Novartis’ Zolgensma received FDA approval

Announcement

Novartis’ intrathecal form of spinal muscular atrophy gene therapy, Zolgensma, received FDA approval. With this approval, Novartis will expand access to this therapy, reaching patients aged 2 years and older. Hearing this advantage from this approval, the Stanford Medicine professor called it a game-changing advance for the healthcare sector. This will pave a new way for the treatments to be effective enough with this breakthrough, excellent spinal muscular atrophy gene therapy for the older patient population.

How is Itvisma connected to Zolgensma?

The Itvisma is also known as onasemnogene abeparvovec-brve or OAV101 IT. It’s an intrathecal form of Zolgensma, which received approval in May 2019 by the FDA as the first-ever gene therapy to enter the healthcare sector for spinal muscular atrophy (SMA), mainly for the child population under two years of age. However, the Itvisma is officially applicable for the treatment of not only two-year-old kids, but it also has a license to treat adults and teens with SMA with the proven mutation in the survival motor neuron 1 (SMN1) gene.

With this significant potential covering almost every age population, the Itvisma happens to be a ‘first and the only gene replacement therapy’ claims Novartis. These two formulations of Zolgensma identify the main genetic issue of SMA by delivering the functional copy of the SMN1 gene. This is effective as it's invisible in these patients.

The Novartis’ global development head of Gene Therapy and Neuroscience, Norman Putzki, said, “In the cases of infants and young kids, the STRENGTH (common cold, vomiting and fever) and STEER (an upper respiratory tract infection) have witnessed a transformative result. Though the most prevalent population of patients were mostly too old, so receiving treatment becomes heavy on their immune system.”

The FDA’s approval for Itvisma was regarding the data generated and presented from the Phase 3 STEER study and backed by the open-label, robust Phase 3b STRENGTH study. The studies highlighted statistically noteworthy balancing of the motor potential and improvements in motor function.

Norman further explained, “The intrathecal makes the gene therapy a trustworthy and safe therapy for heavier, older patient groups. Because of these patients, we have measurably vast exposure mainly towards the peak arm of the spectrum that might need a high dose. We can alleviate everything that is regarding dose toxicity by approaching IT with a compact flat dose into the cerebrospinal fluid.”