While hemophilia A and hemophilia B are both bleeding disorders characterized by deficiencies in blood clotting factors, there are differences in one type versus the other, particularly in the specific genetic mutations that cause the disease, and in some of the treatment methods.
Hemophilia A is a rare genetic disorder that affects the blood’s ability to clot properly. It is the most common form of hemophilia, responsible for 80% of all cases.
The U.S. Food and Drug Administration (FDA) has given orphan drug and rare pediatric disease designations to Sernova’s experimental and cell-based hemophilia A treatment program using the Cell Pouch System, the company’s novel medical device. Orphan drug status is awarded to therapies aiming to treat…
GS1191, a gene therapy in the pipeline of Gritgen Therapeutics, increased factor VIII (FVIII) activity in the blood of people with hemophilia AÂ taking part in a small clinical trial in China, resulting in fewer bleeding episodes. The fully enrolled investigator-initiated trial (ChiCTR2300073179) is testing the safety and…
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Baudax Bio’s investigational regulatory T-cell therapy TI-168 for the treatment of hemophilia A with inhibitors. Orphan drug status is given to therapies intended to treat rare conditions, defined as those affecting less than 200,000 people in…
Altuviiio (efanesoctocog alfa) has been approved in Japan and Taiwan as a treatment to prevent and control bleeding in people with hemophilia A. The Japanese Ministry of Health, Labor, and Welfare (MHLW) granted Altuviiio marketing authorization on Sept. 25, according to Sanofi, the company that markets…
About a month ago, my world was turned upside down when my kneecap dislocated while I was simply shifting my leg on a recliner. This unexpected incident brought back memories of my teenage years, when I underwent two surgeries to keep my kneecap in place. To add complexity to…
A person in Germany with severe hemophilia A has been treated with Roctavian (valoctocogene roxaparvovec-rvox), the first time the gene therapy has been used commercially in Europe, according to BioMarin Pharmaceutical, its developer. “Today represents an important milestone for the hemophilia community and for patients and…
Novo Nordisk’s Alhemo (concizumab injection) has been approved in Canada for hemophilia A patients, 12 years or older, who test positive for factor VIII inhibitors. The under-the-skin injectable therapy was recently approved by Health Canada for hemophilia B patients in the same age group with factor…
Roctavian (valoctocogene roxaparvovec-rvox), the first gene therapy approved for adults with severe hemophilia A, is expected to be widely available in the U.S. this month, according to its developer, BioMarin. BioMarin also is working with authorities in Germany, Italy, and France to secure market access to…
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