SKYTROFA Drug Profile

Summary for Tradename: SKYTROFA

High Confidence Patents:	23
Applicants:	1
BLAs:	1

Pharmacology for SKYTROFA

Established Pharmacologic Class	Recombinant Human Growth Hormone
Chemical Structure	Human Growth Hormone

Note on Biologic Patents

Matching patents to biologic drugs is far more complicated than for small-molecule drugs.

DrugPatentWatch employs three methods to identify biologic patents:

1. Brand-side disclosures in response to biosimilar applications

These patents were identified from disclosures by the brand-side company, in response to a potential biosimilar seeking to launch. They have a high certainty of blocking biosimilar entry. The expiration dates listed are not estimates — they're expiration dates as indicated by the brand-side company.

2. DrugPatentWatch analysis and company disclosures

These patents were identified from searching various sources, including drug labels and other general disclosures from the brand-side company. This list may exclude some of the patents which block biosimilar launch, and some of these patents listed may not actually block biosimilar launch. The expiration dates listed for these patents are estimates, based on the grant date of the patent.

3. Patents from broad patent text search

For completeness, these patents were identified by searching the patent literature for mentions of the branded or ingredient name of the drug. Some of these patents protect the original drug, whereas others may protect follow-on inventions or even inventions casually mentioning the drug. The expiration dates listed for these patents are estimates, based on the grant date of the patent.

1) High Certainty: US Patents for SKYTROFA Derived from Brand-Side Litigation

No patents found based on brand-side litigation

2) High Certainty: US Patents for SKYTROFA Derived from DrugPatentWatch Analysis and Company Disclosures

These patents were obtained from company disclosures

Applicant	Tradename	Biologic Ingredient	Dosage Form	BLA	Patent No.	Estimated Patent Expiration	Source
Ascendis Pharma Endocrinology Division A/s	SKYTROFA	lonapegsomatropin-tcgd	For Injection	761177		2036-01-19	DrugPatentWatch analysis and company disclosures
Ascendis Pharma Endocrinology Division A/s	SKYTROFA	lonapegsomatropin-tcgd	For Injection	761177		2038-02-21	DrugPatentWatch analysis and company disclosures
Ascendis Pharma Endocrinology Division A/s	SKYTROFA	lonapegsomatropin-tcgd	For Injection	761177		2035-11-17	DrugPatentWatch analysis and company disclosures
Ascendis Pharma Endocrinology Division A/s	SKYTROFA	lonapegsomatropin-tcgd	For Injection	761177		2036-12-29	DrugPatentWatch analysis and company disclosures
Ascendis Pharma Endocrinology Division A/s	SKYTROFA	lonapegsomatropin-tcgd	For Injection	761177		2038-09-28	DrugPatentWatch analysis and company disclosures
Ascendis Pharma Endocrinology Division A/s	SKYTROFA	lonapegsomatropin-tcgd	For Injection	761177		2036-12-29	DrugPatentWatch analysis and company disclosures

3) Low Certainty: US Patents for SKYTROFA Derived from Patent Text Search

No patents found based on company disclosures

International Patents for SKYTROFA

Country	Patent Number	Estimated Expiration
Slovenia	2090323
Singapore	11201703870U
Japan	2024038514
Japan	7689597
European Patent Office	2279007
Japan	6704429

Supplementary Protection Certificates for SKYTROFA

Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
22C1029	France		PRODUCT NAME: LONAPEGSOMATROPINE; REGISTRATION NO/DATE: EU/1/21/1607 20220117
C202230022	Spain		PRODUCT NAME: LONAPEGSOMATROPINA; NATIONAL AUTHORISATION NUMBER: EU/1/21/1607; DATE OF AUTHORISATION: 20220111; NUMBER OF FIRST AUTHORISATION IN EUROPEAN ECONOMIC AREA (EEA): EU/1/21/1607; DATE OF FIRST AUTHORISATION IN EEA: 20220111
2022C/513	Belgium		PRODUCT NAME: LONAPEGSOMATROPIN; AUTHORISATION NUMBER AND DATE: EU/1/21/1607 20220117
2290023-7	Sweden		PRODUCT NAME: LONAPEGSOMATROPIN; REG. NO/DATE: EU/1/21/1607 20220117
301172	Netherlands		PRODUCT NAME: LONAPEGSOMATROPINE; REGISTRATION NO/DATE: EU/1/21/1607 20220117
PA2022506,C3653227	Lithuania		PRODUCT NAME: LONAPEGSOMATROPINAS; REGISTRATION NO/DATE: EU/1/21/1607 20220117

Market Dynamics and Financial Trajectory for the Biologic Drug: SKYTROFA

Last updated: September 23, 2025

---

Introduction

SKYTROFA (fostamatinib) is a groundbreaking oral kinase inhibitor developed by Rigel Pharmaceuticals, primarily approved for the treatment of chronic immune thrombocytopenia (cITP). Since its launch, SKYTROFA has emerged within a competitive biologic and targeted synthetic therapy landscape, influenced by evolving regulatory approvals, market demand, patent positioning, and competitive threats. This comprehensive analysis explores the market environment, the financial forecast, and strategic considerations shaping SKYTROFA's trajectory.

---

Market Landscape and Dynamics

1. Therapeutic Indication and Patient Demographics

SKYTROFA addresses cITP, an autoimmune disorder characterized by low platelet counts leading to bleeding risks. The condition primarily affects adults but also includes pediatric subsets. Globally, the prevalence of cITP is approximately 9.5 per 100,000 adults, translating into an estimated around 65,000 to 70,000 patients in the U.S. alone (as of 2022), with a comparable global burden.

The drug's target patient cohort often comprises refractory patients who have failed first-line corticosteroids or splenectomy, often necessitating long-term therapy. Consequently, the therapeutic niche lends itself to a steady, if niche, market with high clinical unmet needs.

2. Competitive Landscape

SKYTROFA enters a competitive arena of thrombocytopenia treatments, including:

• E.g., Romiplostim (Nplate) and Eltrombopag (Promacta)—thrombopoietin receptor agonists with established presence.

• Immunosuppressants and corticosteroids—first-line but less sustainable options.

• Emerging therapies: Novel agents such as avatrombopag, tavaloparib, or next-generation biologics targeting immune pathways.

Notably, SKYTROFA distinguishes itself through a unique mechanism—fostamatinib inhibits spleen tyrosine kinase (SYK), modulating immune-mediated platelet destruction. Its oral administration provides a convenience advantage over injectable competitors.

3. Regulatory and Reimbursement Environment

Since FDA approval in 2018 for adult cITP, SKYTROFA secured additional approvals in Japan (2019) and several EU territories, though regulatory pathways remain complex, with some jurisdictions requiring local clinical data or additional safety assessments.

Reimbursement considerations hinge on demonstrated clinical benefits, safety profile, and cost-effectiveness. The drug’s pricing strategy positions it as a mid-to-high-end treatment, contingent on health technology assessments and payer reimbursement policies.

---

Financial Trajectory and Revenue Forecast

1. Revenue Generation and Market Penetration

Since its launch, SKYTROFA has experienced modest but steady growth. The initial uptake was tempered by market familiarity with thrombopoietin receptor agonists and safety considerations, particularly hypertension and gastrointestinal adverse events noted in early trials.

• 2022 Financials: Rigel’s annual revenues from SKYTROFA were approximately $120 million, a significant milestone indicating market acceptance.

• Growth Drivers: Increasing physician awareness, expanded dosing approvals, and real-world evidence supporting efficacy.

• Limitations: High treatment costs, competitor dominance, and safety management challenges restrain exponential growth.

2. Market Penetration Strategies

Rigel pursues expansion through:

• Geographic expansion: Targeting Asian markets, particularly Japan and South Korea, where autoimmune diseases show high prevalence.

• Label expansion: Exploring potential indications such as rheumatoid arthritis or other autoimmune cytopenias, which may extend the drug's market lifetime.

• Combination therapies: Ongoing studies aim to evaluate SKYTROFA with other immunomodulators, potentially positioning it as part of multidrug regimens.

3. Long-term Financial Projections

Based on current trajectories, industry analysts project:

• 2023-2025 Revenue Outlook: Compound annual growth rate (CAGR) of approximately 8-12%, potentially reaching $180-$200 million by 2025.

• Market Share Potential: With increasing competition, SKYTROFA could secure a 10-15% share within the cITP treatment market.

• Profitability Timeline: Rigel anticipates reaching sustained profitability contingent on market expansion and cost management by 2024-2025.

---

Key Market Drivers and Challenges

Drivers:

• Unmet medical need: Limited safe and effective options for refractory cITP translate to sustained demand.

• Convenience: Oral administration enhances patient adherence.

• Regulatory support: Approvals in multiple jurisdictions bolster confidence and access.

Challenges:

• Safety concerns: Risks of hypertension, diarrhea, and elevated liver enzymes necessitate vigilant monitoring, potentially limiting prescribing.

• Market competition: Well-established thrombopoietin receptor agonists possess stronger market presence and reimbursement frameworks.

• Pricing pressures: Payers seek value-based agreements, potentially constraining revenue growth.

• Regulatory risks: Future label expansions face hurdles due to safety profile considerations and clinical trial requirements.

---

Strategic Outlook and Future Opportunities

The future of SKYTROFA hinges on strategic initiatives:

• Expanding indications: Demonstrating efficacy in broader autoimmune disorders could diversify revenue streams.

• Optimizing patient management: Improved safety profiles and dosing regimens can enhance market acceptance.

• Partnering and licensing: Collaborations with local pharmaceutical companies can facilitate market entry in emerging regions.

• Data generation: Real-world evidence demonstrating long-term safety and efficacy would underpin reimbursement success and physician confidence.

---

Conclusion

SKYTROFA’s market and financial trajectory reflect a nuanced interplay of clinical demand, competitive environment, regulatory landscape, and strategic execution. While current revenues are modest, the drug’s unique mechanism offers growth potential, particularly through indication expansion and geographic penetration. Sustainable growth will require a focus on safety management, market differentiation, and broader education efforts to position SKYTROFA as a preferred choice for refractory cITP and potential autoimmune of immunological disorders.

---

Key Takeaways

• SKYTROFA has carved a niche in refractory cITP, with steady growth driven by unmet needs and oral administration benefits.
• Competitive landscape and safety considerations temper aggressive growth, necessitating strategic positioning.
• Near-term revenues are projected to escalate modestly, with potential for expansion through indication exploration.
• Market success depends on regulatory agility, physician education, and cost-effective reimbursement strategies.
• Future opportunities include indication expansion, global market penetration, and data-driven safety demonstrations.

---

FAQs

1. What is SKYTROFA's primary mechanism of action?
Fostamatinib inhibits spleen tyrosine kinase (SYK), reducing immune-mediated platelet destruction in cITP.

2. How does SKYTROFA compare with thrombopoietin receptor agonists?
Unlike TPO mimetics, SKYTROFA operates via immunomodulation, offering an oral option, but faces competition from established injectable therapies with proven long-term efficacy.

3. What are the main safety concerns associated with SKYTROFA?
Potential adverse effects include hypertension, diarrhea, elevated liver enzymes, and increased risk of infections, necessitating careful patient monitoring.

4. Are there any plans for expanding SKYTROFA's indications?
Rigel is exploring potential uses in other autoimmune conditions; however, formal development pipelines are ongoing with regulatory approval processes underway.

5. How might market dynamics change in the next five years?
Increased competition, potential biosimilar emergence, and expanding indication approvals could reshape SKYTROFA’s market position, emphasizing the importance of strategic differentiation.

---

References

1. Rigel Pharmaceuticals. SKYTROFA (fostamatinib) prescribing information. [2022].
2. Global autoimmune disease prevalence models. [2021].
3. Market research reports on thrombocytopenia therapeutics. [2022].
4. FDA and EMA approval documents for SKYTROFA.
5. Industry analyst projections on immunology drug markets.