82% of SMA kids treated pre-symptomatically with Zolgensma hit developmental milestones

 Swiss Pharma giant Novartis (NOVN: VX) has announced positive new data about Zolgensma (onasemnogene abeparvovec), an essential, one-time treatment and the only gene therapy for spinal muscular atrophy (SMA), from the completed two-copy cohort of the Phase III SPR1NT clinical trial that demonstrates age-appropriate development when used pre-symptomatically and rapid, clinically meaningful efficacy in symptomatic children, even those with severe SMA at baseline.

Highlights of the trial results

The data will be presented at the European Academy for Neurology (EAN) Virtual Congress 2021 (June 19–22), but the main findings are as follows:

• 100% of children (14) treated pre-symptomatically in the SPR1NT two-copy cohort survived without respiratory or nutritional support, and sat independently for ≥30 seconds, including 79% (11/14) within the WHO window of expected normal development;
• 82% treated in STR1VE-EU achieved developmental motor milestones not observed in the natural history of SMA Type 1, including patients with more severe disease;
• 79% (11/14) could stand independently, seven of whom achieved this milestone within the WHO window of normal development;
• 100% of patients (14/14) met the secondary endpoint of survival without ventilatory support of any kind at 14 months of age, versus only 26% of patients in the Pediatric Neuromuscular Clinical Research (PNCR) natural history cohort; and
• More than 1,200 patients have now been treated with Zolgensma globally across clinical trials, managed access programs, and in the commercial setting.

SPR1NT trial results ‘nothing short of extraordinary’

“With more than 1,200 children now treated, these data presented at EAN further reinforce the life-changing benefit of a one-time treatment of Zolgensma,” said Dr Shephard Mpofu, senior vice president, chief medical officer, Novartis Gene Therapies.

 “When treated with Zolgensma prior to the onset of symptoms, not only did all patients survive, but were thriving - breathing and eating on their own and sitting independently, with many standing and walking. When you consider these newborns would go on to develop severe symptoms of SMA Type 1, a devastating, progressive disease that robs children of the ability to talk, eat, sit up and even breathe, findings from the SPR1NT trial are nothing short of extraordinary,” Dr Mpofu opined.

‘Most expensive drug in the world’

Zolgensma, which gained conditional approval in Europe in May 2020 and in the USA a year earlier, has a reported list price of £1.8 million ($2.5 million) per dose, and is labelled the most expensive drug in the world. The other treatment available to treat children with SMA is Biogen’s (Nasdaq: BIIB) much older drug Spinraza (nusinersen), but Novartis’ drug is thought to be more cost-effective than Spinraza over the long term. Also available is Roche’s (ROG: SIX) Evrysdi (risdiplam). Zolgensma is forecast to generate global sales of $2.5 billion by 2025 and lead the spinal muscular atrophy (SMA) market, according to data and analytics company GlobalData.

https://www.thepharmaletter.com/article/82-of-sma-children-treated-pre-symptomatically-with-zolgensma-achieved-developmental-motor-milestones