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Phuturdex › Diseases › Amyotrophic lateral sclerosis

Drugs in development · updated 30 October 2026

Amyotrophic lateral sclerosis: drugs in development

Rare diseasePrevalence 1-9 / 100 000 according to Orphanet · ORPHA:803

As of 30 October 2026 there are 82 active clinical trials (recruiting or ongoing, phases I–III) on 79 drugs for amyotrophic lateral sclerosis. 60 are experimental, that is not yet approved in the United States.

82Active trials
60Experimental drugs
14Experimental, in phase III

Summary updated every week · data as of 30 October 2026. In Phuturdex you get the full detail of every drug and an email when an alert is published on the ones you follow.

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Experimental drugs

Not yet approved by the US FDA for any indication.

DrugMost advanced phaseActive trialsReference trial
MasitinibMasitinib AB Science®, Alsitek®III2NCT07174492
NUZ-001II–III2NCT04297683
PridopidineNurzigma®II–III2NCT04297683
Acetyl-l-carnitineII–III1NCT06126315
COYA 302II–III1NCT07161999
DNL343II–III1NCT04297683
FosigotifatorABBV-CLS-7262II–III1NCT04297683
GalunisertibLY2157299II–III1NCT07321860
IbudilastMN-166II–III1NCT04057898
ION363III1NCT04768972
Nerve Growth FactorII–III1NCT06391645
RAPA-501II–III1NCT04220190
TazbentetolII–III1NCT07325591
VerdiperstatII–III1NCT04297683
NL-CHCHD-001I–II4NCT06392126
FHND1002II2NCT07138014
PsilocybinPsilocybineII2NCT06782724
SNUG01I–II2NCT07169175
18F-OP-801I–II1NCT05395624
ALT001II1NCT07833618
AMT-162I–II1NCT06100276
BIIB147I–II1NCT07855211
DazucorilantII1NCT05407324
FasudilII1NCT05218668
Foralumab NasalII1NCT07688239
KamlanoflastI–II1NCT07396818
LTX-002I–II1NCT07660614
MTP-101CI–II1NCT07017946
NB-4746I–II1NCT07369076
NL-TARDB-002I–II1NCT07703462
NL-TARDB-006I–II1NCT07743268
NX210cII1NCT06365216
PLL001I–II1NCT06513546
RE104 for InjectionII1NCT07002034
TideglusibII1NCT05105958
TP04HN106I–II1NCT06726577
TRCN-1023I–II1NCT07674667
UsnoflastII1NCT07023835
VHB937II1NCT06643481
VTx-002I–II1NCT07287397

Sorted by most advanced phase and number of active trials. Showing 40 of 60 drugs.

Approved drugs in new trials

Approved by the US FDA for at least one indication, not necessarily this disease, and now in active trials on it.

DrugMost advanced phaseActive trialsReference trial
RiluzoleExservan®, Tiglutik Kit®III6NCT07174492
AmantadineGocovri, Osmolex Er®II–III1NCT04302870
MemantineNamenda, Memantine Mylan®II–III1NCT04302870
NerandomilastBI 1015550, Jascayd®II–III1NCT07321860
TacrolimusEnvarsus XR, Prograf®II–III1NCT04302870
TrazodoneRaldesy®, Oleptro®II–III1NCT04302870
ZilucoplanZilbrysq®II–III1NCT04297683
ColchicineColchicin, Lodoco®II1NCT07813858
Cromolyn SodiumSodium Cromoglicate, CromoglycateII1NCT07142291
CyclophosphamideCyclophosphamid, CytoxanII1NCT07834450
DarifenacinEnablex®, Emselex®II1NCT06249867
MetforminRiomet Er®, Glumetza®II1NCT04220021
N-AcetylcysteineAcetylcysteine, AcetadoteI–II1NCT07414212
RanolazineAspruzyo Sprinkle®, Ranexa®II1NCT06527222
TofersenQalsody®II1NCT07294144

Sorted by most advanced phase and number of active trials. Showing 15 of 19 drugs.

All 79 drugs, with the details

In Phuturdex every drug has all its trials, sponsors and countries, FDA and EMA approvals, orphan designations and safety alerts. Free until 31 March 2027.

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Broader categories

Source: Phuturdex analysis of ClinicalTrials.gov data (U.S. National Library of Medicine), updated 30 October 2026. Active trials = recruiting or ongoing, phases I–III, with at least one identified drug; phase and sites refer to the trials on this disease. Disease name: Wikidata; NLM MeSH term: D000690 “Amyotrophic Lateral Sclerosis”. Prevalence: Orphanet.

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