An international team has joined efforts to establish guidelines for effectiveness and outcome measurements regarding gene therapies in hemophilia. The CoreHEM project will be led by researchers from McMaster University in Ontario, Canada, in collaboration with the National Hemophilia Foundation (NHF) in the U.S. and the Green Park…

Preventive treatment with Hemlibra (emicizumab) is superior to replacement therapy with factor VIII infusions for reducing bleed rates in people with hemophilia A without inhibitors, a pooled analysis of trials shows. The study “Efficacy of emicizumab prophylaxis versus factor VIII prophylaxis for treatment of hemophilia A…

Last week, I was on Facebook and a post I had been dreaming about appeared on my timeline. The U.S. Food and Drug Administration has approved a new therapy, Hemlibra (emicizumab-kxwh), for patients with hemophilia A and inhibitors. It’s the first new medicine for inhibitors in almost…

MRI screening can identify subclinical joint bleeding in some patients with severe hemophilia A on preventive treatment, according to a study in the Netherlands. Subclinical bleeds occur without symptoms and usually cannot be detected by standard physical and ultrasound examinations. “These MRI findings support the hypothesis that subclinical…

A lower dose of regular, preventive therapy with factor VIII concentrates was better at reducing bleeding events and improving joint function than on-demand treatment in children with hemophilia in Thailand, a study shows. The study, “Prophylactic vs episodic treatment to prevent bleeds and preserve joint function…

Investigational gene therapy SB-525 is well-tolerated and leads to a dose-dependent increase in factor VIII (FVIII), according to interim results from eight patients with severe hemophilia A enrolled in the Phase 1/2 Alta clinical trial. Data showed that patients who received the highest dose of SB-525 — 3e13 vg/kg dose — reached…

Sigilon Therapeutics’ candidate cell therapy for hemophilia A, called SIG-001, delivers sustained production of factor VIII for over six months and corrects bleeding in a hemophilia A mouse model. These results were shared at the 2019 American Society of Gene and Cell Therapy (ASGCT) Annual Meeting, in a…

Pfizer has partnered with Spark Therapeutics to launch a Phase 3 program to evaluate the safety and effectiveness of its factor IX replacement gene therapy for treating hemophilia B. Replacement of factor IX (FIX), the clotting protein lacking in hemophilia B, will be done via the fidanacogene elaparvovec gene…

Europe’s Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion supporting the approval of Hemlibra (emicizumab) for routine prophylaxis of bleeding episodes in hemophilia A patients with factor VIII inhibitors. Hemlibra is a humanized monoclonal antibody that binds to activated factor IX and factor X to…