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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FDA 111

Pharmafile

NOVEMBER 23, 2022

CSL Behring’s haemophilia B gene therapy has recently been approved by US regulators, however the treatment has an enormous price tag. Costing $3.5m per dose, the one-off infusion therapy has become the world’s most expensive medicine currently available.

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PharmaShots

MAY 19, 2023

Alimera Acquires Rights from EyePoint Pharmaceuticals to Commercialise Yutiq in the US Date: May 19, 2023 | Tags: Alimera, EyePoint Pharmaceuticals, Yutiq, Iluvien, chronic non-infectious uveitis, US, Pharma Sobi Reports EMA’s Validation of MAA for Efanesoctocog Alfa to Treat Haemophilia A Date: May 19, 2023 | Tags: Sobi, Efanesoctocog Alfa, (..)

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Medical Schools FDA Immunization Vaccines 40

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 News of the pause came as UK cost-effectiveness watchdog NICE rejected Zynteglo for regular NHS use in first draft guidance, despite a confidential discount to its price tag of around €1.57

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Immunization 52

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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