Analytics company GlobalData has shared its optimism regarding the future of Novartis’ spinal muscular atrophy (SMA) gene transfer therapy – onasemnogene abeparvovec (OAV101 IT) – which could become available to a broader patient population.
This is following positive results from Novartis’ Phase III STEER study (NCT05089656), which met its primary endpoint when testing the intrathecal formulation of the drug.
Smarter leaders trust GlobalData
Novartis’ has already marketed an intravenous formulation of OAV101 IT, called Zolgensma. Approved by the FDA in 2019, it is currently the only gene therapy for SMA.
However, the eligible patient population indicated for Novartis’ Zolgensma is limited; the drug is only available to paediatric patients under two years of age with SMA with biallelic mutations in the survival motor neuron 1 (SMN1) gene.
GlobalData expects that the intrathecal formulation will shakeup the market. According to the company’s managing neurology analyst Christie Wong, “If approved, OAV101 IT could significantly enhance patient outcomes in markets where NBS or early diagnosis remains a challenge.”
GlobalData is the parent company of Clinical Trials Arena.
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By GlobalDataWith FDA approval, Novartis’ gene transfer therapy would be made available to type II SMA patients under 18 years of age, representing a much broader patient population. Key opinion leaders (KOLs) consulted by GlobalData also pointed out that OAV101 IT would offer patients the convenience of a one-time infusion, compared to other treatments on the market. These include Biogen’s once-quarterly IT administration of Spinraza (nusinersen) and Roche’s once-daily Evrysdi (risdiplam).
Considering the growing market in its recent report, Spinal Muscular Atrophy: Opportunity Assessment and Forecast, GlobalData forecasts that, across the seven major markets (France, Germany, Italy, Spain, the UK, the US and Japan), the combined sales of intrathecal and intravenous formulations of OAV101 IT will increase from $660.6m in 2023 to $763.8m by 2033.
The analytics company attributes the predicted growth in the OAV101 IT market to the shift in awareness of SMA and the associated implementation of newborn screening (NBS) across the markets.
Wong commented that: “The KOLs previously interviewed by GlobalData noted that the implementation of NBS initiatives in their countries has greatly facilitated disease detection during the pre-symptomatic stage and has enabled many eligible patients to receive Zolgensma. The availability of OAV101 IT would be most beneficial to patients where NBS for SMA is not yet adopted in their country and/or patients who received a delayed diagnosis.”
The results of the STEER trial will be shared with the FDA and other regulatory bodies. The Phase III study was a randomised, double-blind, sham-controlled trial, which involved 100 paediatric patients aged between two and 17, all of whom had type II SMA. It met its primary endpoint, showing an increase from baseline in Hammersmith Functional Motor Scale – Expanded (HFMSE) total score in patients with SMA treated with intrathecal OAV101 IT.
However, despite optimism for the intrathecal OAV101 IT, the intravenous formulation hasn’t had the smoothest of rides. In October 2019, the FDA implemented a partial clinical hold on OAV101 IT, after results of preclinical animal study showed dorsal root ganglia mononuclear cell inflammation, sometimes accompanied by neuronal cell body degeneration or loss.
The hold was lifted in August 2021, following data from Novartis’ nonclinical toxicology study in non-human primates, which addressed questions around dorsal root ganglia (DRG) injury after injection.
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