The Committee for Medicinal Products for Human Use of the European Medicines Agency Backs Approval of Novartis' Itvisma For Children 2 Years And Older With 5q Spinal Muscular Atrophy

Novartis today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending marketing authorization for Itvisma® (intrathecal onasemnogene abeparvovec). The opinion supports its use for the treatment of children two years and older, teens, and adults living with 5q spinal muscular atrophy (SMA) with a bi-allelic mutation in the survival motor neuron 1 (SMN1) gene.

Itvisma is uniquely designed to address the genetic root cause of SMA with a one-time fixed dose that does not need to be adjusted for age or body weight.1 By replacing the SMN1 gene, Itvisma can improve motor function, offering the potential to reduce the need for chronically administered treatment associated with other available therapies for this population.1

The CHMP opinion is based on data from the registrational STEER study, and supportive Phase IIIb STRENGTH and Phase I/II STRONG studies.1,2,3 In STEER, Itvisma demonstrated a statistically significant 2.39-point improvement in the Hammersmith Functional Motor Scale (HFMSE) with effects sustained over 52 weeks of follow-up.1,2,3 The STEER and STRENGTH studies also showed clinically meaningful benefit for treatment-naïve and pre-treated patients.1,2 



Nicole Gusset, CEO, SMA Europe, said: "Living with SMA affects every stage of life, including education, employment, and independence. Older children, teenagers, and adults face fewer opportunities to benefit from innovative therapies. A positive CHMP opinion for Itvisma is an important step toward addressing this gap."



Professor Tim Hagenacker, Department of Neurology, University Hospital Essen, Essen, Germany, said: "Itvisma met the primary endpoint showing motor function improvement versus placebo. Even a 1-point difference in the HFSME can translate into tangible functional gains for individuals with SMA, such as the ability to grasp a pen. Preserving existing capabilities is critical, as maintaining independence and autonomy is a central goal of care for patients living with a progressive neuromuscular disease."



"Today's positive CHMP opinion is a significant step towards potentially reducing the long-term burden of chronic treatment administration for patients living with SMA in Europe," said Patrick Horber, MD, President, International, Novartis. "Building on the established role of Zolgensma for babies and young children with SMA, this opinion for Itvisma reflects our ambition to expand treatment options for a broader patient population in SMA. It also underscores Novartis' commitment to pushing scientific boundaries to address unmet needs and improve long-term outcomes across the SMA and rare disease community."



Key efficacy results from the registrational STEER study

The most frequently reported adverse reactions were similar across the STEER, STRENGTH and STRONG studies.1,2,3 Results from the STEER and STRENGTH studies were published in Nature Medicine.



Following the CHMP's recommendation for approval, the European Commission (EC) is expected to issue a final decision within approximately two months.