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haemophilia B
This page shows the latest haemophilia B news and features for those working in and with pharma, biotech and healthcare.
Positive results for Sanofi’s haemophilia therapy published in The Lancet
Sanofi’s investigational haemophilia therapy has shown significant improvements in bleed protection across all types of the disorder, according to two phase 3 studies recently published in The Lancet and The ... Haemophilia A and B are lifelong
Latest news
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EC approves first gene therapy for adults with haemophilia B
Specifically, Hemgenix is indicated for adults with severe or moderately severe haemophilia B without a history of factor IX inhibitors. ... we believe can transform the treatment paradigm for both people living with haemophilia B and the healthcare
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Pfizer announces positive phase 3 results for haemophilia B gene therapy
Haemophilia B is a genetic bleeding disorder resulting from missing or insufficient levels of blood clotting factor IX, a protein needed to produce blood clots to stop bleeding. ... In November last year, the US Food and Drug Administration approved CSL
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FDA approves first gene therapy for adults with haemophilia B
The US Food and Drug Administration (FDA) has approved CSL Behring’s (CSL) Hemgenix (etranacogene dezaparvovec) as the first gene therapy for adult patients with haemophilia B. ... For appropriate patients, Hemgenix allows people living with
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Novo Nordisk announces positive phase 3 results for haemophilia treatment
Currently, it is estimated that 30% of people living with haemophilia A and 1-3% of people living with haemophilia B have inhibitors. ... to our haemophilia offering, especially in the haemophilia B with inhibitor population who currently have limited
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FDA removes clinical hold on uniQure’s haemophilia gene therapy
The US Food and Drug Administration (FDA) has removed a clinical hold on Dutch biotech uniQure’s haemophilia B gene therapy, following concerns over a case of cancer in a patient ... In December 2020, the FDA placed a hold on uniQure’s haemophilia B
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20 for 2020 – Five pharma leaders to watch in 2020
pipeline. The company submitted its lumasiran candidate for primary hyperoxaluria to the FDA and has another ATTR amyloidosis candidate – called vutrisiran – in late-stage along with Sanofi-partnered fitusiran for haemophilia
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Deal Watch January 2018
Paying $105 per share in cash (64% premium over Bioverativ’s closing price on 19 January), Sanofi gains the marketed products, Eloctate (haemophilia A therapy) and Alprolix (haemophilia B therapy), as ... Portfolio/pipeline in haemophilia and other
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Deal Watch August 2016
premiums. One commentator suggested BioMarin could be a Plan B for Sanofi. ... This will help with the collaboration with Spark Therapeutics that is developing a treatment for haemophilia B that incorporates a bio-engineered rAAV vector and the academic
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A rare opportunity
Sobi, handing Sobi commercialisation control over the haemophilia A treatment just four months after it received EU approval. ... Last month Alprolix was approved by the European Commission, becoming the first licenced long-acting treatment for
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Deal Watch February 2016
Indeed Baxalta already has ongoing gene therapy programmes for the treatment of haemophilia A and B.
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