Novartis set to launch new Zolgensma trial to expand use

pharmafile | August 3, 2021 | News story | Manufacturing and Production, Medical Communications, Research and Development, Sales and Marketing  

Novartis has announced that it will launch a new Phase III study to expand the use of Zolgensma – the world’s most expensive drug – following the lifting of an FDA mandated clinical trial halt.

The Swiss company will study the use of the drug for patients aged between 2 and 18 for the treatment of spinal muscular atrophy (SMA), it said on Tuesday.

The decision comes after the FDA said existing trials may proceed, thereby lifting the partial clinical trial hold imposed in October 2019, after Novartis supplied new data.

Shephard Mpofu, Chief Medical Officer at Novartis Gene Therapies, said: “We are very pleased that our comprehensive nonclinical data package has addressed all issues identified related to DRG toxicity and the FDA has reached the decision that we may proceed with our OAV-101 IT clinical trial programme and initiate the STEER trial.

“We believe that all patients diagnosed with SMA should be able to benefit from the transformative impact of gene therapy and we remain confident that investigational OAV-101 IT is a viable potential treatment path for older patients who often have ongoing unmet needs, and for whom a one-time treatment could be especially compelling.”

In March Novartis announced it was letting go of 400 staff and closing a US gene therapy location as sales of Zolgensma stalled last year. The sales have been reportedly hurt by blocked efforts to expand its use in older patients after safety concerns prompted Novartis to pause a trial in order to collect more data for the FDA.

The STEER trial will build upon previous studies that showed that treatment with Zolgensma (OAV-101 IT) led to significant increases in Hammersmith Functional Motor Scale-Expanded (HFMSE) scores and a clinically meaningful response in older patients between ≥2 years and <5 years old with SMA Type 2. Additionally, STEER will add to the clinical data and emerging real-world evidence for the use of gene therapy to treat SMA.

SMA is a rare, genetic neuromuscular disease caused by a lack of a functional SMN1 gene, resulting in the irreversible loss of motor neurons, affecting muscle functions, including breathing, swallowing, and basic movement. Left untreated, patients with SMA Type 2 are unable to walk and will require a wheelchair, and more than 30% will die by age 25.

Kenneth Hobby, President of Cure SMA, said: “We are very pleased to see that a plan has been reached from Novartis, the FDA and EMA working together to move this IT approach forward.

“This route of administration has the potential to open up access for older patients to all the benefits of gene therapy. We have seen the interest among our symptomatic patients and their families in gene therapy, and this study is an important step in understanding its potential to address unmet needs that remain in the SMA community.”

Kat Jenkins

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