Margarida Azevedo, MSc,  —

UniQure‘s investigational gene therapy AMT-060 for severe hemophilia B has received PRIME designation by the European Medicines Agency (EMA). The EMA designation was based on the results of a Phase 1/2 open-label, dose-escalating study of AMT-060 (NCT02396342). The study included 10 patients, each…

The lives of four inspiring patients with bleeding disorders were introduced at the “Hope Ignites: Stories to Inspire” patient program at the Hemophilia Federation of America (HFA) annual symposium recently in Providence, Rhode Island. Sponsored by Octapharma USA, the “Hope Ignites” symposium was for patients with hemophilia to offer…

South Korea has approved an Investigational New Drug (IND) application for Catalyst Biosciences’ experimental therapy CB 2679d/ISU304, a highly potent next-generation coagulation Factor IX variant for the treatment of hemophilia B. Catalyst focuses on developing medications for hematology conditions (blood disorders), which include coagulation factors that can prevent…

Novo Nordisk’s Refixia (nonacog beta pegol, N9-GP) received a positive opinion by the Committee for Medicinal Products for Human Use (CHMP) for treatment of adolescents and adults with hemophilia B. The CHMP, under the European Medicines Agency (EMA), recommends the granting of a marketing authorization in Europe for…

The European Medicines Agency (EMA) will include SPK-9001 — an investigational drug for patients living with hemophilia B —  in Priority Medicines (PRIME), a voluntary European regulatory initiative to support medicines that target an unmet medical need. PRIME aims to optimize development plans and speed up evaluation so that these priority medicines can…

My Life, Our Future (MLOF), a national program founded by leaders in the hemophilia and blood disorder community, marked Feb. 28 — Rare Disease Day — by launching the world’s largest research repository of its kind to researchers and scholars. The program is opening to U.S. scientists and will expand to worldwide scientists in…

A hemophilia patient has died in a Phase 3 trial evaluating Roche’s  experimental treatment emicizumab (ACE910), raising further concern about its safety. Preliminary indications are that the patient died of a rectal hemorrhage and not emicizumab therapy, however, the company said. The death follows reports of serious blood-clotting events in…

An analysis was highly critical of the Orphan Drug Act, used by the U.S. Food and Drug Administration since 1983 to stimulate research into rare diseases. The study found that the act is not “sufficiently” effective in meeting the needs of patients with less common diseases, like hemophilia, while pharmaceutical companies are finding it profitable,…

Researchers have developed a protein drug that may teach the body to tolerate, rather than reject, the clotting factor treatment given to hemophilia patients. The new drug, tested in dogs, may lead to novel human therapies. The study, “Oral Tolerance Induction in Hemophilia B Dogs Fed with Transplastomic Lettuce,” appeared in the…

Being unemployed or under-educated can have a negative effect on hemophilia patients’ quality of life, including their pain levels and ability to function, according to the results of a questionnaire that were presented at the ASH Annual Meeting and Exposition. In the patients surveyed, increased pain, functional impairment and reduced…