PhuturdexDrug Pipeline Intelligence

Phuturdex › Diseases › Amyloidosis

Drugs in development · updated 30 October 2026

Amyloidosis: drugs in development

Rare diseasePrevalence 1-9 / 100 000 according to Orphanet · ORPHA:69

As of 30 October 2026 there are 28 active clinical trials (recruiting or ongoing, phases I–III) on 24 drugs for amyloidosis. 13 are experimental, that is not yet approved in the United States.

28Active trials
13Experimental drugs
7Experimental, 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.

Sign in free →

Experimental drugs

Not yet approved by the US FDA for any indication.

DrugMost advanced phaseActive trialsReference trial
ALXN2220III2NCT06183931
EtentamigABBV-383, TNB-383BIII2NCT07793422
NucresiranIII2NCT07052903
[68Ga]CBP8III1NCT05619302
NTLA-2001III1NCT06128629
NXC-201III1NCT07709715
SCTC21CIII1NCT07388602
AZD0120I–II1NCT07081646
FKC289I–II1NCT07765524
124I AT-01I1NCT06974877
GC012FI1NCT07250269
JNJ-79635322I1NCT05652335
Technetiumtechnetium-99mI1NCT05951816

Sorted by most advanced phase and number of active trials.

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
DexamethasoneDexametasone, DextenzaIII7NCT07901725
BortezomibVelcade, Bortezomib Hospira®III5NCT07901725
CyclophosphamideCyclophosphamid, CytoxanIII5NCT07901725
DaratumumabDarzalexIII4NCT07901725
VutrisiranAmvuttra®III3NCT06679946
AcoramidisAttruby®, Beyonttra®III2NCT07116473
EplontersenWainua (Autoinjector)®, Wainua®III1NCT05667493
LinvoseltamabLynozyfic®III1NCT07901725
Belantamab mafodotinGSK2857916, Blenrep®I–II2NCT05145816
VenetoclaxVentoclax, Venclexta®II2NCT06629818
PegfilgrastimArmlupeg®, Stimufend®I1NCT02909036

Sorted by most advanced phase and number of active trials.

All 24 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.

Open in Phuturdex →

Specific forms

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: D000686 “Amyloidosis”. Prevalence: Orphanet.

ItalianoFrançaisDeutsch