Nusinersen is a Antisense Oligonucleotide owned by Biogen, and is involved in 18 clinical trials, of which 8 were completed, 9 are ongoing, and 1 is planned.

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.

The revenue for Nusinersen is expected to reach a total of $14.5bn through 2038. This change impacts the valuation of this asset and is an important factor to understand the current value of the drug in a clinical process. View the complete picture with the Nusinersen NPV Report.

Nusinersen is currently owned by Biogen.

Nusinersen Overview

Nusinersen (Spinraza) is an antisense oligonucleotide. It is formulated as a powder for solution and solution for the intrathecal route of administration. Spinraza is indicated for the treatment of spinal muscular atrophy in pediatric and adult patients. ISIS-SMNRx is based on antisense technology platform.

Biogen Overview

Biogen is a biopharmaceutical company that discovers, develops, and delivers drugs and biosimilars for the treatment of various neurological and neurodegenerative diseases. The company’s marketed products include Avonex (interferon beta-1a), Tysabri (natalizumab), Tecfidera (dimethyl fumarate), Fampyra (prolonged-release fampridine tablets), and Plegridy (peginterferon beta-1a) for the treatment of multiple sclerosis (MS); Spinraza (nusinersen) for spinal muscular atrophy (SMA); and Fumaderm (fumaric acid esters) for severe plaque psoriasis. It has several product candidates targeting various indications such as MS, Parkinson’s disease, CNS and neuromuscular disorders, Alzheimer’s disease, and idiopathic pulmonary fibrosis and stroke. The company sells its products through direct sales force, marketing groups, and distributors in the Americas, Europe, Asia, and other territories. Biogen is headquartered in Cambridge, Massachusetts, the US.

The company reported revenues of (US Dollars) US$10,981.7 million for the fiscal year ended December 2021 (FY2021), a decrease of 18.3% over FY2020. In FY2021, the company’s operating margin was 25.9%, compared to an operating margin of 33.8% in FY2020. In FY2021, the company recorded a net margin of 14.2%, compared to a net margin of 29.8% in FY2020. The company reported revenues of US$2,508.5 million for the third quarter ended September 2022, a decrease of 3.1% over the previous quarter.

Quick View – Nusinersen

Report Segments
  • Innovator (NME)
Drug Name
  • Nusinersen
Administration Pathway
  • Intrathecal
Therapeutic Areas
  • Central Nervous System
Key Companies
Highest Development Stage
  • Marketed


GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article.

To create this model, GlobalData takes into account factors including patent law, known and projected regulatory approval processes, cash flows, drug margins and company expenses. Combining these data points with GlobalData’s world class analysis creates high value models that companies can use to help in evaluation processes for each drug or company.

The rNPV method integrates the probability of a drug reaching a clinical stage into the cash flow at that time, which provides a more accurate valuation, as it considers the probability that the drug never makes it through the clinical pathway to commercialization. GlobalData’s rNPV model uses proprietary likelihood of approval (LoA) and phase transition success rate (PTSR) data for the indication in the highest development stage, which can be found on GlobalData’s Pharmaceutical Intelligence Center.