News

The purpose of SMA Europe is to provide a framework to stimulate collaboration and accelerate translational research pathways in SMA and promote patient care.

  1. European Medicines Agency grants positive recommendation for conditional marketing authorisation of gene therapy product onasemnogene abeparvovec (Zolgensma®)

    On 27th March, the European Medicines Agency recommended a conditional approval of the breakthrough gene therapy drug onasemnogene abeparvovec (Zolgensma®) for treating spinal muscular atrophy.

    SMA Europe is thrilled that a second therapy that addresses the causes of this life-limiting disease has moved closer to patients. It is a culmination of efforts that have been invested over many years into the development of this innovative, ground-breaking treatment. The global SMA patient community has played an important role in these efforts, funding much of the preclinical research that made the discovery and the development of this therapy possible and providing support during clinical development.

    SMA Europe would like to draw particular attention to Martine Barkats’s pioneering work on scAAV9-mediated gene delivery to treat spinal muscular atrophy at Généthon, a biotechnology institute funded by one of SMA Europe’s founding members, AFM Téléthon. SMA Europe is proud to have awarded Dr Barkats funding for this research.

    Indication

    The European Medicines Agency has recommended Zolgensma® for use in the following categories of patients:

    • patients with 5q spinal muscular atrophy (SMA) having a mutation in both copies of the SMN1 gene and a clinical diagnosis of SMA Type 1, or
    • patients with 5q SMA having a mutation in both copies of the SMN1 gene and up to 3 copies of the SMN2

    Given the drug’s safety profile, SMA Europe understands that Zolgensma® will be limited to patients with a body weight not exceeding 21 kilograms, without age-based restrictions.

    SMA Europe welcomes the unexpected broad indication proposed by the European Medicines Agency for Zolgensma®, including the body weight as the limiting factor. At the same time, SMA Europe is concerned about the manner in which the patient populations have been defined.

    SMA Europe is strongly opposed to limiting access to treatment based on the patients’ disease “type”. Modern neurology describes spinal muscular atrophy as a spectrum disorder in which clinical care should not be determined by the patient’s historical functional abilities. SMA Europe has been consistently calling upon the pharmaceutical industry to design drug development around patients’ current function and disease trajectories and not around the traditional “types”. SMA Europe is, therefore, gravely concerned that a proportion of patients in the European Union may not be able to access this innovative therapy due to the use of an outdated, unreliable classification in Zolgensma® indication.

    Furthermore, SMA Europe finds it concerning that access to an SMN2-independent therapy, as is Zolgensma®, would be offered depending on the patient’s number of SMN2 gene copies. This is particularly alarming in the light of the published evidence that the commonly used methodologies to quantify SMN2 gene copies have an unusually low reliability. It needs to be noted that patients in the US, Japan and other countries can currently access Zolgensma® irrespective of the number of the SMN2 gene copies they have.

    Type of marketing authorisation

    SMA Europe acknowledges that a conditional marketing authorisation in the European Union may pose a challenge to accessing Zolgensma® as a reimbursed therapy in individual countries with different regulatory systems. SMA Europe understands the cautious approach of healthcare regulators to cell and gene therapies and stands ready to work with all stakeholders to facilitate early access to Zolgensma® followed by full regulatory approval in the European Union.

    Access for patients

    SMA Europe, as a long-time funder of preclinical research into SMA treatments, is well aware of the investment required to develop a new advanced therapy. To-date, SMA Europe has committed more than €5 million to scientific research into spinal muscular atrophy and is proud to have helped develop Zolgensma®.

    Nevertheless, SMA Europe is concerned that the manufacturer’s pricing expectations may prevent wide access to this breakthrough therapy both in Europe and beyond. As representatives of the European SMA community, SMA Europe urge all stakeholders to work together towards making Zolgensma® widely available across Europe, fully recognising both the ethical imperative to offer effective treatments to patients and the obligation to ensure long-term sustainability of taxpayer-funded healthcare systems.

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