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Hemophilia B News

Gene Therapy Offers Hope to Some Hemophilia Patients

Posted 2 days 12 hours ago by Drugs.com

TUESDAY, Dec. 6, 2016 – Preliminary research suggests that a single dose of an experimental gene therapy may help patients with hemophilia B. As explained by the National Hemophilia Foundation, hemophilia B is a genetic disorder where people lack a protein needed for blood clotting called factor IX, or that the type of factor IX they produce is defective. The condition can lead to excess bleeding and occurs in about one in every 5,000 births, the foundation said. But researchers said they've never seen a medical treatment boost factor IX to the level seen in the new trial. According to the findings, patients who got the gene therapy were able to stop getting infusions of the clotting factor. "Our goal in this trial was to evaluate the safety of the gene therapy product and secondarily, to determine if we could achieve levels of factor IX that could decrease bleeding events in ... Read more

Related support groups: Blood Disorders, Bleeding Disorder, Hemophilia B, Hemophilia

2 New Findings Offer Hope for Those With Severe Hemophilia

Posted 26 May 2016 by Drugs.com

WEDNESDAY, May 25, 2016 – Two new studies could pave the way to major changes in how doctors treat severe cases of hemophilia – a rare genetic disorder that can cause uncontrolled bleeding. Both studies tackle a key challenge: Up to one-third of children with severe hemophilia develop antibodies against the standard therapy. But one study highlights the value of an old therapy, while the other shows promising early results with an experimental drug. Experts said both should stir discussion among doctors, patients and parents who deal with hemophilia. But they were especially hopeful about the new drug, known as emicizumab. In the United States, about 20,000 people – mostly boys and men – are living with hemophilia, according to the U.S. Centers for Disease Control and Prevention. The disorder is caused by a defect in one of the genes that controls proteins needed for normal blood ... Read more

Related support groups: Blood Disorders, Bleeding Disorder, Anemia, Blood Transfusion, Hemophilia A, Hemophilia B, Hemophilia, Coagulation Defects and Disorders, Hemophilia A with Inhibitors, Blood Cell Transplantation

Review Finds Mixed Success With Hemophilia Treatment

Posted 16 Mar 2016 by Drugs.com

WEDNESDAY, March 16, 2016 – Though the past 50 years have brought major treatment advances, men with severe hemophilia are still at high risk for bleeding and physical disability, experts say. Hemophilia is a genetic disease that prevents blood from clotting normally, leading to an increased risk of serious bleeding. More common in men than in women, it affects about one out of every 5,000 men, according to the U.S. Centers for Disease Control and Prevention. Researchers analyzed data from nearly 7,500 male hemophilia patients in the United States between 1998 and 2011. Their findings were published online March 16 in the journal Blood. "Our analysis provides a snapshot of how improvements in care have translated into outcomes across different generations of men with hemophilia," study author Dr. Paul Monahan said in a journal news release. Monahan is a former professor of hematology ... Read more

Related support groups: Blood Disorders, Bleeding Disorder, Hemophilia A, Hemophilia B, Hemophilia, Coagulation Defects and Disorders, Hemophilia A with Inhibitors

Idelvion Approved for Hemophilia B

Posted 8 Mar 2016 by Drugs.com

MONDAY, March 7, 2016 – Idelvion has been approved by the U.S. Food and Drug Administration to replace a deficient blood clotting factor among people with hemophilia B. The product – which has the generic name of "coagulation Factor IX, albumin fusion protein" – is created using recombinant DNA technology that links the blood clotting protein known as Factor IX to another blood protein, albumin. This technology allows the protein to last longer when given intravenously, the FDA said in a news release. People with the rare genetic disorder hemophilia B are prone to recurring episodes of serious bleeding, primarily into the joints. Most people with the disorder are male, although it occasionally affects females, the agency said. The new product is meant to control bleeding episodes and to reduce their frequency. It can also be given to prevent bleeding after surgery, the FDA said. ... Read more

Related support groups: Hemophilia B, Coagulation Factor Ix, Idelvion

FDA Approves Idelvion (coagulation factor IX (recombinant), albumin fusion protein) to Treat Patients with Hemophilia B

Posted 8 Mar 2016 by Drugs.com

March 4, 2016 – The U.S. Food and Drug Administration today approved Idelvion, Coagulation Factor IX (Recombinant), Albumin Fusion Protein, for use in children and adults with Hemophilia B. Idelvion is the first coagulation factor-albumin fusion protein product to be approved, and the second Factor IX fusion protein product approved in the U.S. that is modified to last longer in the blood. “The approval of Idelvion provides another important therapeutic option for children and adults with Hemophilia B to help prevent or control bleeding and reduce the frequency of bleeding episodes,” said Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research. According to the Centers for Disease Control and Prevention, Hemophilia B is a rare inherited bleeding disorder that prevents blood from clotting normally. The disorder primarily affects males and, rarely, f ... Read more

Related support groups: Hemophilia B, Coagulation Factor Ix, Idelvion

Kids' Hemophilia Drugs a Big Part of State Medicaid Spending

Posted 31 Jul 2015 by Drugs.com

FRIDAY, July 31, 2015 – Treatment costs for one childhood illness, hemophilia, may use up a big chunk of a state's Medicaid budget, a new study out of California shows. The researchers found that treatments for hemophilia – a rare, inherited disorder in which blood does not clot normally – accounted for the largest share of spending on outpatient drugs among publicly insured children in California with serious chronic illnesses. The study "underscores the potential effect of new, expensive but [effective] pharmaceuticals on public insurance programs for children with chronic illness," wrote a group led by Sonja Swenson of Stanford University. Her team published the findings July 28 in the Journal of the American Medical Association. The researchers tracked 2010-2012 data from more than 34,300 publicly insured children and young adults under the age of 21 in California with serious ... Read more

Related support groups: Hemophilia A, Hemophilia B, Hemophilia, Antihemophilic Factor, Advate, ReFacto, Humate-P, Wilstart, Monarc-M, NovoEight, Fanhdi, Koate-DVI, Monoclate-P, Obizur, Haemate P, Kogenate, Alphanate, Recombinate, Helixate FS, Kogenate FS

Emergent BioSolutions Announces FDA Approval of Ixinity [coagulation factor IX (recombinant)] for Hemophilia B

Posted 1 May 2015 by Drugs.com

GAITHERSBURG, Md., April 30, 2015 (GLOBE NEWSWIRE) – Emergent BioSolutions Inc. (NYSE:EBS) today announced that the U.S. Food and Drug Administration (FDA) has approved Ixinity [coagulation factor IX (recombinant)], an intravenous recombinant human coagulation factor IX therapeutic for the control and prevention of bleeding episodes and for perioperative management in adults and children, ≥12 years of age, with Hemophilia B. Hemophilia B is a bleeding disorder caused by a mutation on the factor IX gene resulting in a deficiency of clotting factor IX in the blood, which controls bleeding. "Emergent is committed to making a positive impact on the Hemophilia B community, including people living with this chronic bleeding disorder as well as their families, caregivers, and healthcare providers," said Barry Labinger, Executive Vice President and President, Biosciences Division at Emergent B ... Read more

Related support groups: Hemophilia B, Coagulation Factor Ix, Ixinity

Long-Acting Clotting Agent Alprolix Approved for Form of Hemophilia

Posted 31 Mar 2014 by Drugs.com

MONDAY, March 31, 2014 The drug Alprolix has been approved by the U.S. Food and Drug Administration as the first long-acting hemophilia B clotting agent, the FDA said in a news release. The product, formally known as "coagulation factor IX recombinant Fc Fusion protein" is the first hemophilia B remedy designed to require less frequent injections than prior treatments, the FDA said. It was approved to help control and prevent bleeding episodes and to manage bleeding during surgery. Hemophilia B is a blood-clotting disorder, primarily affecting males, caused by an abnormal Factor IX gene. Affecting some 3,300 people in the United States, it can lead to potentially deadly bleeding episodes. Alprolix was evaluated in clinical studies involving 123 people, ages 12-71, with severe hemophilia B. No safety issues were identified during the trials, the FDA said. The product is manufactured by ... Read more

Related support groups: Hemophilia B, Rixubis, BeneFix, Alphanine SD, BeneFIX 250 Int'l Units, Coagulation Factor Ix, Mononine

FDA Approves Alprolix (recombinant coagulation factor IX) for patients with Hemophilia B

Posted 28 Mar 2014 by Drugs.com

March 28, 2014 – The U.S. Food and Drug Administration today approved Alprolix, Coagulation Factor IX (Recombinant), Fc Fusion Protein, for use in adults and children who have Hemophilia B. Alprolix is the first Hemophilia B treatment designed to require less frequent injections when used to prevent or reduce the frequency of bleeding. Alprolix is approved to help control and prevent bleeding episodes, manage bleeding during surgical procedures, and prevent or reduce the frequency of bleeding episodes (prophylaxis). Alprolix consists of the Factor IX molecule linked to a protein fragment, Fc, which is found in antibodies. This makes the product last longer in circulation. “The approval of this product provides another therapeutic option for the treatment and prevention of bleeding in patients with Hemophilia B,” said Karen Midthun, M.D., director of the FDA’s Center for Biologics Ev ... Read more

Related support groups: Hemophilia B, Factor Ix Complex

FDA Approves Rixubis - First Recombinant Coagulation Factor IX For Use in Preventing Bleeding Episodes

Posted 27 Jun 2013 by Drugs.com

June 27, 2013 – The U.S. Food and Drug Administration yesterday approved Rixubis [Coagulation Factor IX (Recombinant)] for use in people with hemophilia B who are 16 years of age and older. Rixubis is indicated for the control and prevention of bleeding episodes, perioperative (period extending from the time of hospitalization for surgery to the time of discharge) management, and routine use to prevent or reduce the frequency of bleeding episodes (prophylaxis). An inherited blood clotting disorder mainly affecting males, Hemophilia B is caused by mutations in the Factor IX gene and leads to deficiency of Factor IX. Hemophilia B affects about 3,300 people in the United States. Individuals with Hemophilia B can experience potentially serious bleeding, mainly into the joints, which can be destroyed by such bleeding. “As the first recombinant coagulation factor IX indicated s ... Read more

Related support groups: Hemophilia B, Coagulation Defects and Disorders, Hemophilia, Coagulation Factor Ix

Gene Therapy a Boon for 6 Hemophilia Patients

Posted 12 Dec 2011 by Drugs.com

SATURDAY, Dec. 10 – A single treatment of gene therapy dramatically improved symptoms and quality of life in a small group of men with hemophilia B, an uncommon form of the bleeding disorder, a new study suggests. "It is a small trial in that only six patients have been treated thus far, but it has been rather successful and I think it will encourage further use of this approach in the very near future, both by us and others," said senior author Dr. Andrew Davidoff, chairman of the department of surgery at St. Jude Children's Research Hospital in Memphis. "At this point, we're a little shy of being able to call it a cure, but it's a good start." Six severely affected patients were infused with a correct version of the gene that makes a protein called factor IX, which is essential for blood clotting. Following treatment, four were able to stop receiving protein injections to prevent ... Read more

Related support groups: Hemophilia A, Hemophilia B

Using Drug for Prevention Might Help in Hard-to-Treat Hemophilia

Posted 2 Nov 2011 by Drugs.com

WEDNESDAY, Nov. 2 – A compound currently used to stop bleeding episodes in a subset of hard-to-treat hemophilia patients also seems to be effective in preventing bleeds before they start. Although this study, published in the Nov. 3 edition of the New England Journal of Medicine, was a small one and didn't last long, the results do raise the possibility that, over time, this type of use will also prevent the much-feared joint damage that is a hallmark of the condition. "This study shows that it is at least possible to keep these people relatively healthy, without bleeds, by using chronic prophylactic [preventive] agents," said Dr. Thomas Harrington, director of the Adult Hemophilia and Adult Sickle Cell programs at the University of Miami Miller School of Medicine. He was not involved with the study. The research was funded by Baxter BioScience, which makes FEIBA (Factor VIII Inhibitor ... Read more

Related support groups: Hemophilia A, Hemophilia B

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Novoseven, Rixubis, BeneFix, factor ix complex, Idelvion, NovoSeven RT, Alprolix, Ixinity, Bebulin VH, view more... Bebulin, BeneFIX 250 Int'l Units, Proplex T, coagulation factor viia, Mononine, Alphanine SD, Konyne 80, coagulation factor ix, Profilnine SD