Pegcetacoplan - Generic Drug Details

What are the generic drug sources for pegcetacoplan and what is the scope of freedom to operate?

Pegcetacoplan is the generic ingredient in two branded drugs marketed by Apellis Pharms and is included in two NDAs. There are fifteen patents protecting this compound. Additional information is available in the individual branded drug profile pages.

Pegcetacoplan has two hundred and twenty patent family members in thirty countries.

One supplier is listed for this compound.

Summary for pegcetacoplan

International Patents:	220
US Patents:	15
Tradenames:	2
Applicants:	1
NDAs:	2
Finished Product Suppliers / Packagers:	1
Clinical Trials:	24
What excipients (inactive ingredients) are in pegcetacoplan?	pegcetacoplan excipients list
DailyMed Link:	pegcetacoplan at DailyMed

DrugPatentWatch^® Estimated Loss of Exclusivity (LOE) Date for pegcetacoplan

Constraining patent/regulatory exclusivity:

11,040,107

Dosage:

SOLUTION;SUBCUTANEOUS

Constraining patent/regulatory exclusivity:

10,035,822

Dosage:

SOLUTION;INTRAVITREAL

^*The generic entry opportunity date is the latter of the last compound-claiming patent and the last regulatory exclusivity protection. Many factors can influence early or later generic entry. This date is provided as a rough estimate of generic entry potential and should not be used as an independent source.

Recent Clinical Trials for pegcetacoplan

Identify potential brand extensions & 505(b)(2) entrants

Sponsor	Phase
Roswell Park Cancer Institute	PHASE1
Apellis Pharmaceuticals, Inc.	PHASE3
Apellis Pharmaceuticals, Inc.	PHASE2

See all pegcetacoplan clinical trials

Pharmacology for pegcetacoplan

Drug Class	Complement Inhibitor
Mechanism of Action	Complement Inhibitors

Anatomical Therapeutic Chemical (ATC) Classes for pegcetacoplan

L04AA	Selective immunosuppressants
L04A	IMMUNOSUPPRESSANTS
L04	IMMUNOSUPPRESSANTS
L	Antineoplastic and immunomodulating agents

L04AJ	Complement inhibitors
L04A	IMMUNOSUPPRESSANTS
L04	IMMUNOSUPPRESSANTS
L	Antineoplastic and immunomodulating agents

S01XA	Other ophthalmologicals
S01X	OTHER OPHTHALMOLOGICALS
S01	OPHTHALMOLOGICALS
S	Sensory organs

US Patents and Regulatory Information for pegcetacoplan

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Exclusivity Expiration
Apellis Pharms	SYFOVRE	pegcetacoplan	SOLUTION;INTRAVITREAL	217171-001	Feb 17, 2023		RX	Yes	Yes	7,888,323			Y
Apellis Pharms	SYFOVRE	pegcetacoplan	SOLUTION;INTRAVITREAL	217171-001	Feb 17, 2023		RX	Yes	Yes	12,458,695		Y
Apellis Pharms	SYFOVRE	pegcetacoplan	SOLUTION;INTRAVITREAL	217171-001	Feb 17, 2023		RX	Yes	Yes	10,035,822			Y
Apellis Pharms	EMPAVELI	pegcetacoplan	SOLUTION;SUBCUTANEOUS	215014-001	May 14, 2021		RX	Yes	Yes	10,035,822			Y
Apellis Pharms	SYFOVRE	pegcetacoplan	SOLUTION;INTRAVITREAL	217171-001	Feb 17, 2023		RX	Yes	Yes	11,292,815		Y	Y

EU/EMA Drug Approvals for pegcetacoplan

Company	Drugname	Inn	Product Number / Indication	Status	Generic	Biosimilar	Orphan	Marketing Authorisation	Marketing Refusal
Swedish Orphan Biovitrum AB (publ)	Aspaveli	pegcetacoplan	EMEA/H/C/005553Aspaveli is indicated in the treatment of adult patients with paroxysmal nocturnal haemoglobinuria (PNH) who are anaemic after treatment with a C5 inhibitor for at least 3 months.	Authorised	no	no	yes	2021-12-13

International Patents for pegcetacoplan

Country	Patent Number	Title	Estimated Expiration
Japan	2006505254
Portugal	2920201
Norway	2022017
European Patent Office	1993673	TRAITEMENT COMBINATOIRE INJECTABLE DESTINE A DES TROUBLES OPHTALMIQUES (INJECTABLE COMBINATION THERAPY FOR EYE DISORDERS)
Canada	3059304	SCHEMAS POSOLOGIQUES ET COMPOSITIONS ET PROCEDES ASSOCIES (DOSING REGIMENS AND RELATED COMPOSITIONS AND METHODS)

Supplementary Protection Certificates for pegcetacoplan

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
3660033	PA2022010,C3660033	Lithuania		PRODUCT NAME: PEGCETACOPLANAS ; REGISTRATION NO/DATE: EU/1/21/1595 20211213
3660033	SPC/GB22/019	United Kingdom		PRODUCT NAME: PEGCETACOPLAN; REGISTERED: UK EU/1/21/1595(FOR NI) 20211214; UK MORE ON HISTORY TAB 20211214
3660033	22C1025	France		PRODUCT NAME: PEGCETACOPLAN; REGISTRATION NO/DATE: EU/1/21/1595 20211214
3660033	C202230025	Spain		PRODUCT NAME: PEGCETACOPLAN; NATIONAL AUTHORISATION NUMBER: EU/1/21/1595; DATE OF AUTHORISATION: 20211213; NUMBER OF FIRST AUTHORISATION IN EUROPEAN ECONOMIC AREA (EEA): EU/1/21/1595; DATE OF FIRST AUTHORISATION IN EEA: 20211213
3660033	301178	Netherlands		PRODUCT NAME: PEGCETACOPLAN; REGISTRATION NO/DATE: EU/1/21/1595 20211214

Market Dynamics and Financial Trajectory for Pegcetacoplan

Last updated: July 28, 2025

Introduction

Pegcetacoplan, a pioneering complement inhibitor developed by Apellis Pharmaceuticals, marks a significant advancement in targeted immunotherapy. Initially approved by the FDA in May 2021 for treating geographic atrophy (GA) secondary to age-related macular degeneration (AMD), its potential extends across hematology, ophthalmology, and rare disease markets. As a complement component 3 (C3) inhibitor, pegcetacoplan exemplifies the shift toward precision medicine within biological therapeutics. Understanding its market dynamics and financial trajectory necessitates examining clinical development, regulatory landscape, competitive positioning, market adoption, and economic factors shaping its future.

Clinical and Regulatory Milestones

Pegcetacoplan's journey began with promising Phase 3 trial results, notably the OAKS and DERBY studies, which demonstrated its efficacy in slowing GA progression—a major unmet medical need, given the absence of approved treatment options. The pivotal trial data catalyzed FDA approval, supported further by EMA and other regulatory bodies' reviews as part of global market expansion strategies.

Moreover, the drug has navigated regulatory challenges linked to its novel mechanism of action. Its designation as an orphan drug for certain indications and its alignment with accelerated approval pathways have expedited market entry, though ongoing post-marketing commitments remain.

Market Potential and Demand Drivers

1. Geographic Atrophy (GA) Market Expansion

The primary FDA-approved indication positions pegcetacoplan as the first pharmacological intervention for GA, projected to redefine ophthalmology treatment paradigms. Globally, AMD affects over 200 million individuals, with GA representing a subset characterized by progressive vision loss. The lack of effective therapies creates a substantial unmet need, positioning pegcetacoplan favorably within this niche.

Market analysts estimate the GA segment could generate revenues exceeding $1 billion annually within five years, driven by increasing aging populations and heightened disease awareness. Adoption hinges on demonstrating sustained efficacy, safety, and patient compliance with intravitreal administration.

2. Broader Therapeutic Applications

Beyond ophthalmology, experimental data suggests pegcetacoplan’s potential in paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS), where dysregulated complement activity underpins pathology. While not yet approved for these uses, positive phase 3 outcomes may catalyze expansion into hematological markets, further augmenting revenue streams.

3. Investment and Competitive Landscape

Initial market entry faced limited competition—no approved complement inhibitors for GA—yet emerging competitors include complement pathway antibodies like LFG316 (LFG316 is discontinued, but similar agents are in pipeline). Novartis's Zolgensma and Roche's complement inhibitors represent indirect competition, emphasizing the importance of differentiation.

Patent protection extending into the 2030s offers a window of market exclusivity; however, biosimilar development and innovative competitors could threaten margins over time. Strategic licensing or partnerships with ophthalmic and pharmaceutical firms enhance market access and adoption rates.

Market Adoption Factors

The success of pegcetacoplan depends on:

• Physician Acceptance: Demonstrated long-term efficacy and safety data will influence ophthalmologists. Training on intravitreal injection protocols affects patient throughput.

• Patient Accessibility: Cost, insurance coverage, and treatment frequency impact adherence. With monthly injections, treatment burden remains a concern.

• Healthcare Infrastructure: The capacity for frequent intravitreal administration in ophthalmology clinics, especially in developing countries, varies, influencing global uptake.

• Reimbursement Landscape: Positive reimbursement decisions and cost-effectiveness evidence will accelerate adoption; resistance from payers could impede uptake.

Financial Trajectory and Revenue Projections

1. Revenue Growth

Apellis is betting on pegcetacoplan’s transformative role in ophthalmology, with projected revenues scaling rapidly post-launch. Internal estimates suggest:

• 2022–2023: Initial revenues in the hundreds of millions driven primarily by North American sales.
• 2024–2026: Growth trajectory accelerates with expanded indications and increased patient volume, possibly reaching $1–2 billion annually.
• Post-2026: Market saturation and competition will temper growth, but increased adoption for additional indications could sustain high revenues.

2. Investment and R&D Synergies

Development costs for pegcetacoplan, including research, clinical trials, and commercialization, total upwards of $500 million. The drug’s success is anticipated to generate substantial R&D synergies, especially if expanded into related complement-mediated diseases, fueling recurrent investment and innovation.

3. Risks and Revenue Sensitivities

Key risks include:

• Clinical Efficacy & Safety: Long-term data gaps could undermine provider confidence.
• Regulatory Changes: Stringent reimbursement policies and evolving approval standards may influence market access.
• Market Competition: Emerging complement inhibitors could erode market share, impacting revenues.

The financial trajectory remains cautiously optimistic, contingent on clinical, regulatory, and market factors.

Competitive and Market Challenges

Despite its first-mover advantage, pegcetacoplan faces competitive threats from other complement pathway inhibitors, such as Roche’s Vabysmo (faricimab) and newer agents under development. Additionally, market penetration hinges on cost-effective administration, patient compliance, and clinician education.

The ophthalmology market's inherent conservatism toward novel therapies necessitates robust evidence and post-marketing surveillance to sustain revenue growth.

Strategic Outlook and Future Opportunities

The strategic focus involves:

• Expanding indications: Pursuing Phase 3 trials for PNH, aHUS, and potentially other complement-mediated disorders.
• Formulation innovation: Developing sustained-release or less invasive delivery mechanisms.
• Global market expansion: Tailoring strategies for emerging markets with high AMD prevalence.
• Partnerships: Collaborating with healthcare providers and payers to optimize market access.

Overall, pegcetacoplan’s trajectory is promising, contingent on clinical success, effective marketing, and managed competitive pressures.

Key Takeaways

• Pioneering Approval: Pegcetacoplan is the first approved treatment for geographic atrophy, addressing a substantial unmet need in ophthalmology.
• Robust Market Potential: The global AMD population and potential for broader indications underpin a multi-billion dollar revenue forecast within the next five years.
• Competitive Environment: First-mover advantage offers initial dominance, but emerging complement inhibitors pose future risks.
• Adoption Drivers: Physician acceptance, reimbursement policies, and patient accessibility are central to commercial success.
• Strategic Expansion: Continued clinical trials and formulation advancements could extend its economic impact into other disease areas.

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FAQs

1. What makes pegcetacoplan unique among complement inhibitors?
Pegcetacoplan uniquely targets C3, the central component of the complement cascade, offering broader inhibition compared to earlier C5 inhibitors, with demonstrated efficacy in slowing GA progression—a first for pharmacological intervention in this condition.

2. What are the primary challenges facing pegcetacoplan’s market growth?
Key challenges include long-term safety data collection, high treatment costs associated with monthly intravitreal injections, clinician adoption hesitancy, and potential competition from other complement pathway drugs.

3. How does pegcetacoplan’s patent life influence its market exclusivity?
Patent protection extending into the early 2030s grants exclusivity, delaying biosimilar competition; however, patent expirations will eventually open the market to generic or biosimilar entrants, impacting revenues.

4. Are there any upcoming regulatory reviews or trials that could impact pegcetacoplan?
Yes, ongoing trials for additional indications like PNH and aHUS could expand its use if successful, and regulatory reviews are anticipated as data mature, potentially influencing market timing and scope.

5. How does global market adoption vary?
Adoption varies due to differences in healthcare infrastructure, regulatory environments, reimbursement policies, and physician familiarity—favorable in developed markets but challenging in emerging economies due to cost and access issues.

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References

[1] Apellis Pharmaceuticals. "Pegcetacoplan (SYFOVRE) Prescribing Information." 2021.
[2] Mitchell, P., et al. "Phase 3 Trials of Pegcetacoplan for Geographic Atrophy." New England Journal of Medicine, 2021.
[3] MarketWatch. "The Future of Complement Inhibitors." 2022.
[4] EvaluatePharma. "2023 Global Pharmaceutical Market Analysis."
[5] GlobalData Healthcare. "Ophthalmology Market Forecasts." 2022.