ALS Drug Pipeline
Every ALS therapy currently approved or in clinical development.
What does this mean for me?
There are currently 3 FDA-approved drugs for ALS, with 12 more in Phase 3 trials. Below you can see what's in development, who each therapy is for, and where you may be able to access it.
This information is for educational purposes only. Talk to your neurologist about treatment options.
Showing 19 of 19 drugs.
Phase 1
1 drugs
Phase 2
1 drugs
Phase 3
12 drugs
FDA Review
0 drugs
No drugs match
Approved
3 drugs
Drug details
Riluzole — Rilutek (also Tiglutik, Exservan)
Sanofi · 1995
How it works
Reduces excess glutamate, a brain chemical that overstimulates and damages motor neurons.
Technical: Anti-glutamate
Delivery
Oral tablet / liquid / film
Who it's for
All ALS
Status
approved — FDA Approved — available to patients now.
First ALS drug approved. Slows progression by ~3 months. Generic available. Also marketed as Tiglutik (liquid, 2018) and Exservan (oral film, 2019).
Edaravone — Radicava
Mitsubishi Tanabe · 2017
How it works
Mops up harmful free radicals that damage motor neuron cells.
Technical: Antioxidant / free radical scavenger
Delivery
IV infusion or oral suspension
Who it's for
All ALS
Status
approved — FDA Approved — available to patients now.
Slowed functional decline by 33% in initial trial. Mixed results in subsequent studies. Oral form approved 2022.
Tofersen — Qalsody
Biogen / Ionis · 2023
How it works
Uses a small piece of genetic code to silence a faulty gene that produces toxic proteins in motor neurons.
Technical: Antisense oligonucleotide targeting SOD1
Delivery
Intrathecal injection
Who it's for
SOD1-ALS (~2% of patients)
Status
approved — FDA Approved — available to patients now.
First gene-targeted ALS therapy. Accelerated approval April 2023. Reduced plasma NfL by 55%. Confirmatory ATLAS trial in 14 countries, completion estimated 2026.
AMX0035 — Relyvrio
Amylyx · 2022
How it works
Combination drug aimed at reducing cellular stress and protecting motor neurons.
Technical: Sodium phenylbutyrate / taurursodiol
Delivery
Oral suspension
Who it's for
All ALS
Status
withdrawn — Previously available; pulled from market.
FDA approved September 2022. Relyvrio voluntarily withdrawn from market in 2024 after the Phase 3 PHOENIX trial failed to show efficacy.
Masitinib
AB Science
How it works
Calms down inflammation-driving cells around motor neurons.
Technical: Tyrosine kinase inhibitor (anti-inflammatory)
Delivery
Oral
Who it's for
All ALS
Status
recruiting — Currently enrolling patients in clinical trials.
Previous Phase 2/3 showed slowed progression. New Phase 3 validated by FDA and EMA, enrollment expected within 1 year.
CNM-Au8
Clene Nanomedicine
How it works
Boosts the energy supply (mitochondria) inside motor neurons.
Technical: Gold nanocrystal catalytic agent
Delivery
Oral
Who it's for
All ALS
Status
active — Trial ongoing; not currently enrolling new patients.
Targets mitochondrial function and energy metabolism. Also an active arm in the HEALEY platform trial.
Ulefnersen (ION363 / BIIB105)
Ionis Pharmaceuticals
How it works
Uses a small piece of genetic code to silence a faulty gene that produces toxic proteins in motor neurons.
Technical: Antisense oligonucleotide targeting FUS
Delivery
Intrathecal
Who it's for
FUS-ALS
Status
active — Trial ongoing; not currently enrolling new patients.
Gene-targeted therapy for FUS-ALS patients. Also referred to as Jacifusen.
Lithium Carbonate
TRICALS / UMC Utrecht
How it works
Mood stabilizer (precision medicine)
Technical: Mood stabilizer (precision medicine)
Delivery
Oral
Who it's for
UNC13A genotype-specific patients
Status
recruiting — Currently enrolling patients in clinical trials.
First precision-medicine platform trial in Europe. Run on the MAGNET adaptive platform.
Methylcobalamin
Eisai
How it works
High-dose vitamin B12 thought to support nerve health.
Technical: Ultra-high dose vitamin B12
Delivery
Intramuscular injection
Who it's for
All ALS
Status
active — Trial ongoing; not currently enrolling new patients.
Showed potential in Japanese trials.
Ibudilast (MN-166)
MediciNova
How it works
Anti-inflammatory; inhibits macrophage migration
Technical: Anti-inflammatory; inhibits macrophage migration
Delivery
Oral
Who it's for
All ALS
Status
active — Trial ongoing; not currently enrolling new patients.
Anti-neuroinflammatory candidate.
Verdiperstat
Biohaven
How it works
Blocks an enzyme (MPO) that drives harmful brain inflammation.
Technical: MPO inhibitor (targets neuroinflammation)
Delivery
Oral
Who it's for
All ALS
Status
active — Trial ongoing; not currently enrolling new patients.
Active arm in the HEALEY platform trial.
Pridopidine
Prilenia
How it works
Activates a receptor in nerve cells to support survival and reduce stress.
Technical: Sigma-1 receptor agonist (neuroprotective)
Delivery
Oral
Who it's for
All ALS
Status
active — Trial ongoing; not currently enrolling new patients.
Active arm in the HEALEY platform trial.
Reldesemtiv
Cytokinetics
How it works
Helps muscles respond more strongly to weakened nerve signals.
Technical: Fast skeletal muscle troponin activator
Delivery
Oral
Who it's for
All ALS
Status
active — Trial ongoing; not currently enrolling new patients.
Targets muscle function directly.
TPN-101
Transposon Therapeutics
How it works
Blocks rogue genetic elements that may trigger inflammation in motor neurons.
Technical: Reverse transcriptase inhibitor
Delivery
Oral
Who it's for
All ALS (esp. C9orf72)
Status
active — Trial ongoing; not currently enrolling new patients.
Targets retrotransposon activity.
AP-101
AL-S Pharma
How it works
Anti-misfolded SOD1 antibody
Technical: Anti-misfolded SOD1 antibody
Delivery
IV infusion
Who it's for
SOD1-ALS
Status
active — Trial ongoing; not currently enrolling new patients.
Targets toxic misfolded SOD1 protein.
WVE-004
Wave Life Sciences
How it works
Uses a small piece of genetic code to silence a faulty gene that produces toxic proteins in motor neurons.
Technical: Antisense oligonucleotide targeting C9orf72
Delivery
Intrathecal
Who it's for
C9orf72-ALS (~40% of familial ALS)
Status
active — Trial ongoing; not currently enrolling new patients.
For the most common genetic form of familial ALS.
BIIB078
Biogen
How it works
Uses a small piece of genetic code to silence a faulty gene that produces toxic proteins in motor neurons.
Technical: Antisense oligonucleotide targeting C9orf72
Delivery
Intrathecal
Who it's for
C9orf72-ALS
Status
discontinued — Development halted by sponsor.
Discontinued due to lack of efficacy in Phase 1 trial.
Jacifusen
Ionis
How it works
Uses a small piece of genetic code to silence a faulty gene that produces toxic proteins in motor neurons.
Technical: Antisense oligonucleotide targeting FUS
Delivery
Intrathecal
Who it's for
FUS-ALS
Status
active — Trial ongoing; not currently enrolling new patients.
Same compound as Ulefnersen above.
Zilucoplan
UCB Pharma
How it works
Blocks part of the immune system (the complement cascade) that may attack motor neurons.
Technical: Complement C5 inhibitor
Delivery
Subcutaneous injection
Who it's for
All ALS
Status
failed — Did not meet trial endpoints; development paused or stopped.
HEALEY platform arm — failed to meet endpoints.
Data Behind the Pipeline
Drug development depends on the open ALS research ecosystem. These public datasets power the trials, biomarkers, and discoveries shown above.
PRO-ACT
Where trial data comes from
The largest open-access pooled dataset of completed ALS clinical trials — 8,500+ patient records and 8M+ data points — used to design new trial protocols and benchmark drug efficacy.
Visit PRO-ACTTarget ALS Data Engine
Where biomarker discovery happens
Open biofluid, tissue, and multi-omics datasets from the Target ALS multicenter consortium — the foundation for identifying targets and biomarkers in next-generation ALS therapies.
Visit Target ALSExplore the full list of public ALS datasets on the Research Data portal.