BAY 94-9027, developed by Bayer, is an investigational treatment for severe hemophilia A. It is a PEGylated molecule of recombinant factor VIII in which the B-domain, a region within the factor, is deleted. The therapy is produced using a baby hamster kidney cell line (BHK-21), and has…
BAX 855 (Adynovate) is an approved factor replacement therapy, developed by Baxter International to prevent and treat bleeding in people with hemophilia A. How BAX 855 is manufactured BAX 855 (or rurioctocog alfa pegol) is a PEGylated molecule of full-length recombinant factor VIII (FVIII) produced in Chinese hamster ovary…
The recommended treatment regimen for patients with severe hemophilia to prevent bleeding episodes is to replace the deficient clotting factor. However, currently available replacement clotting factors are limited by their relatively short half-lives and require intravenous injections up to three times a week to maintain protective levels. This can have…
Prophylaxis is commonly referred to as a preventive measure in hemophilia of regularly infusing blood clotting factor concentrates to avoid bleeding. This approach stemmed from research that patients with mild-to-moderate hemophilia (or those who have clotting factor levels of 1 percent or more) rarely experience spontaneous bleeds and have less joint damage…
Signs are appearing outside restrooms in the United Kingdom to advise members of the public that not all disabilities are visible. MORE: Learn more about hemophilia and what it really means to have the blood disorder. According to a report from the BBC, the signs are being posted to help those…
The National Center on Birth Defects and Development Disabilities has an online booklet that helps hemophiliacs and their families construct a plan in the event of an emergency. MORE: Initial results from phase 3 hemophilia trial of Emicizumab in children looks promising. The plan focuses on all aspects…
Shire has launched a Phase 3 clinical trial (NCT02585960) with Adynovate, a drug approved to help treat and control bleeding in children and adults with hemophilia A. The trial is currently recruiting participants. Adynovate is an injectable recombinant factor VIII (FVIII), designed using pegylation technology, which allows it to last…
A Phase 3 extension trial has confirmed Alprolix’s long-term ability to prevent bleeding episodes in hemophilia B patients, and its safety. Those in the B-YOND extension trial did not develop an immune response against Alprolix. Adults and adolescents were treated for three years, and children under 12 for 18 months. “These results…
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 four…
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, with…
Get regular updates to your inbox.
