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). . Concerns about the high price tag have been raised in an article in Nature, particularly whether it will be affordable for low to middle-income countries. .

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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. Haemophilia B is a rare condition. per dose, it is the most expensive single-use gene therapy in the US.

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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 News of the pause came as UK cost-effectiveness watchdog NICE rejected Zynteglo for regular NHS use in first draft guidance, despite a confidential discount to its price tag of around €1.57

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pharmaphorum

AUGUST 26, 2022

BioMarin Pharma has its landmark first approval for Roctavian – the first for a haemophilia gene therapy – and will now have to see if that can convert that into a viable business in Europe. million spread over five years.

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pharmaphorum

NOVEMBER 16, 2022

However, with a price tag of €1.58 However, a recently released report from the organisation could blow the price tag of Zolgensma out of the water. Both of the treatments are gene therapies for haemophilia, and both are one-time therapies. How to navigate the new climate. At more than $2.1

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