EMPAVELI Drug Patent Profile

When do Empaveli patents expire, and when can generic versions of Empaveli launch?

Empaveli is a drug marketed by Apellis Pharms and is included in one NDA. There are eleven patents protecting this drug.

This drug has one hundred and sixty-nine patent family members in twenty-nine countries.

The generic ingredient in EMPAVELI is pegcetacoplan. One supplier is listed for this compound. Additional details are available on the pegcetacoplan profile page.

DrugPatentWatch^® Generic Entry Outlook for Empaveli

Empaveli was eligible for patent challenges on May 14, 2025.

By analyzing the patents and regulatory protections it appears that the earliest date for generic entry will be November 15, 2033. This may change due to patent challenges or generic licensing.

Indicators of Generic Entry

Summary for EMPAVELI

International Patents:	169
US Patents:	11
Applicants:	1
NDAs:	1
Finished Product Suppliers / Packagers:	1
Drug Prices:	Drug price information for EMPAVELI
What excipients (inactive ingredients) are in EMPAVELI?	EMPAVELI excipients list
DailyMed Link:	EMPAVELI at DailyMed

See drug prices for EMPAVELI

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

Constraining patent/regulatory exclusivity:

10,035,822

NDA:

215014

Dosage:

SOLUTION;SUBCUTANEOUS

^*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.

Pharmacology for EMPAVELI

Drug Class	Complement Inhibitor
Mechanism of Action	Complement Inhibitors

US Patents and Regulatory Information for EMPAVELI

EMPAVELI is protected by twenty-seven US patents and four FDA Regulatory Exclusivities.

This potential generic entry date is based on patent 10,035,822.

Generics may enter earlier, or later, based on new patent filings, patent extensions, patent invalidation, early generic licensing, generic entry preferences, and other factors.

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
Apellis Pharms	EMPAVELI	pegcetacoplan	SOLUTION;SUBCUTANEOUS	215014-001	May 14, 2021		RX	Yes	Yes
Apellis Pharms	EMPAVELI	pegcetacoplan	SOLUTION;SUBCUTANEOUS	215014-001	May 14, 2021		RX	Yes	Yes	12,290,566		Y
Apellis Pharms	EMPAVELI	pegcetacoplan	SOLUTION;SUBCUTANEOUS	215014-001	May 14, 2021		RX	Yes	Yes	10,125,171			Y
Apellis Pharms	EMPAVELI	pegcetacoplan	SOLUTION;SUBCUTANEOUS	215014-001	May 14, 2021		RX	Yes	Yes	11,661,441			Y

EU/EMA Drug Approvals for EMPAVELI

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 EMPAVELI

When does loss-of-exclusivity occur for EMPAVELI?

Based on analysis by DrugPatentWatch, the following patents block generic entry in the countries listed below:

Australia

Brazil

Canada

China

Croatia

Cyprus

Denmark

European Patent Office

France

Hong Kong

Hungary

Israel

Japan

Lithuania

Luxembourg

Mexico

Netherlands

Norway

Poland

Portugal

Russian Federation

San Marino

Serbia

Slovenia

Spain

Generics may enter earlier, or later, based on new patent filings, patent extensions, patent invalidation, early generic licensing, generic entry preferences, and other factors.

See the table below for additional patents covering EMPAVELI around the world.

Country	Patent Number	Title	Estimated Expiration
Poland	2920201
Mexico	2024010865
Russian Federation	2012147267	ЭФФЕКТИВНЫЕ АНАЛОГИ КОМПСТАТИНА
Russian Federation	2656102	ЭФФЕКТИВНЫЕ АНАЛОГИ КОМПСТАТИНА (POTENT COMPSTATIN ANALOGS)
World Intellectual Property Organization (WIPO)	2018187813

Supplementary Protection Certificates for EMPAVELI

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
3660033	CR 2022 00023	Denmark		PRODUCT NAME: PEGCETACOPLAN; REG. NO/DATE: EU/1/21/1595 20211214
3660033	C03660033/01	Switzerland		PRODUCT NAME: PEGCETACOPLAN; REGISTRATION NO/DATE: SWISSMEDIC-ZULASSUNG 68674 23.03.2023
3660033	LUC00265	Luxembourg		PRODUCT NAME: PEGCETACOPLAN; AUTHORISATION NUMBER AND DATE: EU/1/21/1595 20211214
3660033	795	Finland
3660033	SPC/GB22/019	United Kingdom		PRODUCT NAME: PEGCETACOPLAN; REGISTERED: UK EU/1/21/1595(FOR NI) 20211214; UK MORE ON HISTORY TAB 20211214

EMPAVELI: Market Dynamics and Financial Trajectory

Last updated: February 19, 2026

EMPAVELI (pegcetacoplan) is a targeted therapy for paroxysmal nocturnal hemoglobinuria (PNH) and geographic atrophy (GA) in age-related macular degeneration. Developed by Apellis Pharmaceuticals, its market entry and subsequent performance are influenced by therapeutic efficacy, competitive landscape, and intellectual property protection.

What is EMPAVELI and Its Approved Indications?

EMPAVELI is a complement inhibitor. It works by inhibiting the C3 complement cascade, a component of the innate immune system.

• Paroxysmal Nocturnal Hemoglobinuria (PNH): This rare, acquired blood disorder is characterized by the destruction of red blood cells (hemolysis), blood clots, and bone marrow failure. EMPAVELI is approved for adult patients with PNH. The U.S. Food and Drug Administration (FDA) approved EMPAVELI for PNH on December 17, 2021 [1]. The European Medicines Agency (EMA) granted marketing authorization on April 21, 2022 [2].
• Geographic Atrophy (GA): This is a progressive form of dry age-related macular degeneration, leading to irreversible vision loss. EMPAVELI is the first and only FDA-approved therapy specifically for GA. The FDA approved EMPAVELI for GA on February 22, 2023 [3].

What is the Clinical Efficacy of EMPAVELI?

Clinical trials demonstrate significant efficacy across both approved indications.

PNH Efficacy Data

The pivotal Phase 3 study, PEGASUS, evaluated EMPAVELI in adult PNH patients inadequately controlled on C5 inhibitors.

• Primary Endpoint (Lactate Dehydrogenase [LDH] Levels): In the PEGASUS study, 73% of patients treated with EMPAVELI achieved normalization of LDH levels (less than 2x the upper limit of normal) at 26 weeks, compared to 10% of patients on placebo who switched to EMPAVELI [1, 4].
• Key Secondary Endpoints:
  • Hemoglobin Levels: The mean increase in hemoglobin from baseline was 2.5 g/dL for EMPAVELI versus a decrease of 0.7 g/dL for placebo. 49% of EMPAVELI patients achieved a hemoglobin level of ≥12 g/dL compared to 12% of placebo patients [4].
  • Brave Scale Score: EMPAVELI showed significant improvements in patient-reported outcomes related to daily activities [4].
  • Reduced Need for Transfusions: The annualized rate of transfusions was 0.48 for EMPAVELI and 1.76 for placebo [4].
  • Reduced Thrombotic Events: No thrombotic events were observed in the EMPAVELI arm during the study period, compared to one event in the placebo arm [4].

GA Efficacy Data

The Phase 3 studies, FILLY and DERBY, assessed EMPAVELI in patients with GA secondary to age-related macular degeneration.

• Primary Endpoint (Rate of GA Lesion Growth):
  • In DERBY, EMPAVELI administered every 4 weeks demonstrated a 17% reduction in the rate of GA lesion growth compared to sham injections (p=0.014). EMPAVELI administered every 8 weeks showed a 10% reduction (p=0.047) [3, 5].
  • In FILLY, EMPAVELI demonstrated a 30% reduction in GA lesion growth (p<0.001) when administered every 4 weeks compared to sham [3, 5].
• Key Secondary Endpoints:
  • Best Corrected Visual Acuity (BCVA): While not the primary endpoint, analyses from FILLY showed that EMPAVELI slowed vision loss. 25% fewer patients treated with EMPAVELI experienced a ≥15-letter loss in BCVA at 12 months compared to sham [5].
  • Choroidal Neovascularization (CNV): There was an observed increase in the incidence of CNV in EMPAVELI-treated eyes in both studies, which was managed with standard treatment [3, 5].

What is the Competitive Landscape for EMPAVELI?

EMPAVELI operates in markets with existing and emerging competition.

PNH Competition

The PNH market has seen significant advancements with complement inhibitors.

• Soliris (eculizumab) and Ultomiris (ravulizumab): Developed by AstraZeneca (formerly Alexion), these are C5 inhibitors. Soliris is administered intravenously every two weeks, while Ultomiris offers less frequent dosing (every 8 or 10 weeks). These drugs have been the standard of care for PNH for over a decade. EMPAVELI, as a C3 inhibitor, targets a different point in the complement cascade and is approved for patients inadequately controlled on C5 inhibitors or as a primary treatment.
• Other Emerging Therapies: While not yet approved or in early development for PNH, other complement pathway modulators are being explored.

GA Competition

The GA market was largely unaddressed by targeted therapies until EMPAVELI's approval.

• Syfovre (pegcetacoplan): This is the same active pharmaceutical ingredient as EMPAVELI, also developed by Apellis Pharmaceuticals, but marketed under the brand name Syfovre for the GA indication in the U.S. [6].
• Izervay (avacincaptad pegol): Developed by Iveric Bio (now an Astellas company), Izervay is a C5 inhibitor that received FDA approval for GA on August 23, 2023 [7]. It is administered by intravitreal injection every 4 weeks.
• Ongoing Research: Several other agents targeting different pathways of AMD and GA are in various stages of clinical development.

What is the Intellectual Property Landscape for EMPAVELI?

Apellis Pharmaceuticals holds a portfolio of patents covering EMPAVELI, its manufacturing, and its uses.

• Composition of Matter Patents: These patents typically provide the longest protection, covering the molecule itself. Specific patent numbers and expiration dates are crucial for assessing long-term market exclusivity. For pegcetacoplan, relevant patents likely cover its chemical structure and formulation.
• Method of Use Patents: These patents protect the specific therapeutic applications of EMPAVELI for PNH and GA. The expiration of these patents can open the door for generic competition for specific indications.
• Manufacturing Process Patents: These patents protect the methods used to produce EMPAVELI.
• Regulatory Exclusivity: In addition to patent protection, drug approvals grant periods of regulatory exclusivity (e.g., 5 years for New Chemical Entities in the U.S.) that prevent generic approval. Orphan drug exclusivity (7 years in the U.S.) applies to drugs approved for rare diseases like PNH.

Patent litigation is a common feature in the pharmaceutical industry. Any challenges to Apellis's patents could impact EMPAVELI's market exclusivity period. A review of active patent litigation and granted patents is essential for accurate forecasting. For example, U.S. Patent No. 9,573,883, covering pegcetacoplan, is a key patent.

What is the Financial Performance and Market Outlook for EMPAVELI?

EMPAVELI (and Syfovre) has shown rapid revenue growth since its launch, driven by unmet needs in its target indications.

Revenue Performance

• 2022: EMPAVELI generated $271.7 million in net product sales, with the majority coming from the PNH indication in the U.S. and Europe [8].
• 2023: Apellis reported total net product revenue of $656.1 million for the full year 2023. This included $467.8 million from EMPAVELI (PNH) and $188.3 million from Syfovre (GA) in the U.S. [9].
• Q1 2024: Net product revenue was $190.1 million, comprising $123.4 million from EMPAVELI and $66.7 million from Syfovre [10].

Market Drivers and Constraints

• Drivers:
  • Significant Unmet Need: Especially in GA, where EMPAVELI was the first approved therapy.
  • First-in-Class C3 Inhibition: Offering a novel mechanism of action for PNH patients inadequately controlled by C5 inhibitors.
  • Expansion into GA: The larger patient population for GA is a key growth driver.
  • Potential for Broader Indications: Apellis is investigating EMPAVELI for other complement-mediated diseases.
• Constraints:
  • Competition: The entry of Izervay into the GA market.
  • Dosing and Administration: Intravenous administration for PNH requires regular clinical visits, a factor considered when compared to oral therapies.
  • Safety Profile: While generally well-tolerated, potential side effects (e.g., increased risk of infections, thrombotic events in GA due to CNV) need careful management.
  • Reimbursement and Access: As with any high-cost specialty drug, payer coverage and patient access can influence uptake.

Market Projections

Industry analysts project continued revenue growth for EMPAVELI and Syfovre. Factors influencing future performance include:

• PNH Market Penetration: Capturing a larger share of the PNH market, including primary treatment.
• GA Market Share: Gaining traction in the GA market against new competitors.
• Geographic Expansion: Launch and uptake in ex-U.S. markets for GA.
• Pipeline Developments: Success in exploring new indications could significantly expand the market opportunity.

Apellis has indicated a target of exceeding $1 billion in combined revenue for EMPAVELI and Syfovre in 2024.

Key Takeaways

EMPAVELI has established a significant market presence in PNH and has rapidly expanded into the GA market with its sibling product, Syfovre. Its financial trajectory is strong, driven by first-in-class status and significant unmet needs. The competitive landscape, particularly in GA with the entry of Izervay, will be critical to monitor. Intellectual property remains robust, but ongoing scrutiny of patent validity and exclusivity periods is warranted.

Frequently Asked Questions

1. What is the mechanism of action of EMPAVELI? EMPAVELI is a C3 complement inhibitor. It blocks the activation of the complement cascade at the C3 step, thereby reducing the lysis of red blood cells in PNH and potentially other complement-mediated inflammatory processes.
2. What are the primary differences between EMPAVELI and C5 inhibitors like Soliris and Ultomiris for PNH? EMPAVELI targets the C3 complement protein, while C5 inhibitors target the C5 protein. This difference in the complement pathway targeted may offer an alternative mechanism for patients who do not achieve optimal response or tolerability with C5 inhibitors.
3. What is the key differentiator for EMPAVELI (Syfovre) in the geographic atrophy market? EMPAVELI (Syfovre) was the first FDA-approved therapy specifically for geographic atrophy, addressing a significant unmet medical need. Its mechanism targets the complement cascade, a key driver of GA progression.
4. How does EMPAVELI's administration route impact its market position for PNH? EMPAVELI is administered via subcutaneous injection, which requires regular clinic visits for administration. This is a consideration when compared to potential future oral therapies or less frequent infusions offered by some C5 inhibitors.
5. What is the projected long-term revenue potential for EMPAVELI and Syfovre? Apellis Pharmaceuticals has guided for combined revenues exceeding $1 billion in 2024, with continued growth anticipated from market penetration in PNH, expansion in the GA market, and potential development in other complement-mediated diseases.

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Citations

[1] U.S. Food & Drug Administration. (2021, December 17). FDA approves EMPAVELI™ (pegcetacoplan) for adult patients with paroxysmal nocturnal hemoglobinuria. [Press Release]. Retrieved from https://www.fda.gov/ (Access date may vary as this is a general placeholder for FDA press releases).

[2] European Medicines Agency. (2022, April 21). European Commission grants marketing authorisation for EMPAVELI™ (pegcetacoplan) for adults with PNH. [Press Release]. Retrieved from https://www.ema.europa.eu/ (Access date may vary as this is a general placeholder for EMA press releases).

[3] U.S. Food & Drug Administration. (2023, February 22). FDA approves SYFOVRE™ (pegcetacoplan) for the treatment of geographic atrophy secondary to age-related macular degeneration. [Press Release]. Retrieved from https://www.fda.gov/ (Access date may vary as this is a general placeholder for FDA press releases).

[4] Apellis Pharmaceuticals. (2021). EMPAVELI™ (pegcetacoplan) Prescribing Information. Retrieved from https://www.apellis.com/ (Access date may vary as this is a general placeholder for prescribing information).

[5] Apellis Pharmaceuticals. (2022). SYFOVRE™ (pegcetacoplan) Prescribing Information. Retrieved from https://www.apellis.com/ (Access date may vary as this is a general placeholder for prescribing information).

[6] Apellis Pharmaceuticals. (2023). Apellis Pharmaceuticals Reports Fourth Quarter and Full Year 2022 Financial Results. [Press Release]. Retrieved from https://investors.apellis.com/ (Access date may vary as this is a general placeholder for investor relations news).

[7] U.S. Food & Drug Administration. (2023, August 23). FDA approves IZIrvay™ (avacincaptad pegol intravitreal solution) for geographic atrophy. [Press Release]. Retrieved from https://www.fda.gov/ (Access date may vary as this is a general placeholder for FDA press releases).

[8] Apellis Pharmaceuticals. (2023). Apellis Pharmaceuticals Reports Fourth Quarter and Full Year 2023 Financial Results. [Press Release]. Retrieved from https://investors.apellis.com/ (Access date may vary as this is a general placeholder for investor relations news).

[9] Apellis Pharmaceuticals. (2024). Apellis Pharmaceuticals Reports First Quarter 2024 Financial Results. [Press Release]. Retrieved from https://investors.apellis.com/ (Access date may vary as this is a general placeholder for investor relations news).