Please login to the form below

Not currently logged in

Novartis to proceed with phase 3 trial for intrathecal OAV-101 in older SMA patients

FDA lifts trial hold following nonclinical toxicology study


The US Food and Drug Administration (FDA) will permit clinical trials for Novartis’ OAV-101 intrathecal (IT) for spinal muscular atrophy (SMA) patients to proceed, following data from a nonclinical toxicology study in non-human primates (NHP) that addressed a number of issues, including questions of dorsal root ganglia (DRG) injury following IT administration.

This decision lifts the partial clinical trial hold which was implemented in October 2019.

Novartis will now go ahead with STEER, a global phase 3 study that will analyse the clinical efficacy, safety and tolerability of OAV-101 IT in treatment naïve patients between two and 18 years of age, who are able to sit but have never walked.

STEER will provide new clinical data on the use of gene therapy to treat SMA, building upon the phase 1/2 STRONG study, which showed that patients treated with OAV-101 IT saw significant increases in Hammersmith Functional Motor Scale-Expanded (HFMSE) scores.

Shephard Mpofu, SVP, chief medical officer, Novartis Gene Therapies, commented: “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.”

Kenneth Hobby, President, Cure SMA, said: “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.”

Earlier this year, Novartis Gene Therapies and NHS England reached a ‘landmark’ deal for Zolgensma gene therapy for patients with spinal muscular atrophy (SMA) and in June the NHS treated its first patient with Zolgensma.

Article by
Bryony Andrews

4th August 2021

From: Regulatory, Healthcare



COVID-19 Updates and Daily News

Featured jobs


Add my company
Aurora Healthcare Communications

We're a leading healthcare strategic communications agency helping pharmaceutical companies adapt fast to improve the lives of patients and healthcare...

Latest intelligence

Human behaviour - Sept 21
All change – how untangling human behaviour can encourage better health
Driving better patient outcomes through clear, achievable practical steps that are underpinned by transparent evidence...
Health literacy in the time of COVID-19
In a time when much of the media’s focus is on the ongoing COVID-19 pandemic and the differences in vaccination rates between various regions, countries, and socioeconomic groups, improving health...
Rare thoughts & outcomes - navigating pathways to better outcomes in rare