmaceutical group Shire said on Sunday it had obtained a preliminary injunction in a Hamburg court against rival Roche over its hemophilia drug emicizumab, alleging incomplete and misleading statements surrounding the treatment.Roche said in an emailed statement it would […]
Genentech Announces Positive Phase III Results for Emicizumab at ISTH 2017
Note: The following is an edited version of a press release from Genentech. Read the press release from Genentech in it’s entirety here. Genentech, a member of the Roche Group, announced today that data from HAVEN 1, a Phase III study evaluating once-weekly subcutaneous emicizumab prophylaxis (preventative) in adults and adolescents with hemophilia A with inhibitors, were published […]
CDC Health Advisory: Patients Receiving Eculizumab (Soliris®) at High Risk for Invasive Meningococcal Disease Despite Vaccination
Per an early release of the Centers for Disease Control and Prevention (CDC) Morbidity and Mortality Weekly Report (MMWR), new information was released today regarding Eculizumab (Soliris®) in patients with the rare blood disorders, paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome. Read the MMWR article in full here.