SWITZERLAND – Novartis has announced that the U.S. Food and Drug Administration (FDA) has determined that Zolgensma clinical trials for spinal muscular atrophy (SMA) patients may proceed, thereby lifting the partial clinical trial hold initiated in October 2019.
The decision to lift the hold was based on data from Novartis’ comprehensive nonclinical toxicology study in non-human primates (NHP) that addressed all issues identified previously.
“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 program and initiate the STEER trial,” said Shephard Mpofu, M.D., SVP, Chief Medical Officer, Novartis Gene Therapies.
Novartis will launch a new phase three study to expand the use of Zolgensma, the world’s most expensive one-time therapy at US$2.1 million per patient treatment.
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 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.
Data shows that if left untreated, patients with SMA are unable to walk and will require a wheelchair, and more than 30% will die by age 25. One out of 10,000 children get affected by the disorder.
This disease results in death or the need for permanent ventilation by the age of two in 90 per cent of cases.
Last year, Novartis said it had received approval from US regulatory for Zolgensma. The Swiss pharmaceutical company said Zolgensma was a one-time treatment for SMA.
However, Novartis said in March it was laying off 400 people and closing a U.S. gene therapy location as sales momentum for Zolgensma stalled.
Zolgensma’s sales have been hurt, in part, 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 U.S. Food and Drug Administration.
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