Scientists Use New Protein Replacement Therapy to Treat Hemophilia B in Mice
Scientists managed to treat hemophilia B using a new method of protein replacement therapy in a mouse model of the condition.
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Orphan Drug Act May Be Helping Pharmaceuticals More Than Patients, Analysis Argues
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…
Plant-Based Hemophilia Therapy Tested in Dogs Shows Promise, Says Study
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…
Age at Start of Therapy in Advanced Hemophilia Patients Determines Improvements in Joint Motion
Therapy to prevent bleeding (called prophylaxis) in patients with severe hemophilia A led to decreased joint bleeding when it was started at any age. However, improved joint motion was only effective if prophylaxis was started before age 4 in non-obese patients, according to researchers. The study, “Prophylaxis Usage, Bleeding…
Medicaid-Covered Men with Hemophilia Spend Less on Healthcare Than Employer-Insured Peers
Men with hemophilia who have a Medicaid health plan spend less on healthcare than those with employer-sponsored health insurance, due to lower reimbursement. However, Medicaid spends more on extensive healthcare users than employer-sponsored plans, according to a new study from the Centers for Disease Control and Prevention (CDC). The study,…
New Formula of Recombinant Factor VIII in Kids with Severe Hemophilia A Is Effective, Trial Shows
A new, modified version of recombinant factor VIII (rFVIII), called rVIII-SingleChain, showed great efficacy and safety in treating bleeding events in children with severe hemophilia A. The study, “Safety, efficacy and pharmacokinetics of rVIII-SingleChain in children with severe hemophilia A: results of a multicenter clinical trial,” was published in…
Lacking Employment, College Education Negatively Impacts Hemophilia Patients, Study Finds
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…
EMA Names Investigational Gene Therapy, BMN 270, a Priority Medicine for Hemophilia A
BioMarin Pharmaceuticals is moving forward with BMN 270, an experimental gene therapy for the treatment of hemophilia A, and is preparing new clinical trials expected to begin this year. The European Medicines Agency (EMA)’s Committee for Advanced Therapies (CAT) and its Committee for Medicinal Products for Human Use (CHMP) agreed that BMN 270…
Uniqure’s Hemophilia B Treatment Granted FDA Breakthrough Therapy Designation
The U.S. Food and Drug Administration (FDA) has granted uniQure’s AMT-060, an investigational gene therapy, breakthrough therapy designation for patients with severe hemophilia B. The regulatory agency based its decision on data from the ongoing, dose-ranging Phase 1/2 clinical trial (NCT02396342) evaluating AMT-060 for hemophilia B. The study’s estimated completion date…
Catalyst to Present Data on Clotting Factor Candidates at Hemophilia Congress
Catalyst Biosciences will present three posters with data from its two investigational candidates for hemophilia at the upcoming 10th Annual Congress of the European Association of Haemophilia and Allied Disorders (EAHAD) Feb. 1-3 in Paris, France. The first poster presentation is for a next-generation factor VIIa, marzeptacog alfa (activated), that showed…
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