Hemophilia News Today

Hemophilia News Today is an online resource that offers daily news on research, scientific developments, and helpful tips for individuals with Hemophilia and their caregivers.

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Women, girls in focus on World Hemophilia Day

1 week ago | hemophilianewstoday.com | Margarida Maia |Jennifer Lynne |Marisa Wexler |Mary Chapman

World Hemophilia Day is April 17, and this year it will focus on the challenges of women and girls living with hemophilia and other bleeding disorders. The World Federation for Hemophilia (WFH) said the theme of its campaign is “Access for all: women and girls bleed too.”“Today, women and girls are still underdiagnosed and underserved,” the foundation says on its World Hemophilia Day website.
Gene therapy BBM-H901 approved for hemophilia B in China

2 weeks ago | hemophilianewstoday.com | Marisa Wexler

The gene therapy BBM-H901 (dalnacogene ponparvovec) has been approved in China for adults with moderate to severe hemophilia B, making it the first approved gene therapy for the disorder in the country. BBM-H901 was developed and will be manufactured by Belief Biomed (BBM), but it will be commercialized in mainland China, Hong Kong, and Macau by Takeda China.
Altuviiio approved to treat hemophilia A in Canada

2 weeks ago | hemophilianewstoday.com | Marisa Wexler

Health Canada has approved Altuviiio (efanesoctocog alfa) to treat children, adolescents, and adults with hemophilia A, with the aim of preventing and controlling bleeds, including those occurring around the time of surgery. “This approval represents important progress in the treatment of patients with hemophilia A in Canada,” Stephanie Veyrun-Manetti, general manager of specialty care and country lead at Sanofi Canada, said in a company press release.
Hemophilia B gene therapy Hemgenix now reimbursed in Germany

3 weeks ago | hemophilianewstoday.com | Lindsey Shapiro

The cost of the one-time hemophilia B gene therapy Hemgenix (etranacogene dezaparvovec) will now be covered for eligible patients in Germany through the country’s public healthcare system. This comes through a reimbursement price agreement between developer CSL Behring and GKV-Spitzenverband, the German National Association of Statutory Health Insurance Funds.
Hemophilia patients' bone health screening rates low at US HTCs

3 weeks ago | hemophilianewstoday.com | Andrea Lobo |Patricia Inacio |Alliah Czarielle |G Shellye Horowitz

Despite high rates of bone health issues in hemophilia and von Willebrand disease (VWD), the routine use of DEXA scans remains low across hemophilia treatment centers (HTCs) in the U.S., a study reports. DEXA, or dual-energy X-ray absorptiometry, is a type of imaging test that uses low-dose X-rays to measure bone density and check for osteoporosis, which is where bones become weaker and more likely to break.
Fitusiran, now Qfitlia, approved by FDA for hemophilia A, B patients

3 weeks ago | hemophilianewstoday.com | Lindsey Shapiro |Marisa Wexler |Patricia Inacio |Margarida Maia

The U.S. Food and Drug Administration (FDA) has approved fitusiran as a routine prophylactic therapy to prevent or reduce the frequency of bleeding episodes in people with hemophilia A or B, ages 12 and older, with or without inhibitors, or antibodies that can make treatment less effective. The now-approved therapy from Sanofi, given by subcutaneous, or under-the-skin, injections, will be sold under the brand name Qfitlia.
On Alhemo, patients without inhibitors report better life quality

3 weeks ago | hemophilianewstoday.com | Andrea Lobo |Margarida Maia |Cazandra Campos- MacDonald |Lindsey Shapiro

Prophylactic, or preventive, treatment with Alhemo (concizumab) is associated with better quality of life and a lower treatment burden for people with hemophilia A or B without inhibitors, according to patient-reported data from a Phase 3 trial. The ongoing study, called explorer8 (NCT04082429), is investigating the efficacy and safety of Alhemo, when given via once-daily subcutaneous (under-the-skin) injections, in people with hemophilia A or B without inhibitors.
The bleeding disorders community can relate to March Madness

4 weeks ago | hemophilianewstoday.com | Jennifer Lynne |Joe MacDonald |Cazandra Campos- MacDonald

It’s that time of year again when brackets are busted, Cinderella stories happen, and basketball fans everywhere are glued to the television for the madness of March. The NCAA basketball tournament is full of buzzer-beaters, heartbreak, and jaw-dropping comebacks. And oddly enough, for many of us living with bleeding disorders, it all feels strangely familiar. This year, I filled out my bracket with optimism, placing my hopes in the Wisconsin Badgers.
Acquired hemophilia tied to bleeding, blood clot-related events in US

1 month ago | hemophilianewstoday.com | Andrea Lobo |Esteban Cerezo |Margarida Maia |Jennifer Lynne

About 30% of people hospitalized due to acquired hemophilia have a bleeding event, while 17% report a thrombotic, or blood clot-related complication, such as stroke and deep vein thrombosis, a nationwide U.S. study finds. Patients ages 65 years and older, and those with thrombotic events were more likely to die during a hospitalization. Also, about a third were readmitted within 30 days of being discharged, mainly due to bleeding events and infections.
Takeda to discontinue Hemofil M, Recombinate for hemophilia A

1 month ago | hemophilianewstoday.com | Margarida Maia |Steve Bryson |Patricia Inacio

Takeda Pharmaceuticals has decided to discontinue Hemofil M (human antihemophilic factor) and Recombinate (recombinant antihemophilic factor), both of which are approved as replacement therapies to prevent and control bleeding episodes in children and adults with hemophilia A.