SMA medicines landscape

In Focus

Approved medicines for SMA

There are currently three medicines for SMA approved in most countries across Europe. Reimbursement of these across Europe varies massively. All three medicines increase the production of SMN, a protein essential for the survival of motor neurons.

Spinraza™

This medicine is marketed by the pharmaceutical company Biogen and was approved for the treatment of SMA in Europe in June 2017 by the European Commission.

Zolgensma™

This medicine is marketed by the pharmaceutical company Novartis Gene Therapies (formerly: AveXis) and approved for the treatment of SMA in Europe in May 2020 by the European Commission.

Evrysdi™

This medicine is marketed by the pharmaceutical company Roche and approved for the treatment of SMA in Europe in March 2021 by the European Commission.

 

“Our goal must be to gather evidence and drive research
further to personalize medicine, so that each individual
patient may have access to the therapy that provides
individual optimal therapeutic benefit.”

Dr Nicole Gusset, President of SMA Europe

SMA Europe in the medicines landscape

  • We are actively working with all stakeholders to realise “concepts” and make them accessible to our community. We want to bring the experience from daily living with SMA to the decision making tables early. We are involved in working with pharmaceutical industry, health care professionals and regulatory bodies such as European Medicines Agency (EMA).
  • We have initiated the OdySMA project With OdySMA, we want to mobilise the community to highlight challenges in access to medicines and care; to ensure no one is left behind. With our atlas we  highlight based on evidence gaps and challenges across Europe.
  • We have published articles to elevate the voice of the community into the scientific arena. With our articles we advocate for evidence-based patient-relevant decision-making.

Gusset et al. 2021. Decision for Life. Treatment decisions in newly diagnosed families with spinal muscular atrophy. EJPN: 30; 105-107. doi.org/10.1016/j.ejpn.2020.11.003 

Erbas & Gusset. 2021. The need for evidence-based treatment decisions in spinal muscular atrophy type 0. Ann Clin. Transl. Neurol. 2021. doi: 10.1002/acn3.51459

OdySMA

Decision for life

Treatment Decision

“The lack of data about which treatment is best for an individual patient leads to the risk that decisions today are influenced by subjective and individual preferences of clinicians, by risks and benefits for hospitals, and by wishful thinking of parents e rather than by purely evidence-based medical rationale.”1 

Treatment decisions should always be taken together between the patient/family and a specialised neurologist. Considering that quality of life is strongly influenced by individual perception and that forecasts are challenging, we advocate for fully informed-decision making that also should include the connection to a specialised patient organisation, especially also in newly diagnosed families. 

It is important that individuals with SMA begin therapy as soon after diagnosis as possible (see also New-born Screening Alliance) to prevent additional motor neuron loss.

1:Gusset et al. 2021. Decision for Life. Treatment decisions in newly diagnosed families with spinal muscular atrophy. EJPN.

NBS Alliance

Looking forward

Future of SMA Medicines Landscape

By elevating the voice of the community we aim to influence scientific decision-making towards patient-relevant approaches. And we want to impact the future today. We are committed to understanding open issues and challenges, and opening up new areas for SMA; for example:

  • optimal medicines treatment for each individual living with SMA
  • combination of different treatments:
    • it is still unclear whether combining two similar treatments brings additional benefit
    • but many researchers think that a combination of SMN-enhancing and non-SMN treatments might provide the most benefit
  • regenerative medicines
  • combination of medicines, technology and care products

With SMArt Horizon we as a patient organization are actively scanning the research fields for ground-breaking new research and trying to identify new innovative approaches for the treatment of SMA; and bring these to the community.