Pharmaceutical Technology

NOVEMBER 9, 2022

Despite their potential multi-million-dollar sticker price, if haemophilia treatments by BioMarin and CSL Behring prove durable, they could help patients save money compared to current treatments, experts note. A fair price for CSL Behring’s haemophilia B treatment etranacogene dezaparvovec would be between $2.93–2.96

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Drug Discovery World

DECEMBER 20, 2022

The first gene therapy for haemophilia B could soon be available to patients in Europe following a positive opinion from the Committee for Medicinal Products for Human Use (CHMP). . The post First gene therapy for haemophilia B gets go ahead in Europe appeared first on Drug Discovery World (DDW).

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Drug Discovery World

MAY 22, 2023

The cost 2022 has not been shy of approvals – Uniqure and CSL’s Haemophilia B drug even nabbed the title of “Most Expensive Drug” at $3.5 But are these price tags a barrier to access? million per dose. Simply, yes, as we saw when the European Commission rejected the price point for bluebird bio’s Zynteglo.

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Pharmaceutical Technology

JUNE 21, 2023

CSL announced the first patient with haemophilia B has been treated with its recently approved gene therapy Hemgenix (etranacogene dezaparvovec). Hemgenix is the first and only gene therapy approved for haemophilia B. This followed positive results from CSL’s Phase III HOPE-B trial (NCT03569891).

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pharmaphorum

FEBRUARY 22, 2021

The case comes just weeks after UniQure halted a trial of its haemophilia B gene therapy AMT-061 after an unexpected case of liver cancer. News of the pause came as UK cost-effectiveness watchdog NICE rejected Zynteglo for regular NHS use in first draft guidance, despite a confidential discount to its price tag of around €1.57

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Drug Discovery World

NOVEMBER 9, 2023

Vincent Muczynski, PhD, Director, NovalGen, on: ‘Development of autoregulating FVIII-mimetic bispecific antibodies to reduce risk of prothrombotic events in treatment of haemophilia A’.

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