🔴 BreakingDrug approvalRSSToday
SMA treatment Itvisma gets European regulatory panel OK
A European regulatory panel has recommended approval of Itvisma, a one-time gene therapy treatment for spinal muscular atrophy (SMA) in children ages 2 and older. Gene therapy is a type of treatment that fixes problems in a person's genes to help their body work better. The European Commission will make the final decision on whether to approve this treatment for use in Europe.
WHY IT MATTERSIf approved by the European Commission, Itvisma will become available to European SMA patients ages 2+, offering a one-time treatment option that may slow or stop disease progression rather than requiring ongoing infusions.