News

Cash-strapped governments across the 28-member European Union are struggling to control runaway healthcare expenditures — at exactly the same time as the promise of new but expensive therapies to treat rare diseases has never been greater. That’s the paradox faced by pharmaceutical companies as well as patient advocacy groups in…

Oral hygiene among children and adults with severe hemophilia is poorer than in healthy controls, a single-center study suggests. The study, “Oral hygiene and dentition status in children and adults with hemophilia: A case–control study,” was published in the journal Special Care Dentistry. Dental care is a…

Prophylaxis with Idelvion (rIX‐FP) significantly improved clinical outcomes and quality of life for children with hemophilia B and eased daily life for their caregivers, Phase 3 clinical trial data show. The findings of the study, “Health-related quality of life in paediatric haemophilia B patients treated with…

Treatment of severe hemophilia A with Eloctate and hemophilia B with Alprolix was safe and improved the patients’ annualized bleed rates (ABRs) over four years, according to the results of two extension studies announced by Bioverativ and Sobi. The two open-label extension studies — ASPIRE (NCT01454739), and B-YOND (NCT01425723) — evaluated…

A new analysis of the Phase 3 HAVEN 2 study’s results shows that Genentech’s Hemlibra (emicizumab-kxwh) prophylaxis (preventive treatment) leads to significant reductions in the number of bleeds among hemophilia A children younger than 12 with factor VIII inhibitors. The results showed that Hemlibra administered on different schedules — once a…

Health Canada recently authorized Shire Pharma Canada‘s treatment Adynovate for hemophilia A patients younger than 12 based on results of a Phase 3 clinical trial. Approved Nov. 21, the injectible factor replacement therapy is available to pediatric patients for the prevention and control of bleeding episodes, as well…

NuFactor Specialty Pharmacy is seeking applicants for next year’s Eric Dostie Memorial College Scholarship, established 25 years ago to honor the memory of  an inspirational boy with hemophilia who died at age 5. Every year, 10 people are selected to receive the $1,000 scholarship. It is open to…

Jivi (BAY94-9027), a lab-made version of the blood clotting factor VIII (FVIII), has been approved by the European Commission for the prophylactic (preventative) treatment of hemophilia A in patients older than 12, Bayer announced. The recommended prophylaxis for Jivi administration is every five days, every…

Gene therapy has the potential to stop hemophilia in its tracks. But such therapy will likely be costly, and can carry risks that have generated some apprehension among patients who could benefit most from it. That’s the message from hemophilia patient advocates who spoke at the 2018 NORD Rare Diseases…

A gene therapy from uniQure in early testing, called AMT-180, has the the potential to treat all hemophilia A patients, including those with inhibitors, according to the company. Hemophilia A is caused by missing or defective factor VIII (FVIII), a clotting protein. About 30% of patients with severe hemophilia A develop inhibitors, or…