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

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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 98
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Drug-resistant epilepsy drug Ontozry backed by NICE

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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.

Drugs 96
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Free access to Zolgensma curbed, says Novartis

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” 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.

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NICE says yes to Novartis’ multiple sclerosis therapy Kesimpta

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The cost-effectiveness agency has said that anti-CD20 antibody Kesimpta (ofatumumab) can be prescribed via the NHS in England and Wales as a treatment for adults with RMS with active disease, as either a first-line therapy or after alternative drugs have been tried. It was backed by NICE for NHS use in July 2018.

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

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NICE has rejected routine NHS funding for Roche’s Evrysdi (risdiplam) for spinal muscular atrophy drug in draft guidance, a decision which doesn’t come as a surprise to patient association SMA UK.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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Novartis’ $2 Million Gene Therapy Zolgensma Shows ‘Remarkable’ Results and Offers Hope for Children with SMA

XTalks

With a price tag of over $2.5 In England, five-month old baby Arthur became the country’s first SMA patient to receive the Zolgensma gene therapy last week through the National Health Service (NHS). million, Zolgensma is currently the world’s most expensive drug. Related: Is $2 Million Too Much For FDA-Approved SMA Gene Therapy?