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On August 3, Novartis announced that the United States Food and Drug Administration (FDA) had lifted a partial clinical suspension of OAV-101, an intrathecal (IT) formulation of Zolgensma (onasemnogene abeparvovec). An intravenous (IV) formulation of Zolgensma was approved by the FDA in May 2019 to treat infants from newborn to two years old with spinal muscular atrophy (SMA). It has since been approved in several other markets, including the European Union (EU) and Japan last year.
The decision to lift the suspension of the IT formulation, which was implemented in October 2019, was based on non-clinical data from a toxicology study in non-human primates that addressed safety concerns related to the risk of injury. of the dorsal root ganglia after computer administration. . Novartis will now be able to initiate a pivotal phase III, randomized, double-blind, sham-controlled (STEER) trial to assess the efficacy, safety and tolerability of a single dose of OAV-101 in treatment-naïve patients with SMA Type II aged 2 to 18 years who are able to sit up but have never walked. The computer formulation would allow patients over two years of age to be treated with Zolgensma and would likely more than double the ADS patient population targeted by Novartis, which would have a huge impact on the drug’s potential revenue.
Zolgensma is currently the most expensive drug available, with a price tag of $ 2.1 million. This is an obstacle to the adoption of Zolgensma, as it has led to problems with reimbursement and, consequently, its availability for patients. It will be crucial that Zolgensma can demonstrate significant efficacy in the STEER trial to show that its long-term cost-effectiveness as a single gene therapy is favorable over its competitors, Spinraza from Biogen (nusinersen) and Evrysdi from Roche (risdiplam) . In a previous Phase I / II study, STRONG, patients treated with OAV-101 IT saw significant increases in Hammersmith Extended Functional Motor Scale (HFMSE) scores, so the Phase III trial is promising.
The SMA market is increasingly competitive, with three products on the market. As such, the clinical suspension imposed by the FDA has not only delayed the development of Zolgensma by Novartis, it has also enabled its competitors, Spinraza and Evrysdi, to gain traction in the elderly population of patients with ADS. . Spinraza was the first to come to market, having been approved in 2016 by the FDA, and is currently positioned as the market leader, with reported global sales of $ 2.05 billion last year. However, spinraza represents a chronic treatment option for patients with AS, unlike Zolgensma.
Evrysdi is expected to be a strong competitor in the SMA market, with its oral route of administration allowing convenient home dosing. This would give it a considerable advantage over Spinraza and Zolgensma, which are given intrathecally via an invasive lumbar puncture requiring hospitalization. GlobalData predicts that Evrysdi will generate $ 2 billion worldwide by 2026.
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