Application deadline for NHF's National Youth Leadership Institute is Oct 31
The National Youth Leadership Institute (NYLI) is a program for young adults (18-24) in the bleeding disorder community to gain leadership experience, work with advocacy groups and non-profits, and deliver education to local NHF chapters. The application deadline is Oct 31, 2019. See full article at hemophilia.org.
FDA Approves Wilate for Hemophilia A in Adult and Adolescent Patients
The FDA has approved Octapharma's Wilate for the treatment of adults and adolescents with hemophilia A for routine prophylaxis to reduce the frequency of bleeding episodes, on demand treatment, and control bleeding episodes. See full article at businesswire.com.
Genentech Issues Statement on Hemlibra Particles; MASAC Responds
Genentech issued a statement about the quality assurance of their manufacturing when they found unexpected particles in batches of Hemlibra. These particles were discovered to consist of protein and a non-toxic polymer. Both Genentech and MASAC issued statements explaining their findings and no adverse events are linked to the particulate matter found. See full article at hemophilia.org.
Freeline's Gene Therapy for Hemophilia B Shows Promising Phase 1/2 Results
Early data from Freeline's single infusion gene therapy for patients with hemophilia B, FLT180a, showed a rise in clotting factor IX to 40% with levels remaining stable for over a year. Freeline's experimental therapy uses a AAV capsid to deliver a functional version of human clotting factor. See full article at hemophilianewstoday.com.
Study Shows Hemophilia 3 Times as Prevalent in Men than Originally Thought
Researchers at McMaster University in Canada believe that three times more men in the world have hemophilia than previously believed. The study found that close to 1,125,000 men around the world have hemophilia and that 418,000 have the severe form of the disorder. See full article at globalnews.ca.
BioMarin Explains Phase 3 Results for Hemophilia A Gene Therapy Trial
BioMarin Pharmaceutical explained results from their Phase 3 valrox gene therapy trial. The results showed timing of steroid administration may have played a role in the initial interim results. A delay in starting steroids stems from the study's design, which calls for an on-demand use rather than prophylactic regimen that was used in Phase 2. See full article at biopharmadive.com.
Hemlibra Given with Immune Tolerance Induction Safely Treats Children
A new study published in the journal Haemophilia shows that combining immune tolerance induction can help prevent development of anti-factor VIII inhibitors when used with Hemlibra treating children with severe hemophilia A. See full article at hemophilianewstoday.com.
FDA Grants Orphan Drug Designation to SIG-001 for Hemophilia A
The FDA has granted orphan drug designation to Sigilon Therapeutics' SIG-001. The investigational therapy under evaluation for hemophilia A uses Sigilon's Shielded Living Therapeutics platform to implant cells engineered to produce stable blood plasma levels of factor VIII. See full article at healio.com.
Positive Data from Multiple Studies Shared about Hemlibra at ISTH Congress
Genentech presented data for Hemlibra showing continuing long term safety, efficacy and quality of life benefits to patients taking Hemlibra. The pooled HAVEN studies showed a high proportion of patients experienced zero treated bleeds and maintained this over 83 weeks. Hemlibra is a prophylactic treatment that is administered subcutaneously. See full article at biospace.com.
New Results Presented on Catalyst's MarzAA for Hemophilia A or B
A New results on Catalyst's marzeptacog alfa (MarzAA) showed that they met primary endpoints of significantly reducing the annualized bleeding rate in those with hemophilia A and B. The medication can be dosed subcutaneously, making it significantly easier to administer the therapy. See full article at pharmaphorum.com.
BioMarin Announces Plans to Submit to FDA for Gene Therapy Marketing Auth.
BioMarin announced that they plan to submit marketing applications to both the FDA and the European Medicines Agency for its investigational gene therapy valoctocogene roxaparvovec, for adults with severe hemophilia A. This is expected to represent the first time a gene therapy product for any type of hemophilia will be reviewed for marketing authorization. See full article at prnewswire.com.
Takeda Shares New Data from PROPEL Study on Benefits of Adynovate
The latest results from the PROPEL study of Adynovate for hemophilia A patients shows that targeting prophylactic treatment may enhance a patient's PK profile. The group in the study showed reduced total annual bleeding and reduced spontaneous joint bleeds by adapting the dosing regimen. See full article at finance.yahoo.com.
Bioverativ Presents Positive Data from Completed Phase 1/2 EXTEN-A Study
A study of Bioverativ's BIVV001 showed that the treatment achieved average factor activity levels at seven days post infusion, and significantly extended the half-life of factor VIII to 43 hours. The data reinforces the potential to address the need for protection from bleeds with longer dosing intervals. The treatment is the only factor therapy designed for von Willebrand Disease. See full article at pipelinereview.com.
Sangamo and Pfizer Announce Updated Phase 1/2 Results for Gene Therapy
Sangamo and Pfizer shared data for their SB-525 investigational hemophilia A gene therapy trial at the 2019 ISTH Congress. The data showed that the patients rapidly achieved normal levels of FVIII with no reported bleeding events. The response continues to be durable for 24 weeks, which was as long as their follow up. See full article at pipelinereview.com.
UniQure Announces Positive Follow Up Data from Gene Therapy Studies
Clinical data for uniQure's ongoing Phase 2b study of AMT-061 was shared at an oral presentation at ISTH in Melbourne, Australia. The three patients enrolled in the study all showed sustained increases in their FIX levels 36 weeks after one administration of AMT-061. The study will be followed for 52 weeks to assess FIX activity, bleeding rates and need for FIX replacement therapy. See full article at globenewswire.com.
Study Finds Jivi May Protect Hemophilia A Patients Longer than Eloctate
A Study of Bayer's Jivi, a prophylactic treatment for hemophilia A, shows it has a longer half-life and slower clearance from blood circulation than Eloctate. The study, published in the journal Annals of Hematology, showed that Jivi may protect against bleeding for longer periods. See full article at hemophilianewstoday.com.