Pegcetacoplan - Generic Drug Details

What are the generic drug sources for pegcetacoplan and what is the scope of patent protection?

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

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

One supplier is listed for this compound.

Summary for Pegcetacoplan

International Patents:	223
US Patents:	16
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

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	EMPAVELI	pegcetacoplan	SOLUTION;SUBCUTANEOUS	215014-001	May 14, 2021		RX	Yes	Yes				Y
Apellis Pharms	EMPAVELI	pegcetacoplan	SOLUTION;SUBCUTANEOUS	215014-001	May 14, 2021		RX	Yes	Yes				Y
Apellis Pharms	EMPAVELI	pegcetacoplan	SOLUTION;SUBCUTANEOUS	215014-001	May 14, 2021		RX	Yes	Yes			Y
Apellis Pharms	EMPAVELI	pegcetacoplan	SOLUTION;SUBCUTANEOUS	215014-001	May 14, 2021		RX	Yes	Yes
Apellis Pharms	SYFOVRE	pegcetacoplan	SOLUTION;INTRAVITREAL	217171-001	Feb 17, 2023		RX	Yes	Yes
Apellis Pharms	EMPAVELI	pegcetacoplan	SOLUTION;SUBCUTANEOUS	215014-001	May 14, 2021		RX	Yes	Yes				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
Australia	2013344462
Australia	2018247243
Australia	2020260435
Australia	2023200929
Australia	2025201748
Brazil	112015011244

Supplementary Protection Certificates for Pegcetacoplan

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
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	202240020	Slovenia		PRODUCT NAME: PEGCETACOPLAN; NATIONAL AUTHORISATION NUMBER: EU/1/21/1595; DATE OF NATIONAL AUTHORISATION: 20211213; AUTHORITY FOR NATIONAL AUTHORISATION: EU
3660033	2022C/522	Belgium		PRODUCT NAME: PEGCETACOPLAN; AUTHORISATION NUMBER AND DATE: EU/1/21/1595 20211214
3660033	CA 2022 00023	Denmark		PRODUCT NAME: PEGCETACOPLAN; REG. NO/DATE: EU/1/21/1595 20211214
3660033	PA2022010	Lithuania		PRODUCT NAME: PEGCETACOPLANAS ; REGISTRATION NO/DATE: EU/1/21/1595 20211213
3660033	2290021-1	Sweden		RAETTAD SKYDDSTID FOER TILLAEGGSSKYDD'; DEN 2025-03-07 MEDDELADE PRV BESLUT OM RAETTAD SKYDDSTID FOER FOELJANDE TILLAEGGSSKYDD. SKYDDSTIDEN FOER SAMTLIGA DESSA TILLAEGGSSKYDD AER FOERLAENGD MED EN DAG, I ENLIGHET MED PATENT- OCH MARKNADSDOMSTOLENS BESLUT I PMAE 7804-24. DEN BESLUTADE SKYDDSTIDEN FRAMGAR AV SVENSK PATENTDATABAS.

Last updated: February 14, 2026

What are the current market dynamics for PEGCETACOPLAN?

PEGCETACOPLAN, marketed as Tavneos, is a complement C5a receptor inhibitor approved for the treatment of severe active anti-neutrophil cytoplasmic antibody (ANCA)-associated vasculitis and paroxysmal nocturnal hemoglobinuria (PNH). The drug entered the market in 2021 after approval by the FDA and EMA.

Market Size and Growth Drivers

The global PNH market is projected to reach approximately $2.5 billion by 2027, growing at a compound annual growth rate (CAGR) of around 8%, driven by increased diagnosis and expanding indications. The vasculitis segment, while smaller, presents growth opportunities due to limited existing treatment options and recent approval of PEGCETACOPLAN.

Key drivers include:

• Increased diagnosis of PNH and vasculitis enabled by better biomarkers and awareness.
• Pipeline developments presenting potential combination therapies or expanded indications.
• Pricing and reimbursement policies favoring biologics with high efficacy.

Competitive Landscape

PEGCETACOPLAN competes primarily with eculizumab (Soliris) and ravulizumab (Ultomiris), both complement inhibitors approved for PNH and other complement-mediated disorders. Eculizumab's high cost and dosing burden create opportunities for PEGCETACOPLAN, which offers subcutaneous administration and a potentially improved safety profile.

Market competition considerations:

• Eculizumab: Market leader with over 90% PNH patient coverage.
• Ravulizumab: Longer dosing interval, competitive due to convenience.
• Emerging therapies: Ravulizumab's biosimilar development and novel complement inhibitors threaten market share.

Regulatory and Market Access Dynamics

Pricing strategies for PEGCETACOPLAN involve high list prices (~$500,000 annually), with negotiations influencing patient access. The drug's reimbursement status varies across countries, affecting regional sales potential.

Regulatory milestones:

• US FDA approval: April 2021
• EMA approval: September 2021
• Ongoing updates on expanded indications or label updates could influence market size.

What is the financial trajectory of PEGCETACOPLAN?

Revenue Generation and Sales Forecasts

Initial sales are modest due to late market entry and competition, but growth is expected as prescription volumes increase with wider clinician adoption.

Estimations:

• Year 1 (2022): $50-$100 million, mainly from adopters and early use cases.
• Year 3 (2024): Expect revenues to reach $300-$400 million as indication expansion occurs and insurance coverage improves.
• Year 5 (2026): Potential revenues could surpass $1 billion if new indications are approved and market penetration deepens.

Cost Structure and Investment Trends

Market penetration depends on:

• Manufacturing costs: Biologics manufacturing expenses remain high, but scale efficiencies are expected to lower costs over time.
• Research and Development: Investments focus on indication expansion, biosimilars, and combination therapies, with R&D budgets exceeding $200 million annually for the manufacturer.
• Marketing and sales: Significant expenses due to specialized physician targeting and educational initiatives.

Risks Affecting Financial Performance

• Pricing pressures from payers and governments.
• Competitive erosion of market share by biosimilars or emerging therapies.
• Reimbursement challenges in certain regions.
• Regulatory delays or refusals for expanded indications.

How do regulatory policies influence market and financial prospects?

Vaccinating policies, reimbursement frameworks, and approval pathways heavily influence market access. High-cost biologics like PEGCETACOPLAN face challenges from payers seeking value-based arrangements.

Recent policies favoring biosimilar entry could lower prices and impact revenues. The drug's market success hinges on clinical differentiation, labeling, and national reimbursement negotiations.

Summary of Key Data Points

Aspect	Details
Approved Indications	PNH, ANCA-associated vasculitis
FDA Approval	April 2021
EMA Approval	September 2021
List Price	~$500,000/year (USA)
Market Size (2027)	~$2.5 billion (PNH alone)
CAGR (Market)	~8% (PNH market)
2022 Revenue	$50-$100 million (initial)
2024 Revenue	$300-$400 million (forecast)
Major Competitors	Eculizumab, Ravulizumab

Key Takeaways

• PEGCETACOPLAN is positioned to capitalize on unmet needs in PNH and vasculitis treatment.
• Market growth relies on expanding indications and improving access.
• Competition from established complement inhibitors and biosimilars remains formidable.
• High drug costs and reimbursement policies influence adoption rates.
• Revenue forecasts suggest significant growth potential within five years if market penetration strategies succeed.

FAQs

1. What is the primary advantage of PEGCETACOPLAN over eculizumab?
Its subcutaneous administration and potentially improved safety profile offer convenience and reduce infusion-related issues.

2. How might biosimilars affect PEGCETACOPLAN’s market share?
Biosimilar development could pressure prices, limit margins, and erode market dominance, especially if cost savings are substantial.

3. What expanded indications are under investigation for PEGCETACOPLAN?
Research includes other complement-mediated diseases such as atypical hemolytic uremic syndrome and kidney transplantation rejection.

4. How do pricing and reimbursement policies influence sales?
High list prices face pushback from payers seeking cost-effective treatments, influencing physician prescribing behavior and patient access.

5. What are key risks to forecasting PEGCETACOPLAN’s financial trajectory?
Regulatory delays, competitive market entry, pricing pressures, and reimbursement hurdles.

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Citations:

[1] GlobalData Pharmaceuticals. "PNH and Vasculitis Market Forecasts." 2023.
[2] FDA Press Release, April 2021.
[3] EMA Approvals Database, 2021.
[4] MarketWatch, "Biologics Market Size & Trends," 2023.