Novartis wins conditional EU approval for gene therapy Zolgensma

[May 19, 2020]  ZURICH (Reuters) - Novartis won European approval for its gene therapy Zolgensma for the hereditary disease spinal muscular atrophy (SMA), the Swiss drugmaker said on Tuesday, adding it is in talks over price with countries in hopes of a quick launch.

The European Commission gave conditional approval to the therapy, whose U.S. price is $2.1 million, for patients with a clinical diagnosis of SMA Type 1, the most severe form of the disease, or SMA patients with up to three copies of a specific gene that helps doctors predict how severe the disease will be.

The EU approval covers babies and young children with SMA up to 21 kilograms. The medicine also has approval in Japan.

Novartis got Zolgensma with its $8.7 billion takeover of U.S.-based AveXis in 2018 and has forecast more than $1 billion in sales for the treatment, which in trials has been shown to significantly improve survival and motor function of babies with SMA, in particular those treated before symptoms develop.

Novartis said it is in talks with nations over what it calls its "Day One" access program, which the Basel-based drugmaker said is aimed at speeding up treatment by dealing with payment issues up front, even before national pricing and reimbursement agreements with individual countries are in place.

"The 'Day One' access program ensures the cost of patients treated before national pricing and reimbursement agreements are in place align with the value-based prices negotiated following clinical and economic assessments," Novartis said, adding the medicine will be immediately available in France.

Drug pricing in Europe varies from country to country, often relying on individual negotiations with regulators and pricing watchdogs that can slow down access, including in instances where officials conclude companies are seeking too much money for their medicines relative to the value they bring.

(Reporting by John Miller; editing by Thomas Seythal and Brenna Hughes Neghaiwi)

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