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Novartis has provided an update on two Long-Term Follow-Up studies of Zolgensma, their gene therapy for SMA. Presented at the 2023 Muscular Dystrophy Association Conference, the data reveal that children treated with Zolgensma maintained or achieved additional milestones up to 7.5 years after administration of the drug.

The two Long-Term Follow-Up studies are called LT-001 and LT-002, and are designed to assess the impact of Zolgensma for up to 15 years after single injections of the treatment. Given the time scales involved in obtaining and analysing the trial results, the presented information is accurate as of the 23rd May 2022.

Results from both studies show the continued effectiveness and durability of Zolgensma, which continues to display a favourable benefit-to-risk profile.

Patients who completed the Phase 1 START study of Zolgensma have been enrolled in LT-001. All of the participants received an intravenous (into the blood) injection of Zolgensma after the onset of SMA symptoms.

In contrast, LT-002 includes participants who received Zolgensma either before or after symptoms started, and includes people that received either intravenous or intrathecal (into the spinal fluid) doses of the therapy. These participants were originally enrolled in either a Phase 3 intravenous study of Zolgensma (i.e., STR1VE-US, STR1VE-EU, STR1VE-AP or SPR1NT) or the Phase 1 intrathecal study known as STRONG.

The intrathecal treatment approach is now being referred to as “OAV101 IT” or “OAV101”.

Importantly, people in both trials have all maintained previously achieved motor milestones. Furthermore, in LT-001, three additional participants (30%) also achieved the key milestone of “standing with assistance.” In LT-002, 48 out of 77 (62.3%) participants either achieved a new motor milestone or had already achieved all motor milestones in the study in which they were initially enrolled.

Together, these results show that Zolgensma continues to be effective, safe and durable in a range of people with SMA.