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NICE reaches a deal with Roche on access to oral SMA drug Evrysdi

pharmaphorum

Evrysdi was approved by the Medicines and Healthcare products Regulatory Agency (MHRA) in May, a couple of months after the drug also got a green light in the EU. million price tag make it is the most expensive treatment ever approved for NHS funding.

Drugs 98
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First gene therapy trial for Hunter syndrome opens in Manchester, UK

Drug Discovery World

The UK Medicines and Healthcare Products Regulatory Agency (MHRA), Research Ethics Committee (REC), and Health Research Authority (HRA) have all approved the clinical trial application that was submitted by The University of Manchester in August 2022.

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NICE says yes to Novartis’ multiple sclerosis therapy Kesimpta

pharmaphorum

NICE’s final appraisal document for Kesimpta will be followed by technology appraisal guidance (TAG) that will pave the way for NHS prescribing in England and Wales. The Scottish Medicines Consortium is expected to publish its final advice on the drug later this year.

Sales 110
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NICE ‘no’ to Roche’s risdiplam for SMA not unexpected, says patient group

pharmaphorum

Evrysdi was approved by the Medicines and Healthcare products Regulatory Agency (MHRA) on 20 May, almost two months after the drug go the go-ahead in the EU. million price tag make it is the most expensive treatment ever approved for NHS funding, as it provides “exceptional benefit” to babies with the disease.

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CSL doses first patient with haemophilia B gene therapy Hemgenix

Pharmaceutical Technology

The high price tag may be hard to defend , but with lifetime treatment costs for haemophilia B reaching as high as $23m for some, the single-dose treatment could prove far more cost-effective. Despite the limited market, the price tags of haemophilia make it potentially lucrative. Haemophilia B is a rare condition.