Spinal Muscular Atrophy (SMA) Global Market & Pipeline

- Background of SMA (summary, prevalence)

- Approved treatments for SMA (Spinraza, Zolgensma, Evrysdi), summary information, patents & key clinical data

- Reported net product sales of approved products in SMA every quarter from Q4 2016 up to most recent reported quarter, including global total patients treated with Spinraza from Q3 2017 to Q1 2022 (broken down by US and ROW geographies from Q3 2017 to Q3 2019 as reported) and global total patients treated with Evrysdi from Q3 2021 to Q3 2022.

- Global pipeline of drugs in development for SMA, including discontinued programs (17 listings). Listed for each drug candidate is its mechanism of action, indication being trialled, route of administration and dosage, phase or stage of development, clinical trial identifier, date of clinical trial initiation, no. of trial participants, the primary outcomes of the trials, and additional notes related to the program and company.

- Highlight of apitegromab (SRK-015), including patents, Ph2 clinical trial data summary

Data as of Aug 6 2023.

Delivered as a Microsoft Excel .xlsx document.