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.

CSL Behring Will Discontinue Mononine for Hemophilia B

September 21, 2020

CSL Behring has announced they will discontinue the manufacturing of Mononine to treat hemophilia B patients. The factor medication has been on the market since 1992 but advances in other medications has led to a significant drop in demand from patients. Mononine will be available through mid-2021 giving patients who do use the therapy to adjust to new options on the market.


Genentech Provides Update on Particles in Hemlibra

September 17, 2020

Roche and Genentech have provided an update to the measures they are applying to reduce particles that were identified in Hemlibra doses in 2019. To date the particles have shown no risk or impact to patients and the manufacturer is working on ways to reduce or eliminate them. Genentech has proposed use of a transfer needle with a filter to provide additional confidence for Hemlibra's use. 


Sanofi's BIVV001 Phase 1/2 Trial Results Show Promise

September 17, 2020

Sanofi announced results from their phase 1/2a EXTEN-A trial for their investigational therapy BIVV001 to treat patients with hemophilia B. The trial showed patients achieving a FVIII half-life of 43 hours, a more than three fold increase from conventional therapy. The results appear to show that use of this treatment could reduce severe cases to behave more like a mild case. No detection of inhibitor development or adverse events were reported in the study.


FDA Rejects BioMarin's Gene Therapy for Hemophilia A

August 19, 2020

In a surprising decision, the FDA refused to approve BioMarin's gene therapy valoctocogene roxaparvovec, also known as valrox. The decision requires BioMarin to finish their phase 3 trial and complete two years of follow up data to show the treatment's durability and efficacy. 


CSL Behring and Ferring Have Issued a Recall of Stimate

July 22, 2020

Ferring  Pharmaceuticals has issued a precautionary, voluntary recall of all batches of Stimate Nasal Spray 1.5 mg/mL. An analysis of the solution showed elevated amounts of the desmopressin content as well as benzalkonium chloride.There is a reasonable probability that the use of the product could pose a hazard to those taking it. Please contact your pharmacy or medical professional if you have questions about this recall.


Data from Biopsy Study of BioMarin's Gene Therapy Shows Positive Result

July 20, 2020

BioMarin’s gene therapy for severe hemophilia A can produce the sustained production of the missing factor VIII for nearly four years. The findings from their biopsy study were shared at the 2020 Congress of the International Society on Thrombosis and Haemostasis, which was held online in July 2020. The treatment is under priority review with the FDA.


Gene Therapy from BioMarin Could be the Most Expensive US Drug Ever

July 20, 2020

BioMarin’s gene therapy that is seeking approval to be the first of its kind from Europe and the United States may also be the most expensive drug to ever hit the market. The company is asking for as much as $3 million per patient; raising questions and concerns about the cost and ability for patients to pay for the potentially life changing treatment.


Phase 1/2 Trial of Gene Therapy for Hemophilia B Shows Promise

July 17, 2020

A phase 1/2 clinical trial shows that single dose of the investigational gene therapy FLT180a can lead to clinically meaningful, sustained increases in the activity of Factor IX. This effectively prevents bleeds and the need for factor replacement therapy in people with severe hemophilia B. FLT180a is a gene therapy that is being developed by Freeline using their AAV protein shell called AAVS3. 


Data Shows Hemlibra Continues to Maintain a Favorable Safety Profile

July 15, 2020

A recent clinical trial shows that Hemlibra (emicizumab) continues to be safe when given as a preventative treatment to avoid spontaneous bleeds in people with hemophilia A with inhibitors. In addition, a separate analysis showed that patients taking Hemlibra may  be able to undergo minor surgeries safely without additional prophylactic treatment.  


Roche Posts Update on Hemophilia A Gene Therapy Data

July 13, 2020

Roche has posted updated clinical data on it's gene therapy for hemophilia A. The therapy, SPK-8011, was recently acquired in Roche's takeover of Spark Therapeutics. The study shows the therapy shows improvements in bleeds as far out as three years but it will be 2021 before phase 3 dosing begins. 

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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