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- PMLiVE

Novimmune

- PMLiVE

Sanofi/Sobi’s efanesoctocog alfa recommended by CHMP to treat haemophilia A

The rare genetic condition reduces production of factor VIII, an essential protein for blood clotting

Biomarin

BioMarin announces first patient dosed with haemophilia A gene therapy in Europe

The therapy was granted conditional marketing authorisation by the EC in August 2022

- PMLiVE

FDA approves BioMarin’s Roctavian as first gene therapy for severe haemophilia A

Approximately 2,500 patients are expected to be eligible to receive the one-time therapy

- PMLiVE

Sanofi and Sobi’s haemophilia A treatment shows effective bleed protection in children

Current factor VIII therapies usually require patients to receive treatment multiple times a week

- PMLiVE

Sanofi and Sobi’s once-weekly haemophilia A treatment approved by FDA

Current factor VIII therapies usually require patients to receive treatment every two days

Biomarin

BioMarin’s application for haemophilia A gene therapy accepted by FDA

If approved, the treatment would be the first gene therapy in the US for this indication

- PMLiVE

Pfizer and Sangamo Therapeutics reopen recruitment for haemophilia A gene therapy trial

The trial was voluntarily paused by the two partners last year and later placed on hold by the FDA

- PMLiVE

Sanofi and Sobi’s efanesoctocog alfa granted FDA priority review for haemophilia A

Phase 3 results showed once-weekly doses provided clinically meaningful bleed protection for patients with haemophilia A

- PMLiVE

EC approves BioMarin’s gene therapy Roctavian for severe haemophilia A

Approximately 20,000 adults are affected by haemophilia A across more than 70 countries in Europe, the Middle East and Africa

- PMLiVE

Genentech announces positive phase 3 trial results for haemophilia A

Results from the study showed that Hemlibra demonstrated ‘clinically meaningful’ bleed control in patients with moderate or mild haemophilia A, without factor VIII inhibitors

- PMLiVE

Sanofi and Sobi announce positive phase 3 trial results for severe haemophilia A

Results from the study showed once-weekly efanesoctocog alfa doses provided ‘clinically meaningful’ bleed protection for patients with severe haemophilia A

Roche Basel Switzerland

Roche reinforces commitment to the World Federation of Hemophilia Humanitarian Aid Program

The company renewed its pledge to provide preventative treatment until 2028 for haemophilia A patients

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