haemophilia A

News

Can Pfizer, Sangamo energise haemophilia A gene therapy?

There has been a gene therapy on the market for people with the bleeding disorder haemophilia A since 2022, but take-up has been very slow.

News

Sobi gets EU okay for long-acting haemophilia A drug

The European Commission has approved Sobi’s long-acting factor VIII replacement therapy Altuvoct for the treatment of haemophilia A in the EU, with a broad label that spans all ages and any

R&D

Case Study: MHRA

Following MHRA approval of a clinical trial, a study on Nuwiq, a recombinant FVIII therapy for the treatment of haemophilia A, can proceed.

News

ASH: Pfizer builds case for long-acting haemophilia drug

Pfizer’s once-weekly antibody injection for haemophilia A and B, marstacimab, could be on track for regulatory approvals based on solid results in a phase 3 trial reported

News

BioMarin’s haemophilia A gene therapy gets US approval

The FDA has approved BioMarin Pharma’s Roctavian as the first gene therapy for haemophilia A, giving patients an alternative to regular injections of blood clotting factor

News

Pfizer says haemophilia drug works, but safety will be key

Pfizer has reported that its anti-tissue factor pathway inhibitor marstacimab is effective at reducing bleed rates in patients with haemophilia A and B, working as well as

Can Pfizer, Sangamo energise haemophilia A gene therapy?

Sobi gets EU okay for long-acting haemophilia A drug

Case Study: MHRA

ASH: Pfizer builds case for long-acting haemophilia drug

BioMarin’s haemophilia A gene therapy gets US approval

Pfizer says haemophilia drug works, but safety will be key

Unpacking Trump’s executive order on drug pricing, with Alic...

Health Trends 2025: Reimagining What's Possible

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haemophilia A

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