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     151  0 Kommentare Roche’s Evrysdi approved by European Commission as first and only at home treatment for spinal muscular atrophy

    • Evrysdi has proven efficacy in adults, children and babies two months and older, as shown in two pivotal clinical trials
    • Roche is actively engaging with health authorities in the EU to achieve broad and rapid access for people with SMA
    • More than 3,000 patients now treated with Evrysdi in clinical trial, compassionate use and real-world settings

    Basel, 30 March 2021 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that the European Commission (EC) has approved Evrysdi (risdiplam) for the treatment of 5q spinal muscular atrophy (SMA) in patients two months of age and older, with a clinical diagnosis of SMA Type 1, Type 2 or Type 3 or with one to four SMN2 copies. SMA is a leading genetic cause of death in infants and 5q SMA is the most common form of the disease. The condition causes muscle weakness and progressive loss of movement and significant unmet need remains, particularly in adults living with this condition.

    “Today’s approval of Evrysdi, the first and only SMA treatment with proven efficacy that can be taken at home, potentially transforms treatment options for a broad range of people with SMA living in the EU,” said Levi Garraway, M.D., Ph. D., Roche’s Chief Medical Officer and Head of Global Product Development. “By avoiding the need for in-hospital administration, Evrysdi can reduce the treatment burden on those living with SMA, their caregivers and healthcare systems. We thank the SMA community for their partnership, the trust they have placed in us and their unyielding commitment to achieve this significant milestone.”

    The approval is based on data from two clinical studies, designed to represent a broad spectrum of people living with SMA: FIREFISH in symptomatic Type 1 infants aged 2 to 7 months and SUNFISH in symptomatic Type 2 and 3 children and adults aged 2 to 25 years. SUNFISH is the first and only placebo-controlled trial to include adults with Types 2 and 3 SMA. Evrysdi demonstrated a favourable efficacy and safety profile, with the safety profile established across both trials.

    "We welcome today’s approval of Evrysdi for people with SMA in Europe, and are proud of the role we have played in its development and of our partnership with Roche,” said Dr Nicole Gusset, President of SMA Europe. “A recent survey conducted by SMA Europe showed that a large proportion of people with SMA in the EU were not receiving an approved treatment which leaves them feeling helpless and frustrated. It is vital that we work together with health authorities, regulators and industry to ensure we can get this medicine to the patients who need it as soon as possible.’’

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    Roche’s Evrysdi approved by European Commission as first and only at home treatment for spinal muscular atrophy Evrysdi has proven efficacy in adults, children and babies two months and older, as shown in two pivotal clinical trials Roche is actively engaging with health authorities in the EU to achieve broad and rapid access for people with SMA More than …