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Tuberous Sclerosis Complex

Rare Diseases
9
Pipeline Programs
9
Companies
13
Clinical Trials
1
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
0
1
4
0
3
1
Early DiscoveryClinical DevelopmentMarket

Rare Diseases is a $21.9B market dominated by a single blockbuster (ELIQUIS), with moderate growth and high consolidation among top-tier pharma players.

$21.9B marketMature→ Stable30 products15 companies

Key Trends

  • Market concentration: One product (ELIQUIS) represents 83% of market spending
  • Complement inhibitors and enzyme replacement therapies gaining momentum
  • Active pipeline with 1,790 trials, predominantly Phase 2-3 stage

Career Verdict

Yes, if you seek stability and commercial opportunity with major pharma; proceed cautiously if you want pipeline upside or innovation-driven roles.

AI-generated market analysis based on FDA, CMS, ClinicalTrials.gov, and hiring data

Market Leaders

#1ELIQUISStable
$18.3B
Bristol Myers Squibb·LAUNCH15.0yr
#2XIFAXANDeclining
$1.1B
Bausch Health·LOE_APPROACHING
#3BRILINTAStable
$692M
AstraZeneca·PEAK10.2yr
#4TYVASO DPIStable
$437M
#5STRENSIQStable
$259M

Drug Class Breakdown

FXa Inhibitors (Anticoagulants)
$18.3B(83%)

Mature, patent-protected until 2041

Unknown Mechanism
$1.4B(6%)

Mixed—includes LOE-approaching products

P2Y12 Receptor Antagonists
$692M(3%)

Peak sales, LOE 2036

Prostaglandins/Vasodilators
$437M(2%)

Stable, long patent protection

Complement Inhibitors
$408M(2%)

Emerging class, no LOE data available

Enzyme Replacement Therapies
$40M(0%)

Niche market, specialized patient populations

Career Outlook

Stable

The rare disease market is stable but mature, with job growth concentrated in commercial and medical affairs rather than R&D innovation. Consolidation among top pharma (AstraZeneca, BMS, Amgen) limits opportunities for mid-tier companies, but specialist biotech firms show hiring momentum. Patent cliff risk for ELIQUIS (2041) will create restructuring opportunities and potential job flux in downstream years.

Breaking In

Target commercial or quality assurance roles at AstraZeneca, Capricor, or Amgen to build market knowledge; rare disease expertise is portable and valued across therapeutic areas.

For Experienced Professionals

Seek regulatory or medical affairs leadership roles where salary ceiling is highest ($243K-$257K); consider biotech firms like Insmed or Krystal for upside if pipeline approvals accelerate.

In-Demand Skills

Market access and HCP engagement (medical affairs)Regulatory and compliance expertisePatient stratification and genetic testing knowledgeCommercial analytics and lifecycle management

Best For

Medical Science Liaison (high salary, growing demand)Commercial Manager/Product Manager (39% of jobs)Regulatory Affairs Specialist (premium salary $257K)Health Economics & Outcomes Research (unmet need in data)

Hiring Landscape

$114K-$257K

Rare disease hiring is concentrated in commercial roles (75 jobs at $215K avg) and medical affairs (16 jobs at $243K avg), reflecting a mature market's focus on market access and HCP engagement. Top hiring companies include AstraZeneca (38 jobs) and emerging biotech players like Capricor Therapeutics (30 jobs). Department salaries range from $114K-$257K, with regulatory affairs commanding premium compensation.

195
Open Roles
5
Companies Hiring
5
Departments

Top Hiring Companies

38Growing
18Stable
17Growing
14Stable

By Department

Commercial(39%)
$215K
Medical Affairs(8%)
$243K
Research & Development(10%)
Regulatory Affairs(4%)
$257K
Quality Assurance(4%)
$168K

Commercial and regulatory roles offer strong salaries and growth; R&D hiring is minimal (10%), reflecting a consolidation and maturity phase in the market.

On Market (1)

Approved therapies currently available

Marinus Pharmaceuticals
ZTALMYApproved
ganaxolone
Marinus Pharmaceuticals
Neuroactive Steroid Gamma-Aminobutyric Acid A Receptor Positive Modulator [EPC]oral2022
953K Part D

Competitive Landscape

9 companies ranked by most advanced pipeline stage

Marinus Pharmaceuticals
2 programs
2
1
GanaxolonePhase 31 trial
GanaxolonePhase 31 trial
Active Trials
NCT05323734Completed129Est. Oct 2024
NCT05604170Terminated117Est. Apr 2025
Sandoz
SandozAustria - Kundl
2 programs
1
1
everolimusPhase 31 trial
RAD001Phase 21 trial
Active Trials
NCT01289912Completed52Est. Dec 2014
NCT02962414Active Not Recruiting206Est. Aug 2027
Alliance Pharmaceuticals
2 programs
1
RAD001Phase 2
Early Biomarkers of Autism in Infants With Tuberous Sclerosis Complex (TSC)N/A1 trial
Active Trials
NCT01780441Completed166Est. Dec 2020
UNION therapeutics
UNION therapeuticsDenmark - Hellerup
1 program
1
AspirinPhase 21 trial
Active Trials
NCT03356769Unknown98Est. Nov 2021
Noema Pharma
Noema PharmaSwitzerland - Basel
1 program
1
Basimglurant with crossover to PlaceboPhase 21 trial
Active Trials
NCT05059327Completed61Est. Apr 2025
GRIN Therapeutics
GRIN TherapeuticsNY - New York
1 program
1
RadiprodilPhase 1/21 trial
Active Trials
NCT06392009Active Not Recruiting30Est. Jun 2028
Angeles Therapeutics
Angeles TherapeuticsCA - Los Angeles
2 programs
JASPERN/A1 trial
Potential EEG Biomarkers and Antiepileptogenic Strategies for Epilepsy in TSCN/A1 trial
Active Trials
NCT02687633Completed8Est. Aug 2017
NCT01767779Completed40Est. Dec 2018
Jazz Pharmaceuticals
Jazz PharmaceuticalsCA - Palo Alto
2 programs
GWP42003-PPHASE_31 trial
GWP42003-PPHASE_31 trial
Active Trials
NCT02544763Completed224Est. Feb 2019
NCT02544750Completed199Est. Jun 2021
Genomics
GenomicsUK - Oxford
1 program
Studies in Patients With Tuberous Sclerosis ComplexN/A1 trial
Active Trials
NCT03276195Completed32Est. Nov 2023

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
Marinus PharmaceuticalsGanaxolone
Marinus PharmaceuticalsGanaxolone
Sandozeverolimus
Jazz PharmaceuticalsGWP42003-P
Jazz PharmaceuticalsGWP42003-P
Noema PharmaBasimglurant with crossover to Placebo
UNION therapeuticsAspirin
SandozRAD001
GRIN TherapeuticsRadiprodil
GenomicsStudies in Patients With Tuberous Sclerosis Complex
Angeles TherapeuticsJASPER
Alliance PharmaceuticalsEarly Biomarkers of Autism in Infants With Tuberous Sclerosis Complex (TSC)
Angeles TherapeuticsPotential EEG Biomarkers and Antiepileptogenic Strategies for Epilepsy in TSC

Clinical Trials (13)

Total enrollment: 1,362 patients across 13 trials

Open-label Study of Adjunctive GNX Treatment in Children and Adults With TSC-related Epilepsy

Start: May 2022Est. completion: Apr 2025117 patients
Phase 3Terminated

Adjunctive GNX Treatment Compared With Placebo in Children and Adults With TSC-related Epilepsy

Start: Apr 2022Est. completion: Oct 2024129 patients
Phase 3Completed

Roll-over Study to Collect and Assess Long-term Safety of Everolimus in Patients With TSC and Refractory Seizures Who Have Completed the EXIST-3 Study [CRAD001M2304] and Who Are Benefitting From Continued Treatment

Start: Jun 2017Est. completion: Aug 2027206 patients
Phase 3Active Not Recruiting

An Open-label Extension Trial of Cannabidiol (GWP42003-P, CBD) for Seizures in Tuberous Sclerosis Complex (GWPCARE6)

Start: Aug 2016Est. completion: Jun 2021199 patients
Phase 3Completed

A Randomized Controlled Trial of Cannabidiol (GWP42003-P, CBD) for Seizures in Tuberous Sclerosis Complex (GWPCARE6)

Start: Apr 2016Est. completion: Feb 2019224 patients
Phase 3Completed
NCT05059327Noema PharmaBasimglurant with crossover to Placebo

Basimglurant (NOE-101) in Children, Adolescents, and Young Adults With TSC

Start: Mar 2022Est. completion: Apr 202561 patients
Phase 2Completed

Aspirin as an add-on Treatment of Refractory Epilepsy in Tuberous Sclerosis Complex

Start: Nov 2017Est. completion: Nov 202198 patients
Phase 2Unknown

Trial of RAD001 and Neurocognition in Tuberous Sclerosis Complex (TSC)

Start: Jan 2011Est. completion: Dec 201452 patients
Phase 2Completed

Astroscape: A Study of Radiprodil on Safety, Tolerability, Pharmacokinetics, and Effect on Seizures and Behavioral Symptoms in Patients With TSC or FCD Type II

Start: Jul 2024Est. completion: Jun 202830 patients
Phase 1/2Active Not Recruiting
NCT03276195GenomicsStudies in Patients With Tuberous Sclerosis Complex

Studies in Patients With Tuberous Sclerosis Complex

Start: May 2016Est. completion: Nov 202332 patients
N/ACompleted

Early Behavioral Intervention to Improve Social Communication Function in Infants With Tuberous Sclerosis Complex

Start: Sep 2015Est. completion: Aug 20178 patients
N/ACompleted
NCT01780441Alliance PharmaceuticalsEarly Biomarkers of Autism in Infants With Tuberous Sclerosis Complex (TSC)

Early Biomarkers of Autism in Infants With Tuberous Sclerosis Complex (TSC)

Start: Jan 2013Est. completion: Dec 2020166 patients
N/ACompleted
NCT01767779Angeles TherapeuticsPotential EEG Biomarkers and Antiepileptogenic Strategies for Epilepsy in TSC

Potential EEG Biomarkers and Antiepileptogenic Strategies for Epilepsy in TSC

Start: Sep 2012Est. completion: Dec 201840 patients
N/ACompleted

Related Jobs in Rare Diseases

Phase Legend

PreclinicalLab & animal studies
Phase 1Safety & dosing
Phase 2Efficacy testing
Phase 3Large-scale trials
On MarketApproved & available

Key Insights

3 late-stage (Phase 3) programs, potential near-term approvals
9 companies competing in this space

The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.