Novartis has revealed encouraging results from the Phase III STEER trial, demonstrating the effectiveness of intrathecal onasemnogene abeparvovec (OAV101 IT) in treating spinal muscular atrophy (SMA) in children and young adults.
This study specifically targeted patients with SMA Type 2, aged between two and 18 years, who could sit but had never walked independently.
The trial's primary goal was achieved, showing improvements in motor function as measured by the Hammersmith Functional Motor Scale - Expanded (HFMSE). This scale is widely recognized for assessing motor skills and tracking disease progression in SMA patients. Individuals treated with OAV101 IT exhibited greater gains in motor ability compared to those who received a sham procedure, indicating the drug’s positive impact.
The findings from the STEER study expand on previous data from the Phase I/II STRONG trial, where patients treated with OAV101 IT experienced notable improvements in motor function within one year. These results were particularly evident in children aged two to five years with SMA Type 2. The latest Phase III data further supports the potential of gene therapy to transform SMA treatment.
STEER is a comprehensive Phase III clinical trial involving patients aged two to under 18 years. Participants were randomly assigned to receive either OAV101 IT or a sham procedure. After 52 weeks, those who initially received the sham treatment were given OAV101 IT, and vice versa. The trial assessed motor function through HFMSE scores and secondary measures like the Revised Upper Limb Module (RULM) scale.
OAV101 IT is a gene therapy designed to address SMA through a one-time intrathecal injection. The drug has been tested in multiple studies, including the STEER, STRONG, and STRENGTH trials, involving a total of approximately 170 patients. This extensive clinical program has generated data supporting the therapy’s safety and efficacy, with follow-up periods extending beyond six years.
The Swiss pharmaceutical company’s single-dose treatment, known as Zolgensma, has received approval in major global markets for use in children under the age of 2 who have the condition.
Additional therapies that have been approved include Spinraza, developed by Biogen, and Evrysdi, an oral medication produced by Roche.
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