pharmaphorum

JANUARY 9, 2023

Novartis’ programme providing free access to its spinal muscular atrophy (SMA) gene therapy Zolgensma is being scaled back to a dozen countries worldwide, according to the company. ” Zolgensma is one of the most expensive therapies available, with a price tag of around $2.1 Pause in UK access.

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Pharmaceutical Technology

SEPTEMBER 30, 2022

Spark Therapeutics’ Luxturna, indicated for inherited retinal disease (IRD), was the first gene therapy to be approved, in 2017, with a price tag of $850,000 for each eye. bluebird then broke its own pricing record when the $3m Skysona was approved, and now faces the unique challenge of selling the world's two most expensive drugs.

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XTalks

JUNE 8, 2021

Novartis’ Zolgensma (onasemnogene abeparvovec) gene therapy has been making significant strides as of late, including dosing of the first Spinal Muscular Atrophy (SMA) patient with the treatment in the UK last week. Prior to Zolgensma, treatments could only help manage symptoms and attempt to improve quality of life.

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pharmaphorum

NOVEMBER 19, 2021

The agreement has been hailed by patient associations, as it adds to current treatment options for the progressive muscle wasting disease – Biogen’s injectable Spinraza (nusinersen) and one-shot gene therapy with Novartis’ Zolgensma (onasemnogene abeparvovec) – which are also backed by NICE.

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Pharmaceutical Technology

FEBRUARY 10, 2023

This is not the first treatment to come with a high price tag. It is also likely that further RSA frameworks will be established in Latin America, after Argentina and Brazil recently became pioneers by reimbursing Novartis’s Zolgensma (onasemnogene abeparvovec) under novel RSAs.

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pharmaphorum

JUNE 2, 2021

The orally-administered drug is the third approved treatment for SMA after Biogen’s injectable Spinraza (nusinersen) and Novartis’ one-shot gene therapy Zolgensma (onasemnogene abeparvovec), which was administered to the first NHS patient this week. — Spinal Muscular Atrophy UK (@SMA_UK_) June 2, 2021.

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XTalks

NOVEMBER 24, 2022

Prior to Hemgenix, bluebird bio’s recently approved gene therapy Skysona for the rare neurological disorder cerebral adrenoleukodystrophy (CALD) held the record for the highest-costing drug in the world at $3 million and before that, it was Novartis’ one-dose gene therapy Zolgensma which is priced at about $2 million.

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