The Global Drug sales of Spinraza stood at USD 1,794 Millions in 2022
The indicator recorded a historical decline (CAGR) of5.07% between 2019 to 2022 and is expected to decline by ...
GlobalData projects the indicator to decline at a CAGR of...
Nusinersen (Spinraza) is an antisense oligonucleotide. It is formulated as a powder for solution and solution for the intrathecal route of administration.
Mechanism of Action
Nusinersen is SMN2 gene activator. ISIS-SMNRx compensates for the defect by promoting the production of functional SMN protein. Spinal muscular atrophy is caused by a loss or defect in the survival motor SMN1 gene leading to a decrease in the protein SMN. SMN1 gene produces functional SMN protein in healthy humans. SMN2 gene produces a small amount of SMN protein. ISIS-SMNRx enables the SMN2 gene to produce more functional SMN protein by modulating a cellular process, splicing. The drug candidate keeps exon 7 in the RNA which leads to the production of functional SMN protein.
Spinraza Overview
Spinraza is marketed for the treatment of several indications in Central Nervous System therapy area.
Spinraza is indicated for the treatment of spinal muscular atrophy in pediatric and adult patients. ISIS-SMNRx is based on antisense technology platform.
Spinraza was first approved in 2016 and is sold globally including the US, the UK, France, Germany, and Japan by several pharma giants including Biogen Inc. Spinraza drug sales include the historical sales that are derived from company financial reports, and consensus forecasts are calculated based on company-specific broker reports as well as GlobalData’s proprietary methodology.
Biogen’s Spinraza Set to Increase Its Chances to Maintain a Strong Position in Spinal Muscular Atrophy
On January 8 2021, Biogen announced that the first spinal muscular atrophy (SMA) patient had been treated in its Phase IV RESPOND study evaluating the efficacy and safety of Spinraza (nusinersen) in patients with a suboptimal clinical response to Novartis’ Zolgensma (onasemnogene abeparvovec). RESPOND is a two-year, open-label study that will enroll 60 children up to age three years who have the potential for additional clinical improvement after receiving Zolgensma. There will be two groups in this study. The first group will include 40 infants ages nine months or younger who have two copies of the SMN2 gene and received Zolgensma at ages six months or younger. The second study group will include 20 children ages three years or younger. Participants will receive Spinraza as a 12mg dose that has been split into four loading doses, followed by maintenance doses every four months for the two-year study period.
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Germany
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