pharmaphorum

NOVEMBER 19, 2021

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 92

Drugs Gene Therapy Genetic Disease Gene 92

pharmaphorum

JUNE 2, 2021

NICE has rejected routine NHS funding for Roche’s Evrysdi (risdiplam) for spinal muscular atrophy drug in draft guidance, a decision which doesn’t come as a surprise to patient association SMA UK. Comments can be submitted until 23 June. — Spinal Muscular Atrophy UK (@SMA_UK_) June 2, 2021.

Gene Therapy 52

Gene Therapy Gene Drugs Medicine 52

pharmaphorum

JANUARY 9, 2023

Along with Zolgensma – which made its debut in 2019 – Biogen has had approval to market its antisense-based therapy Spinraza (nusinersen) since 2016, while Roche got a green light for its orally-administered therapy Evrysdi (risdiplam) in 2020. ” Zolgensma is one of the most expensive therapies available, with a price tag of around $2.1

Gene Therapy 114

Gene Therapy Genetics Gene Clinical Trials 114

pharmaphorum

AUGUST 10, 2020

Roche is hoping to undercut hugely expensive rivals after the FDA approved its oral spinal muscular atrophy (SMA) drug Evrysdi (risdiplam). Roche has decided to price Evrysdi according to its weight, costing up to $340,000 per year, with the cost being under $100,000 annually for some younger patients. Zolgensma costs about $2.1m

FDA Approval 57

FDA Approval Drugs Gene Therapy Protein 57