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Clinical Trial Updates (SCD)

Ferriprox® (deferiprone)

  • Product Information

    Product Information

    Scientific name: Deferiprone
    Brand name: Ferriprox®
    RESPONSIBLE: Disc Medicine Inc and Mabwell Therapeutics

  • Clinical Trial/Study Information

    Clinical Trial/Study Information

    Trial Name: N/A
    Code: N/A (Τo be initiated in second half of 2023)
    Phase: 1 (Proof-of Mechanism)
    Eligible patient diagnosis: Healthy Volunteers
    No. of Patients enrolled: N/A
    Study Sites: N/A Sites per country

    Anticipated completion date: 2024
    Scope of the Study / Aim: Targets TMPRSS6 with an antibody to enable the increase of hepcidin and reduction of iron

  • Regulatory Information

    Regulatory Information

    Status: Not Authorised

    Additional notable points:

    • FDA: Received acceptance of an Investigational New Drug (IND) application from the U.S. Food and Drug Administration (FDA) in November 2022.

Update: 30 June 2025

Data presented at the 30th EHA Annual Congress (12 – 15 June) in Milan, Italy showed the real-world safety profile for deferiprone twice-a-day (BID) from 425 patients, children and adults, including 133 with thalassaemia and 197 with SCD, in the USA.

The most frequent adverse events are shown in the table below:

 

 

 

 

 

 

 

 

 

 

 

Source: REAL-WORLD SAFETY PROFILE OF DEFERIPRONE TWICE-A-DAY (DFP BID) FOR… – Sheth S – EHA-4816 – Jun 14 2025

 

Update: 31 March 2025

No update available.

 

Update: 19 December 2024

No update available.

 

Update: 30 September 2024

No update available.

 

Update: 30 June 2024

No update available.

 

Update: 31 March 2024

No update available.

 

Update: 20 December 2023

No update available.

 

Update: 30 September 2023

No update available.

 

Update: 30 June 2023

Health Canada has approved Ferriprox® extended-release (twice daily) tablets (1,000mg) for patients with transfusional iron overload due to thalassaemia when current chelation therapy is inadequate, or SCD or other anemias.

Ferriprox® was previously approved in Canada for the treatment of patients with transfusional iron overload due to thalassemia when current chelation therapy is inadequate in 2015, and subsequently approved for the treatment of iron overload in patients with SCD or other anemias in 2021.

Source: https://www.prnewswire.com/news-releases/chiesi-global-rare-diseases-announces-approval-of-ferriprox-mr-deferiprone-extended-release-tablets-in-canada-301787472.html


Update: 31 March 2023

No update available.

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