Last Update: Aug 1, 2022
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 (latent/inactive) mAb | Type 2 and Type 3 SMA (non-ambulatory) as add-on to background SMN therapy (nusinersen or risdiplam). IV dose | III |
Biohaven Pharmaceuticals | Taldefgrobep Alfa (RG6206/BMS-986089)3 | Anti-myostatin (active) adnectin (Fc-fusion protein) | SMA (ambulatory and non-ambulatory) as adjunctive therapy to nusinersen or risdiplam. SC dose. | III |
Roche/Genentech | GYM329 (RO7204239)4 | Anti-myostatin (active) mAb | SMA age 2-10, ambulatory (in combination with Evrysdi). SC dose. | II/III |
Biogen | BIIB110 (ALG-801) | ActRIIA/B ligand trap | SMA | I |
Ractigen Therapeutics | RAG-06 | RNAa (small activating RNAs) | SMA | Preclin |
Biogen (Ionis) | BIIB1159 | Antisense oligonucleotide | SMA | Preclin |
Shift Pharmaceuticals | E1V1.11 | SMN2 antisense oligonucleotide | SMA | Preclin |
Reborna Biosciences | TEC-1 | SMN2 splicing modifier | SMA | Preclin |
Imago Pharmaceuticals | - | - | SMA | Preclin |
Discontinued/Failed | ||||
Novartis | LMI070 (branaplam)5 | SMN2 RNA splicing modulator | SMA | II |
Roche | Olexisome6 | Mitochondrial pore modulator | Type 2, 3 SMA | II |
Cytokinetics/Astellas | Reldesemtiv | FSTA (fast skeletal muscle troponin activator) | SMA | II7 |
Catalyst Pharmaceuticals | Amifampridine | Potassium channel blocker | SMA Type 3 (ambulant) | II8 |
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