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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.

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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.

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NICE says yes to Novartis’ multiple sclerosis therapy Kesimpta

pharmaphorum

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.

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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. An opinion from the Scottish Medicine Consortium (SMC) on the use of the drug by NHS Scotland is expected to be published early in 2022.

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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NICE has rejected routine NHS funding for Roche’s Evrysdi (risdiplam) for spinal muscular atrophy drug in draft guidance, a decision which doesn’t come as a surprise to patient association SMA UK.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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New purification method could enhance protein drug manufacturing

European Pharmaceutical Review

To create the surface required, the team coated gold nanoparticles with bioconjugates maleimide (MAL) and N -hydroxysuccinimide ester (NHS). The authors explained that these are commonly used for tagging proteins for study or attaching protein drugs to drug-delivering nanoparticles.

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After a slow start for expensive CAR-T therapies, drug developers revisit oral therapies for blood cancer

pharmaphorum

price tag of $475,000 when it was first launched in 2017 – and it becomes apparent that these may not be desirable treatment options for every patient and in every setting. Add to this the considerable cost of these medications – the first approved CAR-T, Novartis’ Kymriah (tisagenlecleucel), had a U.S. Professional reception of the findings.