Novartis’ Gene Therapy: A New Era in SMA Treatment Expansion

Novartis has announced that its intrathecal (IT) formulation of onasemnogene abeparvovec (OAV101 IT) met the primary endpoint in the Phase III STEER study involving patients aged 2–17 years with type II spinal muscular atrophy (SMA). These positive results could expand the eligible patient population for this innovative gene transfer therapy.

Broader Treatment Potential

Novartis’ Zolgensma, an intravenous (IV) formulation of onasemnogene abeparvovec approved in 2019, is currently limited to patients under two years old. The STEER study results suggest that OAV101 IT may offer clinical benefits to older children who can sit but have never walked independently. The safety profile was favorable, with common adverse events including respiratory infections, fever, and vomiting.

Addressing Safety and Approval Path

Although the FDA placed a clinical hold on OAV101 IT in 2019 due to preclinical concerns, it was lifted in 2021 following supportive toxicology data. Novartis plans to submit the STEER results to regulatory agencies, including the FDA, in 2025. If approved, OAV101 IT could significantly expand access to gene transfer therapy.
Convenience and Accessibility

OAV101 IT’s one-time infusion offers an advantage over existing treatments like Biogen’s Spinraza (nusinersen), administered quarterly, and Roche’s daily oral Evrysdi (risdiplam). This makes it especially beneficial for patients in regions without newborn screening (NBS) programs or with delayed diagnoses.

Conclusion

If approved, OAV101 IT could transform SMA care for older pediatric patients, addressing unmet needs and improving outcomes in areas where early diagnosis remains a challenge. This advancement underscores Novartis’ commitment to enhancing treatment options for SMA patients worldwide. Advancements like this highlight the importance of newborn screening, and we encourage you to share your perspectives on its impact.

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