Shots:

• Novartis has received the FDA’s feedback following their review of data from the STRONG study of AVXS-101 (IT) in older patients with SMA. The FDA has recognized the potential of therapy and recommends a pivotal confirmatory study to augment the existing STRONG data- further supporting the regulatory submission for IT formulation
• The new study will not be initiated in the US until the FDA has lifted the hold while the request for a study is unrelated to the partial clinical hold on AVXS-101 IT. Trial design and other details are being evaluated and an update on the overall SMA program will be provided following the discussions with health authorities
• Zolgensma is an approved therapy in the US- Japan- and Brazil for patients with SMA under the age of two. The company is also evaluating branaplam (LMI070- PO- qw) RNA splicing modulator to expand the treatment options for SMA patients

­ Ref: Novartis | Image: Novartis