Last Update: April 27, 2021
Approved therapies
| Company | Product | MoA | Indication | Approval |
|---|---|---|---|---|
| Biogen | Spinraza (nusinersen) | Antisense oligonucleotide to promote exon 7 inclusion in SMN2 mRNA | Treatment of SMA in pediatric and adult patients - intrathecal dose | Dec 2016(US), June 2017 (EU) |
| Novartis | Zolgensma (Onasemnogene abeparvovec-xioi) | AAV9 SMN1 gene replacement | Pediatric patients less than 2 yrs of age with SMA with bi-allelic mutations in SMN1 gene, regardless of type - single dose IV1 | May 2019 (US), May 2020 (EU) |
| Roche (Genentech)/PTC | Evrysdi (risdiplam) | SMN2 splicing modifier | Treatment of SMA age 2 mths+ - oral dose | Aug 2020 (US), Mar 2021 (EU) |
Global pipeline
| Company | Candidate | MoA | Indication | Phase |
|---|---|---|---|---|
| Scholar Rock | Apitegromab (SRK-015)2 | Anti-Myostatin mAb | SMA | II+ |
| Novartis | LMI070 (branaplam) | SMN2 RNA splicing modulator | SMA | II |
| Biogen | BIIB110 (ALG-801) | ActRIIA/B ligand trap | SMA | I |
| Ractigen Therapeutics | RAG-06 | RNAa (small activating RNAs) | SMA | IND-enabling |
| Shift Pharmaceuticals | E1V1.11 | SMN2 antisense oligonucleotide | SMA | Preclin |
| Reborna Biosciences | - | RNA-related | Neuromuscular disease (SMA) | Preclin |
| Imago Pharmaceuticals | - | - | SMA | Preclin |
| Discontinued/Failed | ||||
| Roche | Olexisome3 | Mitochondrial pore modulator | Type 2, 3 SMA | II |
| Cytokinetics/Astellas | Reldesemtiv | FSTA (fast skeletal muscle troponin activator) | SMA | II4 |
If you want to get notified when updates are available, please consider subscribing.