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.
Bayer Joins WFH to Help Hemophilia Patients in Developing Countries
A new five-year alliance between Bayer and the World Federation of Hemophilia (WFH) Humanitarian program will allow training, education, and treatments to healthcare workers in more than sixty underserved countries. The program is likely to help support the needs of nearly 5,000 people living with hemophilia A around the world. See full article at hemophilianewstoday.com.
BioMarin Shares Data for Gene Therapy Before FDA Submission
BioMarin submitted updated data for their hemophilia A gene therapy trial of valoctocogene roxaparvovec (valrox) for approval from the FDA. Clotting factor in seven patients rose to a level that will prevent severe bleeding incidents for at least eight years but there are still concerns about the durability of the product. See full article at biopharmadive.com.
Six Ways for Young Adults to Make Adherence Easier
Most hemophilia patients have parents and caregivers making sure they keep up with their treatment, but once kids hit teen and young adult years they may struggle to stay on top of it. Hemaware provides several ways to help teens and young adults take control and learn to treat themselves, and keep their treatment plan on track. See full article at hemaware.org.
Being Fin's Mom: Life after your newborn has a stroke
As a part of Pediatric Stroke Awareness Month, one of our patients and his mom were on local Denver News Channel 9News. This short segment allowed Fin's mom to share a little of what it's like to manage care of a child who has had a stroke in the womb. See video at 9news.com.
New Data for uniQure's Phase 2 Trial for AMT-061, Hemophilia B Gene Therapy
UniQure shared updated data from their Phase 2 Trial of the investigational gene therapy for hemophilia B at the 2019 Scientific Symposium of Hemostasis & Thrombosis Research Society (HTRS) in New Orleans, LA in May. The results show that the levels of clotting factor continue to maintain increases after six months. See full article at hemophilianewstoday.com.
Signs of Stroke in Babies and Toddlers
May is Pediatric Stroke Awareness Month. While the majority of strokes happen to older people, many may be unaware that stroke can happen to children, even babies in the womb. This video from CHASA shares important information about how to recognize the signs of stroke in infants. See video on YouTube.
Talking About School Shootings
In the wake of the most recent tragedy at the STEM school in Highland's Ranch, parents may want some solid advice on how to talk to their children about school shootings. Children's Hospital Colorado has a straightforward article providing great suggestions on ways to address this scary topic with your kids. See full article at childrenscolorado.org.
Why We Can't Forget Children During Stroke Awareness Month
The month of May has long been recognized as Stroke Awareness Month. Many may know the signs for a stroke in an adult, but stroke occurs in children and infants as well. Pediatric Stroke Warriors shares important facts and information about how vital it is to know about what stroke looks like in children. See full article at pediatricstrokewarriors.org.