Panelists from the August 28th webinar included Dr. Peter Marks, M.D. Ph.D., Director, Center for Biologics Evaluation and Research (CBER) at FDA; Kenneth Hobby, President at Cure SMA; Mary Schroth, MD, Chief Medical Officer at Cure SMA; and Dave Lennon, President of AveXis, a Novartis Company.
Also available is a recording and presentation of the Zolgensma FDA approval webinar from June 2019.
Zolgensma, marketed by AveXis, a Novartis company, is an FDA-approved therapy to treat SMA. It is approved for patients with all forms and types of SMA who are under two years of age at the time of dosing.
Zolgensma is an SMN-enhancing therapy that works by replacing the missing or mutated SMN1 gene. Zolgensma is given via intravenous infusion. A virus, AAV9, carries the replacement gene into the body. This virus “infects” the cells with the new DNA. Zolgensma is a type of treatment that is often called gene therapy or gene replacement therapy.