Weekly use of the experimental therapy efanesoctocog alfa maintains therapeutic levels of factor VIII (FVIII) — the missing clotting factor in hemophilia A — and significantly improves physical and joint health, while reducing pain, in adolescents and adults with a severe form of the disease. These are the…
The first three participants in the B-LIEVE trial all have factor IX (FIX) levels within the normal range more than one month after being treated with FLT180a, Freeline’s experimental gene therapy for hemophilia B, according to the company. “The initial data show that FLT180a provides rapid and consistent…
Once-daily preventive treatment with Novo Nordisk’s experimental therapy concizumab significantly reduced bleeding rates in people with both hemophilia A and B with inhibitors, according to new data from the Phase 3 explorer7 trial. In fact, bleeding rates in patients receiving concizumab in trial decreased by more than 85%. Based…
Mim8, an investigational therapy to prevent bleeding episodes in people with hemophilia A, is safe and well-tolerated at multiple doses, according to data from the Phase 1/2 FRONTIER1 trial. The therapy also showed signs of efficacy, with most patients given higher doses experiencing no bleeds during treatment. “We are…
Nonprofits, scientists, governmental organizations, and the rare disease drug development industry have long cited 7,000 as the average number of rare diseases in the world. But a new analysis shows there are as many as 10,867 rare diseases globally. And that…
Most people with moderate or mild hemophilia A who received preventive treatment with Hemlibra (emicizumab) in the HAVEN 6 clinical trial had no bleeds requiring treatment over a median follow-up of over a year, new data show. The findings were presented at the 30th International Society on Thrombosis and…
Bleeding episodes in people with severe hemophilia A receiving prophylactic (preventive) treatment have the potential to impair patients’ health-related quality of life, according to data from a survey conducted in Europe. Even a single bleed was linked to marked medication-related and non-related health costs. In fact, in terms of…
Patients with hemophilia A who switched to Kovaltry (octocog alfa) — a recombinant, or lab-made, factor VIII (rFVIII) replacement therapy given at home — had significantly fewer bleeds than with their previous standard half-life replacement therapies, a real-world study in Italy reported. The therapy also reduced the frequency…
People in France with severe hemophilia A who switched to Elocta (efmoroctocog alfa) — an extended half-life replacement therapy — had fewer infusions and a reduced rate of joint bleeds, a real-world study reports. Notably, those who switched to Elocta had a higher bleeding rate, including joint bleeds,…
Nearly one-third of people with hemophilia B have antibodies against a subtype of an adeno-associated virus (AAV) — called AAV6 — that is used as an experimental gene therapy carrier, a U.K. study found. Because such antibodies could render the treatment that the carrier holds ineffective, these patients would…
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