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

pharmaphorum

Roche’s oral treatment Evrysdi for the rare genetic disease spinal muscular atrophy (SMA) will be made available on the NHS in England, after NICE reached a three-year access agreement with the company. million price tag make it is the most expensive treatment ever approved for NHS funding.

Drugs 97
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Risk-sharing agreements are growing at a rate of 24%

Pharmaceutical Technology

This is not the first treatment to come with a high price tag. On November 22, 2022, the FDA approved CSL Behring’s Hemgenix (etranacogene dezaparvovec), the first gene therapy treatment for hemophilia B, with a staggering manufacturer price of $3.5

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NICE says yes to Novartis’ multiple sclerosis therapy Kesimpta

pharmaphorum

Novartis has secured backing from NICE for its relapsing multiple sclerosis (RMS) therapy Kesimpta in the UK, just two weeks after the drug was approved by the national drugs regulator. . It was backed by NICE for NHS use in July 2018. The post NICE says yes to Novartis’ multiple sclerosis therapy Kesimpta appeared first on.

Sales 111
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Drug-resistant epilepsy drug Ontozry backed by NICE

pharmaphorum

NHS patients in England and Wales with drug-resistant epilepsy are in line for access to another treatment option, after NICE recommended routine use of Angelini group company Arvelle Pharma’s Ontozry. The post Drug-resistant epilepsy drug Ontozry backed by NICE appeared first on.

Drugs 94
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NICE ‘no’ to Roche’s risdiplam for SMA not unexpected, says patient group

pharmaphorum

NICE has rejected routine NHS funding for Roche’s Evrysdi (risdiplam) for spinal muscular atrophy drug in draft guidance, a decision which doesn’t come as a surprise to patient association SMA UK. “NICE’s initial ‘no’ to approving risdiplam for NHS funding is not unexpected,” said SMA UK this morning.

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Gene Silencing Porphyria Treatment, Givlaari, Finally Wins Over England’s NICE Amid Stellar Long-Term Data

XTalks

After initial rejection from the National Institute for Health and Care Excellence (NICE) last year, the non-departmental public body of the Department of Health in England has now given the green light to the gene silencing treatment Givlaari (givosiran) for the treatment of the rare metabolic disorder, acute intermittent porphyria (AIP).

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Free access to Zolgensma curbed, says Novartis

pharmaphorum

” Zolgensma is one of the most expensive therapies available, with a price tag of around $2.1 ” NHS England said the temporary change in eligibility – which ties in with NICE guidance – has been implemented as there are two other disease-modifying drugs available on the NHS for the treatment of SMA.