Spinal Muscular Atrophy (SMA) Approved Therapies & Global Pipeline

Last Update: Aug 6, 2023

Click to purchase the complete database for corresponding clinical trial identifiers, clinical trial parameters, discontinued programs, notes on Spinraza, Zolgensma, Evrysdia (e.g exclusivity, patents, key clinical data), reported net product sales of Spinraza, Zolgensma and Evrysdi every quarter from Q4 2016 up to Q3 2022 (including additional market data), a highlight of apitegromab, including patents and Ph2 clinical trial data summary.

Approved Therapies

Company Candidate MOA Indications Administration/Dose Approval
Roche (Genentech), PTC TherapeuticsEvrysdi (risdiplam)SMN2 splicing modifierTreatment of SMA (Type 1, 2, 3) in patients aged 2 mth and olderOral QD 0.15-5 mg/kg based on age/weightAug 2020 (US), Mar 2021 (EU)
BiogenSpinraza (nusinersen)Antisense oligonucleotideTreatment of SMA (Type 1, 2, 3) in pediatric and adult patientsIntrathecal injection 12 mg Q2W first 3 doses, 4th dose after 30 days, then maintenance dose Q4MDec 2016 (US), Jun 2017 (EU)
NovartisZolgensma (onasemnogene abeparvovec-xioi)scAAV9 containing SMN1 gene under CAG promoterSMA in patients less than 2 years of ageSingle-dose IV infusion 1.1E14 vg/kgMay 2019 (US), May 2020 (EU)

Global Pipeline

Company Candidate MOA Indications Administration/Dose Phase
Scholar RockApitegromab (SRK-015)Anti-myostatin (latent) IgG4 mAbType 2/3 SMA on background nusinersen or risdiplam therapyIV Q4W 10 or 20 mg/kgIII
Novartis/AvexisOAV101 (AVXS-101, Zolgensma)SMN gene replacement therapyType 2 SMASingle dose intrathecal 1.2E14 vgIII
Biohaven PharmaceuticalsTaldefgrobep alfa (RG6206/BMS-986089)Anti-myostatin (active) adnectin IgG1-Fc fusion proteinSMA ambulant and non-ambulant, ages 4-21SC injection 35mg/50mg weekly (weight-based)III
Roche/GenentechRO7204239 (GYM329)Anti-myostatin (latent) IgG1 mAbSMA age 2-10, ambulant (in combination with Evrysdi), FSHDSC abdominal Q4W w/ oral once daily risdiplamII/III
NMD PharmaNMD670ClC-1 inhibitorPotentially SMA, myasthenia gravisOralI/II
Epirium BioMF-30015-PGDH inhibitorSMAOralPreclin
Ractigen TherapeuticsRAG-06RNAa (small activating RNAs)SMA-Preclin
Shift PharmaceuticalsE1V1.11 Antisense oligonucleotide (PMO blocking E1)SMA-Preclin
Biogen (Ionis)BIIB115 (ION306)Antisense oligonucleotideSMA-Preclin
Reborna BiosciencesTEC-1SMN2 splicing modifierNeuromuscular disease-Disc
AurimMed Pharma--SMA-Disc
Neurotune AGNT 1654 (Agrin)Agrin/neurotrypsin systemPotentially SMA-R&D