Novartis gene therapy set to enhance SMA treatment

OAV101 IT expands potential for type II SMA patients

Novartis has announced that its intrathecal (IT) formulation of onasemnogene abeparvovec (OAV101 IT) achieved the primary endpoint in a phase 3 STEER study.

This success could broaden the eligible patient population for the gene transfer therapy, according to GlobalData. The study involved paediatric patients aged 2–17 with type II spinal muscular atrophy (SMA).

GlobalData’s report reveals that combined sales of IT and intravenous formulations of onasemnogene abeparvovec are projected to increase due to growing SMA awareness and newborn screening (NBS) implementation.

Novartis’ Zolgensma, an IV formulation, was FDA-approved in 2019 for patients under two with SMA. However, the new IT formulation could extend the therapy to older children.

OAV101 IT demonstrated clinical benefits in treatment-naïve type II SMA patients who could sit but had never walked independently.

The safety profile was favourable, with common adverse events including upper respiratory tract infection, pyrexia, and vomiting. “However, safety concerns have previously been raised,” said Christie Wong, Managing Neurology Analyst at GlobalData. “In 2019, the FDA implemented a partial clinical hold due to animal study findings, but this hold was lifted in 2021.”

Novartis plans to share the STEER results with regulatory agencies, including the FDA, in 2025. If approved, OAV101 IT could include type II SMA patients under 18 in the treatment population.

“The availability of OAV101 IT would be most beneficial to patients where NBS for SMA is not yet adopted and those who received a delayed diagnosis,” Wong noted.

Key opinion leaders welcomed the therapy for its convenience of a one-time infusion. “If approved, OAV101 IT could significantly enhance patient outcomes in markets where NBS or early diagnosis remains a challenge,” Wong concluded.