Novartis to Initiate Pivotal Confirmatory Study of AVXS-101 Intrathecal Formulation in Older Patients with SMA

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

Click here ­to­ read full press release/ article | Ref: Novartis | Image: StraitTimes