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’s risdiplam granted PRIME designation by EMA

    Roche's risdiplam granted PRIME designation by EMARoche’s risdiplam has been ganted PRIME designation by the European Medicines Agency (EMA) for treatment of spinal muscular atrophy (SMA)

    • Risdiplam has the potential to be the first oral medicine for the treatment of SMA Types 1, 2 and 3;
    • European Medicines Agency PRIME (PRIority MEdicines) status is granted to medicines that may offer a major therapeutic advantage over existing treatments, or benefit patients without treatment options
    • Risdiplam is currently being investigated in three global, multicentre clinical trials in all types of SMA

    Roche today announced that the European Medicines Agency has granted PRIME (PRIority MEdicines) designation for the company’s investigational oral medicine risdiplam (RG7916) for the treatment of people with SMA. PRIME designation is granted by the EMA to support data generation and development plans for promising medicines, providing a pathway for accelerated evaluation by the agency, and thus potentially enabling them to reach patients earlier.

    Risdiplam, an orally administered, survival motor neuron-2 (SMN2) gene splicing modifier, has shown improvements in motor function in people with SMA Types 1, 2 and 3. An increasing body of clinical evidence suggests that SMA is a multi-system disorder and the loss of SMN protein may affect many tissues and cells beyond the central nervous system. Risdiplam is systemically distributed and designed to durably increase SMN protein levels in the central nervous system and throughout the body.

    Roche leads the clinical development of risdiplam as part of a collaboration with the SMA Foundation and PTC Therapeutics.

    “SMA is the leading genetic cause of death in young children, and families and clinicians continue to seek alternative treatment options for this progressively debilitating and life-threatening disease,” said Sandra Horning, MD, Roche’s Chief Medical Officer and Head of Global Product Development. “The EMA’s decision to grant PRIME designation recognises the potential of the oral systemic agent risdiplam to deliver clinically meaningful results for patients and address a continuing medical need in SMA.”

    PRIME designation for risdiplam is based on data from Part 1 of the pivotal studies FIREFISH (evaluating safety and determining dosage in infants with Type 1 SMA) and SUNFISH (in children and adults with Type 2 and 3 SMA) as well as a continuing medical need for alternative treatments and administration options for patients with SMA.

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