Disease Overview:
Thyroid eye disease (TED) is an autoimmune condition that leads to inflammation of the muscles and fatty tissues located behind the eyes. This swelling can result in the eyes protruding outward, often giving a "bulging" or "staring" appearance, and may also cause redness and puffiness in the eyes and eyelids.
Epidemiology Analysis (Current & Forecast)
TED is the most common cause of orbital inflammation and proptosis in adults. The estimated prevalence of TED is between 90 and 300/100,000 people globally.
Approved Drugs (Current SoC) - Sales & Forecast
Currently, Teprotumumab (Tepezza, Amgen) is the only FDA-approved drug for the treatment of thyroid eye disease.
Pipeline Analysis and Expected Approval Timelines
The TED pipeline includes several emerging therapies targeting different mechanisms of action, including anti-IGF-1R mAb, anti-FcRn mAb, anti-IL mAb, and other approaches.
Competitive Landscape and Market Positioning
Since Tepezza’s debut, the TED pipeline has rapidly evolved, with multiple next-generation biologics and novel mechanisms of action now in late-stage clinical development. These include subcutaneous anti-IGF-1R agents, FcRn antagonists, IL-6R inhibitors, and even oral small molecules, offering potentially better convenience, broader applicability, and reduced healthcare system burden.
1. Tepezza (Teprotumumab, Amgen)
- Mechanism: Anti-IGF-1R monoclonal antibody
- Administration: Intravenous (IV)
- Regulatory Status:
- U.S.: Approved since 2020 (originally developed by Horizon Therapeutics, now under Amgen)
- EU: CHMP positive opinion received (April 2025) – Full EMA approval expected soon
- Market Position:
- First and only approved treatment for TED (U.S.)
- Demonstrated strong efficacy in reducing proptosis and inflammation
- Challenges:
- IV delivery requires hospital-based infusion, reducing convenience
- Side effects, notably hearing impairment, have raised some concerns
- Faces price sensitivity issues and payer pressure due to high cost
- Positioning: Gold standard but at risk from more convenient and potentially safer next-gen alternatives
2. Viridian Therapeutics Portfolio
a. Veligrotug (VRDN-001)
- Mechanism: Anti-IGF-1R mAb (same target as Tepezza)
- Route: IV
- Status:
- Phase III
- FDA Breakthrough Therapy Designation (May 2025) – eligible for priority review
- BLA submission expected in H2 2025
- EU submission in H1 2026
- U.S. launch (if approved): H2 2026
- Differentiators:
- Potentially better safety/tolerability profile
- Faster onset and shorter duration of treatment than Tepezza, based on early data
- Positioning: Direct competitor to Tepezza on efficacy; potential to take share based on safety, dosing, and pricing flexibility
b. VRDN-003
- Mechanism: Anti-IGF-1R mAb
- Route: Subcutaneous (SC) – key innovation
- Status:
- Phase III; topline data expected H1 2026
- BLA filing expected end of 2026
- Differentiators:
- SC delivery enables outpatient/self-administration
- Designed for better patient adherence and convenience
- May allow for faster uptake in markets where infusion logistics are a barrier
- Positioning: Most patient-friendly anti-IGF-1R candidate; may significantly erode Tepezza's market if efficacy matches
3. Other Emerging Therapies – Novel Mechanisms
a. Batoclimab (Immunovant)
- Mechanism: Anti-FcRn mAb – reduces IgG recycling
- Route: SC
- Status: Phase III, topline data expected H2 2025
- Positioning:
- Targets autoantibody reduction — could provide disease-modifying benefit
- SC route improves convenience
- If successful, may be used in combination or as monotherapy in broader autoimmune management
- Key Differentiator: FcRn pathway – alternative to IGF-1R strategy
b. Efgartigimod (argenx)
- Mechanism: FcRn antagonist
- Route: SC
- Status: Phase III
- Positioning:
- Already approved in other autoimmune indications (e.g., gMG)
- Safety and familiarity could facilitate off-label interest or faster uptake upon approval
- Differentiator: Platform molecule – possible cross-indication bundling for payers
c. Enspryng (Satralizumab, Roche/Chugai)
- Mechanism: Anti-IL-6R mAb
- Route: SC
- Status: Phase III; filing expected 2026
- Positioning:
- IL-6 blockade provides anti-inflammatory effect, addressing TED from a cytokine perspective
- Proven molecule in NMOSD – safety already established
- Differentiator: Unique MoA within this space; potential for combination or second-line use
d. Linsitinib (Sling Therapeutics)
- Mechanism: Small molecule IGF-1R inhibitor (oral)
- Route: Oral
- Status: Phase II/III
- Confirmatory Phase III to start in 2025
- Phase II/III data released Jan 2025
- Positioning:
- Only oral candidate – huge advantage in patient experience
- Small molecule may allow for cost advantage and better access
- Risks:
- Needs to prove efficacy comparable to mAbs
- Tolerability and selectivity concerns for systemic oral IGF-1R blockade
Market Positioning Summary Table
| Product | Developer | MoA | Route | Launch Window | Positioning |
| Tepezza | Amgen | Anti-IGF-1R | IV | EU 2025 | First-to-market, efficacy leader, IV burden |
| Veligrotug | Viridian | Anti-IGF-1R | IV | 2026 (U.S.) | Tepezza challenger with improved safety/dosing |
| VRDN-003 | Viridian | Anti-IGF-1R | SC | 2026+ | Most patient-centric; key threat to Tepezza |
| Batoclimab | Immunovant | Anti-FcRn | SC | 2026 | MoA innovation, SC, autoimmune platform |
| Efgartigimod | Argenx | Anti-FcRn | SC | TBD | Platform familiarity, payer bundling potential |
| Enspryng | Roche | Anti-IL-6R | SC | 2026 | Unique anti-inflammatory angle |
| Linsitinib | Sling | IGF-1R inhibitor | Oral | 2027+ | Only oral option, cost/access differentiator |
Notably, innovation is also advancing in the form of gene therapy approaches. Kriya Therapeutics' KRIYA-586, an AAV-based gene therapy encoding an anti-IGF-1R antibody, is currently in preclinical development. Delivered via focal peribulbar injection, KRIYA-586 aims to provide a long-acting, potentially one-time treatment for TED.
In September 2024, Kriya announced positive preclinical data, highlighting the potential for gene therapy to disrupt the current biologics-dominated landscape.
Key Companies:
Target Opportunity Profile (TOP)
Target Opportunity Profile (TOP) helps to highlight what emerging therapies need to demonstrate to surpass approved treatments like Tepezza (teprotumumab) in Thyroid Eye Disease (TED).
Target Opportunity Profile (TOP) – Thyroid Eye Disease (TED)
| Parameter | Optimal Profile (Emerging Therapies Must Show) | Tepezza (Approved Benchmark) | Improvement Opportunity |
| Efficacy | ≥83% proptosis response, ≥53% diplopia resolution, durable ≥6–12 months | ~83% proptosis response, ~53% diplopia, relapse in some | Longer-lasting effects, better diplopia response |
| Onset of Action | Visible benefit within 2–4 weeks | Benefit is typically visible within 6 weeks | Faster symptom relief |
| Safety & Tolerability | No hearing loss, minimal hyperglycemia, no infusion reactions | Hearing loss (~10–15%), hyperglycemia, infusion AEs | Improved safety profile, broader patient eligibility |
| Mechanism of Action | Novel or dual pathway (e.g., IGF-1R + FcRn), applicable across TED stages | IGF-1R blockade | Broader or complementary immune modulation |
| Route of Administration | Subcutaneous or oral preferred | Intravenous (IV) | Less invasive, more convenient |
| Dosing Frequency | Weekly to monthly, ≤12 weeks per course, or single treatment (gene therapy) | Every 3 weeks for 8 doses | Reduced treatment burden |
| Durability of Response | ≥1 year remission post-treatment | Durable in some, relapse risk exists | Longer sustained remission, fewer re-treatments |
| Formulation | SC autoinjector, oral tablet, or focal injection; room-temperature stable | IV infusion; requires cold-chain logistics | Easier administration and storage |
| Patient Access | First-line or steroid-refractory; broad payer coverage | Approved in moderate-to-severe, active TED | Earlier line use and better affordability |
Key Differentiation Goals for Pipeline Therapies
- Match or exceed efficacy while improving safety and patient convenience
- Deliver cost savings with equal or better outcomes
- Offer simplified administration (e.g., SC, oral, gene therapy) to improve adherence and access
- Address relapse and long-term control, not just symptom suppression
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