In our Newsroom we share some recent headlines and links to articles that may be of interest to our bleeding and clotting disorder patients, families, and providers.
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Freeline Shares Positive Data from Gene Therapy Trial

July 18, 2022

Factor IX levels were normal for the first three participants in the B-LIEVE trail a month after being treated with FLT180a, Freeline's experimental gene therapy for hemophilia B. The early data showed rapid and consistent elevations in FIX to normal levels, which can prevent bleeding and regular factor replacement for people with hemophilia B. 

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Sanofi Shares Data from Two Phase 3 Studies

July 10, 2022

Sanofi presented data on its hopeful treatments at the International Society on Thrombosis and Haemostasis 2022 Congress held in London. The positive data on their efanesoctocog alfa, and fitusiran provided safety and bleeding rates of the two trials for the treatments, in the hopes they can provide more normalcy for patients.

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NHF Requests Risk Evaluation and Mitigation Study for Pending Gene Therapies

July 8, 2022

The National Hemophilia Foundation submitted a citizen's petition to the FDA regarding the pending approval of two gene therapies. The request is to ensure the health and well-being of people with hemophilia who may receive a gene therapy product. 

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Elocta (Eloctate in the US) Reduced Elbow, Knee Bleeds in Severe Hemophilia A Study

June 10, 2022

A study in Spain showed that one year of treatment with Elocta (known in the United States as Eloctate) reduced the frequency of bleeds in the elbows and knees of adults with severe hemophilia A. Reduced pain intensity in elbows was also reported as well as improved strength in hamstring muscles at the back of the legs. 

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Bayer Announces Discontinuation of Kogenate FS

June 2, 2022

Bayer announced that they are discontinuing the recombinant factor VIII product Kogenate FS. The therapy was first approved in 1993 by the FDA for both adults and children with hemophilia A. The company cited an increasing shift from the product to newer extended half-life products. 

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FDA Grants Breakthrough Status to efanesoctocog alfa (BIVV001) for Hemophilia A

June 1, 2022

The FDA has granted Breakthrough Therapy designation to efanesoctocog alfa (BIVV001) for the treatment of people with hemophilia A. Breakthrough drugs are placed on a fast-track approval system and given intensive guidance from the FDA. The therapy is developed for prevention of bleeding via once weekly prophylactic infusions. 

ResearchYellow

Emicizumab Safe, Effective in Hemophilia A Patients with Inhibitors

May 9, 2022

An analysis presented at the 2022 American Society of Pediatric Hematology/Oncology Conference shows that emicizumab is safe and effective in pediatric patients with hemophilia A. The research indicates improvement regardless of evaluated dosage. 

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Arthritis Medicine Helps Prevent FVIII Inhibitors in Animal Study

May 9, 2022

A study using rats found that treatment with abatacept, a medication approved for rheumatoid arthritis, prevented formation of inhibitors against hemophilia A replacement therapies. 

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FDA Clears Pfizer to Resume Hemophilia Gene Therapy Trial

May 3, 2022

The US Food and Drug Administration has cleared Pfizer to resume dosing patients with an experimental gene therapy for hemophilia that was put on hold last year due to the risk of blood clots. The company plans to begin treating patients again later this year.

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Research Shows Eptacog Beta Safe and Effective in Pediatric Patients

April 29, 2022

Findings from a phase 3 clinical study suggests that the recombinant factor VIIa bypassing agent eptacog beta was used safely and effectively to treat and control bleeding in children ages 12 or younger with hemophilia A or B with inhibitors. 

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Bone Disorders More Common for Female Hemophilia Carriers and VWD Patients

April 20, 2022

An analysis of US insurance data shows that osteoporosis and other bone disorders are more common in hemophilia carriers than in the general population. Research also showed a higher rate of bone problems among people with von Willebrand disease.

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Rebinyn Shows Positive Data in Canadian Real World Study

April 13, 2022

In a real world study in Canada, hemophilia B patients who switched to Rebinyn, an extended half-life factor IX (FIX) replacement therapy, had better responses than with their previous standard or extended half-life FIX replacement therapies.

News Content Disclaimer:

Links to articles listed may take readers to websites separate and out of the control of the CU School of Medicine and the Hemophilia and Thrombosis Center. The CU School of Medicine and the Hemophilia and Thrombosis Center are not responsible for any errors, inaccuracies or opinions that may be expressed in any of the linked articles.

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