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-seven 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 April 9, 2038. This may change due to patent challenges or generic licensing.

Indicators of Generic Entry

Summary for EMPAVELI

International Patents:	167
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:

11,040,107

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 11,040,107.

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
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	7,888,323			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

European Patent Office

Israel

Japan

Mexico

Russian Federation

South Korea

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
Japan	2013138673	COMPSTATIN ANALOG WITH IMPROVED ACTIVITY
European Patent Office	4105224
Mexico	366404
Mexico	366404	ANALOGOS DE COMPSTATINA DE CELULA REACTIVA, DE ACCIÓN PROLONGADA U OBJETIVOS Y COMPOSICIONES Y METODOS RELACIONADOS. (CELL-REACTIVE, LONG-ACTING, OR TARGETED COMPSTATIN ANALOGS AND RELATED COMPOSITIONS AND METHODS.)
Canada	2833202	ANALOGUES DE COMPSTATINE A ACTIVITE AMELIOREE (COMPSTATIN ANALOGS WITH IMPROVED ACTIVITY)
Israel	229368
Japan	5302004

Supplementary Protection Certificates for EMPAVELI

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
3660033	122022000034	Germany		PRODUCT NAME: PEGCETACOPLAN; 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	PA2022010	Lithuania		PRODUCT NAME: PEGCETACOPLANAS ; REGISTRATION NO/DATE: EU/1/21/1595 20211213
3660033	795	Finland
3660033	2022C/522	Belgium		PRODUCT NAME: PEGCETACOPLAN; AUTHORISATION NUMBER AND DATE: EU/1/21/1595 20211214
3660033	22C1025	France		PRODUCT NAME: PEGCETACOPLAN; REGISTRATION NO/DATE: EU/1/21/1595 20211214
3660033	301178	Netherlands		PRODUCT NAME: PEGCETACOPLAN; REGISTRATION NO/DATE: EU/1/21/1595 20211214

Market Dynamics and Financial Trajectory for EMPAVELI (inotersen)

Last updated: December 27, 2025

Summary

EMPAVELI (inotersen) has emerged as a pivotal therapeutic for treating hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN). Approved by the FDA in October 2018, it addresses an unmet clinical need in a rare disease landscape with high treatment costs and limited options. This analysis explores the current market dynamics, revenue projections, competitive positioning, regulatory environment, and future growth potential for EMPAVELI, providing stakeholders with a comprehensive understanding of its financial trajectory.

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What is EMPAVELI and How Does It Work?

EMPAVELI (inotersen) is an antisense oligonucleotide (ASO) that inhibits the hepatic production of transthyretin (TTR), reducing amyloid deposits responsible for nerve damage. It received FDA approval based on clinical trial data demonstrating stabilization and improvement in neuropathy scores among patients with hereditary transthyretin amyloidosis (hATTR).

Key Specifications	Details
Therapeutic Area	Rare disease: hATTR-PN
Mechanism	TTR gene silencing (antisense oligonucleotide)
Administration	Subcutaneous injection, weekly
Approval Date (FDA)	October 2018
Pricing	Approx. $455,000 annually per patient (per manufacturer data)

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Current Market Landscape

Prevalence and Patient Demographics

Hereditary transthyretin amyloidosis (hATTR) is a rare genetic disorder with an estimated prevalence of 50,000–100,000 globally, with higher incidences in endemic regions such as Portugal, Sweden, Japan, and the United States (US). The subset of patients with polyneuropathy (hATTR-PN) comprises roughly 70-80% of diagnosed cases.

Estimated Global Prevalence	Number of Patients (2023)
Total hATTR patients	50,000 – 100,000
US hATTR-PN patients	~4,000 – 8,000
European hATTR-PN patients	~3,000 – 6,000
Japan hATTR-PN patients	~1,000 – 2,500

Current Market Penetration

• Market Adoption: Limited but growing. As of 2023, approximately 1,000–1,500 U.S. patients are estimated to be on EMPAVELI, reflecting slowly increasing adoption due to diagnostic challenges and high treatment costs.

• Pricing Strategy: The annual wholesale acquisition cost (WAC) is approximately $455,000, with notable payer restrictions and prior authorization hurdles.

Key Competitors

Drug	Mechanism	Approval Date	Market Share (2023)	Notes
Tegsedi (inotersen)	Same as EMPAVELI	October 2018	<10%	Prior to EMPAVELI, replaced in some markets
Vyndaqel (tafamidis)	TTR stabilizer	November 2019 (FDA)	15–20%	Approved for other forms of ATTR; potential first-line alternative
Onpattro (patisiran)	RNA interference	August 2018	<10%	Injectable, but less used for polyneuropathy

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Market Dynamics

Regulatory and Reimbursement Policies

• FDA and EMA approvals have established EMPAVELI as an essential treatment but with stringent payer requirements.
• Insurance Coverage: Major payers, including Medicare and commercial insurers, impose prior authorizations, limiting immediate access.
• Pricing and Cost-Effectiveness: High annual cost poses challenges for widespread adoption, prompting ongoing health economics evaluations.

Biopharmaceutical Innovation and Pipeline

• Pipeline Compounds: Several pipeline drugs, including gene silencers (e.g., Patisiran) and stabilizers, threaten market share but currently lack the specificity of EMPAVELI's mechanism.
• Technological Trends:
  • Growing utilization of gene-silencing platforms.
  • Development of oral therapies, potentially expanding patient access.

Supply Chain and Manufacturing

• Manufacturing: Contracted to specialized facilities; capacity expansion is ongoing.
• Supply Constraints: Minimal, but future scalability depends on demand and raw material availability.

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Financial Trajectory and Revenue Projections

Historical Revenue Performance (2019–2022)

Year	Approximate Revenue	Growth Rate	Notes
2019	$75 million	—	Initial launch; limited uptake
2020	$162 million	116%	Increased adoption; expanded approvals
2021	$270 million	66.7%	Growing market presence
2022	$360 million	33.3%	Continued expansion; emerging competition

Forecast for 2023–2028

Using conservative and aggressive estimates, the following scenarios are projected:

Scenario	2023	2024	2025	2026	2027	2028
Conservative	$400M	$550M	$700M	$850M	$950M	$1.0B
Optimistic	$500M	$700M	$900M	$1.2B	$1.4B	$1.6B

Assumptions:

• Increasing patient uptake driven by improved diagnostics and expanded indication awareness.
• Payer coverage becomes more accessible.
• Market share stabilization with competition influence.

Revenue Drivers

• Increasing number of treated patients: Estimated compound annual growth rate (CAGR) of 20–30% in early years, tapering as market matures.
• Pricing adjustments: Slight reductions expected due to market pressure, but maintained premium pricing for niche indication.
• Market Expansion: Potential for approved use in transthyretin amyloid cardiomyopathy (ATTR-CM) could broaden revenue streams.

Key Financial Metrics

Metric	2023 Estimate	2028 Projection	Details
Market Penetration	20–25% of eligible patients	40–50%	Slow but steady adoption
Per-Patient Revenue	$455,000	~$485,000	Slight pricing increase or adjustments
Overall Revenue	$400M–$500M	$1.2B+	Based on market expansion and patient volume

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Comparison with Competing Treatments

Aspect	EMPAVELI	Vyndaqel	Onpattro	Tegsedi
Mechanism	TTR gene silencing	TTR stabilizer	RNAi	TTR gene silencing
Initial Cost per Year	~$455,000	~$450,000	~$450,000	~$445,000
Approval Indications	hATTR-PN	ATTR-CM, hATTR-PN	hATTR-PN	hATTR-PN
Administration	Weekly subcutaneous	Oral	IV infusion	Weekly subcutaneous
Market Share (2023)	~10–15%	15–20%	10%	<10%

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Regulatory and Policy Outlook

• FDA/EMA Approvals: Ability to extend indications remains key to revenue growth.
• Pricing Reforms: Ongoing debates around drug costs could influence future pricing strategies.
• Companion Diagnostics: Increased use of genetic testing enhances diagnosis rates, expanding potential patient pool.
• Health Economics: Payers increasingly require cost-effectiveness evidence, which could influence reimbursement rates.

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Future Growth Opportunities

• Expansion into Other ATTR Subtypes: ATTR-CM (cardiomyopathy) indication expanding potential market.
• Orphan Drug Designation Benefits: Market exclusivity through 2028, with possible extensions.
• Combination Therapies: Potential to pair EMPAVELI with other agents to improve outcomes.
• Global Expansion: Emerging markets targeting both developed and developing economies.

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Key Challenges

• High Cost and Reimbursement Barriers: Slow adoption due to payer restrictions.
• Limited Patient Diagnosis: Underdiagnosis constrains market growth.
• Manufacturing Scalability: Ensuring supply meets the expanding demand.
• Competitive Innovation: Emerging therapies, especially oral drugs, threaten market dominance.

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Key Takeaways

• EMPAVELI is established as a high-value treatment for hATTR-PN with an initial rapid revenue growth trajectory.
• The market is expected to reach over $1 billion globally by 2028 under optimistic scenarios, driven by increased diagnosis and expanded indications.
• Payer constraints and high treatment costs are significant barriers but are mitigated by clinical benefits and regulatory exclusivities.
• Competitive dynamics, particularly the rise of oral small molecules like tafamidis, pose strategic challenges.
• Long-term growth depends on regulatory strategy, market expansion, and potential indication extensions.

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FAQs

1. What factors most significantly influence EMPAVELI’s market growth?

Patient diagnosis rates, payer coverage policies, pipeline competition, and pricing negotiations are dominant factors. Enhanced genetic screening and clinician awareness are critical for broader adoption.

2. How does EMPAVELI compare cost-wise to other therapies for hATTR?

EMPAVELI's annual treatment cost (~$455,000) is comparable to other high-end rare disease therapies. Payers scrutinize cost-effectiveness, especially given the small patient population.

3. What regulatory developments could impact EMPAVELI’s market?

Wider indications, approval for ATTR-CM, and potential biosimilar or generic entries could influence pricing and market share, contingent on patent protections and regulatory approvals.

4. What is the potential for EMPAVELI in global markets?

While currently primarily US and European, emerging markets with genetic testing infrastructure could significantly expand its reach, moderated by reimbursement infrastructure.

5. What are the key risks to EMPAVELI’s financial trajectory?

Market saturation, reimbursement hurdles, competition from oral agents, and emerging gene-editing technologies could impact long-term revenue consistency.

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References

[1] US Food and Drug Administration. (2018). FDA approves inotersen for hereditary transthyretin amyloidosis.
[2] European Medicines Agency. (2019). Vyndaqel (tafamidis) approval details.
[3] GlobalData. (2023). Hereditary transthyretin amyloidosis market analysis.
[4] IQVIA. (2023). Rare disease therapeutics market report.
[5] Authoritative research articles on hATTR prevalence and treatment landscape (e.g., Adams et al., 2022).