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Market access of Spinraza (Nusinersen) for spinal muscular atrophy: intellectual property rights, pricing, value and coverage considerations

Gene Therapy volume 24, pages 539–541 (2017)Cite this article

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Over the last few decades, research into treatments for neuromuscular diseases (NMD) has progressed enormously. A very rare NMD is spinal muscular atrophy (SMA), one of the most frequent genetic causes of death for infants.1 SMA originates from a reduced amount of the survival motor neuron (SMN) protein in the body, due to loss-of-function mutations in one of the two SMN genes (SMN1, SMN2), mainly the SMN1 gene.2 Groundbreaking research led to a therapeutic pathway based on gene therapy, to promote the remaining SMN2 gene, existing in all patients, to function like the missing SMN1 gene.3

In December 2016, Spinraza (Nusinersen), Biogen (Durham, NC, USA) was the first treatment to be registered as an orphan drug by the US Food and Drug Administration for SMA. In Europe, a gene therapy in principle classifies as an Advanced Therapy Medicinal Product (ATMP), covered by a regulatory framework with particular incentives for drug developers.4 For Nusinersen, the European Medicines Agency recently granted the Accelerated Assessment status in 2016.5 With respect to its price, it has been reported that the annual wholesale costs of treating a patient with Nusinersen will amount to $750 000 for the first year and $375 000 for subsequent years.6 Biogen argues that this price is in line with that of other orphan drugs for rare diseases.

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Acknowledgements

We thank the two anonymous referees for their insightful feedback.

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  1. KU Leuven Department of Pharmaceutical and Pharmacological Sciences, Leuven, Belgium

    S Simoens & I Huys

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  1. S Simoens
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Correspondence to S Simoens.

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Competing interests

SS and IH are the founders of the KU Leuven Fund on Market Analysis of Biologics and Biosimilars following Loss of Exclusivity (MABEL), in which Biogen participates. The authors declare no conflict of interest.

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Simoens, S., Huys, I. Market access of Spinraza (Nusinersen) for spinal muscular atrophy: intellectual property rights, pricing, value and coverage considerations. Gene Ther 24, 539–541 (2017). https://doi.org/10.1038/gt.2017.79

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