How many drugs treat Tuberous sclerosis syndrome?

Drug Landscape tracks 5 marketed treatments for Tuberous sclerosis syndrome (deduplicated by molecule). 0 Phase 3 candidates and 0 Phase 2 candidates are in the pipeline.

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Last reviewed 2026-06-10 · How we verify

Tuberous sclerosis syndrome — Treatment Landscape & Competitive Intelligence

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

Tuberous sclerosis syndrome (disease) competitive landscape: 5 marketed treatments tracked, 0 Phase 3 candidates, 0 Phase 2 candidates. Recent regulatory actions and upcoming PDUFA dates across the entire treatment set.

5 marketed 0 Phase 3 0 Phase 2 Live · 30-min refresh

Marketed treatment landscape

Approved drugs treating Tuberous sclerosis syndrome, deduplicated by molecule. See the full disease page for line-of-therapy detail.

Drug	Generic	Sponsor	Class	Target	First approval
Afinitor	everolimus	Novartis	Kinase Inhibitor [EPC]	Serine/threonine-protein kinase mTOR	2009-01-01
Rapamune	sirolimus	Pfizer	mTOR Inhibitor Immunosuppressant [EPC]	Serine/threonine-protein kinase mTOR	1999-01-01
Rapamune	Rapamune	Wyeth is now a wholly owned subsidiary of Pfizer		Peptidyl-prolyl cis-trans isomerase FKBP3, Sodium/nucleoside cotransporter 1, ATP-binding cassette sub-family G member 2
Afinitor	Afinitor	Central European Society for Anticancer Drug Research		Multidrug resistance protein 1, Peptidyl-prolyl cis-trans isomerase FKBP1A, Serine/threonine-protein kinase mTOR
Rapamycin	Rapamycin	Institut National de la Santé Et de la Recherche Médicale, France		Peptidyl-prolyl cis-trans isomerase FKBP3, Sodium/nucleoside cotransporter 1, ATP-binding cassette sub-family G member 2

Phase 3 pipeline

No Phase 3 pipeline candidates tracked.

Phase 2 pipeline

No Phase 2 pipeline candidates tracked.

Recent regulatory actions (last 90 days)

No regulatory actions in the last 90 days for this treatment set.

Upcoming PDUFA dates (next 180 days)

No PDUFA dates in the next 180 days for this treatment set.

Sponsor landscape

Central European Society for Anticancer Drug Research · 1 drug in Tuberous sclerosis syndrome
Institut National de la Santé Et de la Recherche Médicale, France · 1 drug in Tuberous sclerosis syndrome
Novartis · 1 drug in Tuberous sclerosis syndrome
Pfizer · 1 drug in Tuberous sclerosis syndrome
Wyeth is now a wholly owned subsidiary of Pfizer · 1 drug in Tuberous sclerosis syndrome

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Every new regulatory action, PDUFA date, or trial completion in Tuberous sclerosis syndrome:

Cite this brief

Drug Landscape (2026). Tuberous sclerosis syndrome — Treatment Landscape & Competitive Intelligence Brief. https://druglandscape.com/ci/disease/tuberous-sclerosis-syndrome. Accessed 2026-06-10.

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Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing