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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FDA Insurance 115

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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FDA 105

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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pharmaphorum

FEBRUARY 22, 2021

That therapy uses an adeno-associated virus (AAV) as a vector rather than a lentivirus, a vector type that features in approved gene therapies such as Roche’s Luxturna and Novartis’ Zolgensma. million in Europe. The post Zynteglo halt re-ignites viral vector safety concerns; analysts appeared first on.

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Immunization 52

European Pharmaceutical Review

MAY 11, 2023

In this Q&A, Dr Sitra Tauscher-Wisniewski, Vice President of Clinical Development & Analytics of Novartis Gene Therapies elaborated on the key findings and discussed the importance of collaboration in fast-tracking access to gene therapies such as Zolgensma. 1 This is assuming a $1M price tag for all gene therapies.

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FDA 72