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. Roche provides the community with a summary of their 2020 work

    Roche has provided us with a summary of its work in the community throughout this year, including information about the risdiplam clinical development programme and progress towards their goal of making risdiplam available to all patients who can benefit from the treatment.

    1. Ensuring patient safety, uninterrupted access to treatment and to support the wider community: Roche has been working closely with clinical trial sites and partners, to safeguard the seamless continuation of their clinical studies and mitigate risks where possible. A number of new initiatives were introduced to safely and comfortably enable the continued participation of individuals in studies. These include:
        • A contactless home drug delivery service
        • Remote monitoring to maintain oversight of the general health of participants in situations where visiting the clinic or hospital for a study appointment was no longer feasible
        • Home nursing services (in countries where this is permitted and feasible)
    2. Initiation of a global Pre-Approval Access (PAA)/ Compassionate Use (CU) programme for risdiplam in countries where possible (e.g. where applicable laws and regulations allow such programmes and which fulfil the criteria based on applicable company policy). The programme is offering patients who have no other treatment options and are facing the most urgent medical need, the opportunity to access risdiplam prior to regulatory approval. Eligibility criteria vary from country to country. Currently, the risdiplam PAA/CU programme is open in more than 50 countries worldwide.
    3. Amendments to the eligibility criteria for the PAA/CU programme (in response to COVID-19 pandemic pressure on health systems): to also include patients whose current treatment has been interrupted as a direct consequence of the COVID-19 outbreak. This change to the risdiplam PAA/CU programme applies to patients who are not able to continue receiving their therapies due to the COVID-19 outbreak and who could subsequently face the risk of their condition worsening due to treatment interruption. People interested in accessing risdiplam via PAA/CU should discuss their options with their treating physician. The decision to apply for the programme is one that should be made by the treating physician after she/he has explored and discussed all possible options with the patient or family.
    4. Joined our European Alliance for Newborn Screening in SMA: This initiative, led by us, advocates for newborn screening programmes in Europe to include a test for SMA by 2025.

    Clinical Trial Data Updates

    During 2020, Roche announced positive data from three clinical trials, evaluating the efficacy and safety of risdiplam in a broad spectrum of patients living with SMA. Today, more than 450 people are participating in the risdiplam clinical development programme.

    Roche has shared some very helpful lay summaries which explains general information about the studies, such as who took part, the results of Parts 1 and 2, the scales used to measure movement and how the studies helped patients and researchers:

    1. About FIREFISH
    2. About SUNFISH

    For more information