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.

New Data: Hemgenix Reduces Bleeds in Hemophilia B Better than Standard Replacement Therapy

March 15, 2023

The first gene therapy approved for adults with hemophilia B has been shown to work better than standard preventative replacement therapy. The therapy increased sustainability of factor IX, lowering the both spontaneous and joint bleeds.


FDA Extends Review of Gene Therapy Roctavian for Hemophilia A

March 8, 2023

The FDA extended their review of BioMarin's Roctavian (valoctocogene roxaparvovec) for adults with hemophilia A. The FDA had planned a decision on the therapy in March but pushed it out to June 30, 2023 to have more time to go over new clinical trial data submitted by BioMarin.


FDA Approves Efanesoctocog alfa (Altuviiio) to Treat Hemophilia A

February 24, 2023

The FDA has approved Altuviiio (previously referred to as efanesoctocog alfa), a high-sustained factor VIII replacement therapy for prophylaxis and on-demand treatment for adults and children with hemophilia A. The once-weekly dose provides normal to near-normal factor levels for most of the week.


Emicizumab (Hemlibra) Appears Safe, Effective in Mild or Moderate Hemophilia

February 13, 2023

During a recent trial, emicizumab (Hemlibra) appears to be an effective and safe therapy for those with mild or moderate hemophilia. The results were consistent with other studies of the treatment, regardless of severity. 


Study Shows Efanesoctocog Alfa Prophylaxis Effective in Hemophilia A

February 6, 2023

Results from the XTEND-1 study looking at efanesoctocog alfa show that the once-weekly treatment resulted in superior clinical outcomes compared to on-demand therapy and prior treatment with recombinant or plasma-derived factor VIII. 


Changes Coming to State Medicaid and CHIP Programs

February 2, 2023

Patients and families who currently have health care coverage with Medicaid or the Children's Insurance Program may soon need to take steps to find out if they are eligible to continue coverage. 


Roctavian Trial Update Shows Most Patients Bleed Free at Three Years

January 13, 2023

Three years after receiving the one-time gene therapy valoctocogene roxaparvovec, known as Roctavian, the majority of men with hemophilia A in the study were off replacement therapies and had no bleeds requiring treatment. 


FDA Clears Test to Monitor Effectiveness of Hemophilia B Treatments

January 11, 2023

The US Food and Drug Administration cleared CRYOcheck Chromogenic Factor IX assay, a test that is intended to be used by clinical labs to detect deficiencies in factor IX, the cause of hemophilia B. The test is designed to help have a better understanding of a patient's levels of factor IX more quickly. 


Pfizer Announces Updates for Investigational Gene Therapy for Hemophilia B

January 4, 2023

Pfizer shared positive top-line results from their phase 3 BENEGENE-2 clinical study that is evaluating the investigational gene therapy fidanacogene elaparvovec for the treatment of adult males with hemophilia B. The results showed a 71% reduction in ABR and was generally well tolerated.


Data Shows SerpinPC Reduces Bleeds for All Hemophilia Types

December 16, 2022

Data shared at the recent American Society of Hematology meeting showed that SerpinPC was found safe and reduced bleeding episodes in both hemophilia A and B up to 18 months. The trial is currently ongoing and the medication is given as a subcutaneous (under the skin) injection.


Phase 1/2 Study Shows Durability of Investigational Gene Therapy SPK-8011 in Patients with Hemophilia A

December 12, 2022

Spark Therapeutics announced updated results for their Phase 1/2 clinical trial of investigational SPK-8011 gene therapy to treat hemophilia A. The data showed participants experienced sustained levels of factor and showed a 92% reduction in annualized bleed rates. 


FDA Approves First Gene Therapy for Hemophilia B

November 22, 2022

The US Food and Drug Administration approved Hemgenix (etranacogene dezaparvovec) an adeno-associated virus vector-based gene therapy for the treatment of adults with Hemophilia B. The therapy is a one-time product given in a single dose IV infusion. 

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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