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 thirteen patents protecting this compound. Additional information is available in the individual branded drug profile pages.

Pegcetacoplan has one hundred and ninety-three patent family members in twenty-six countries.

One supplier is listed for this compound.

Summary for pegcetacoplan

International Patents:	193
US Patents:	13
Tradenames:	2
Applicants:	1
NDAs:	2
Finished Product Suppliers / Packagers:	1
Clinical Trials:	19
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	Phase 2
National Cancer Institute (NCI)	Phase 2
Swedish Orphan Biovitrum	Phase 2

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	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	10,125,171			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	10,035,822			Y
Apellis Pharms	SYFOVRE	pegcetacoplan	SOLUTION;INTRAVITREAL	217171-001	Feb 17, 2023		RX	Yes	Yes	10,875,893			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/005553 Aspaveli 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
European Patent Office	2707384	ANALOGUES DE LA COMPSTATINE CIBLÉS, À LONGUE DURÉE D'ACTION, RÉACTIFS AUX CELLULES, ET LEURS UTILISATIONS (CELL-REACTIVE, LONG-ACTING, OR TARGETED COMPSTATIN ANALOGS AND USES THEREOF)
Australia	2006302212	Compstatin and analogs thereof for eye disorders
World Intellectual Property Organization (WIPO)	2007084765
Russian Federation	2013149956	КЛЕТОЧНО-РЕАКТИВНЫЕ АНАЛОГИ КОМПСТАТИНА, АНАЛОГИ КОМПСТАТИНА ДЛИТЕЛЬНОГО ДЕЙСТВИЯ ИЛИ АНАЛОГИ КОМПСТАТИНА НАЦЕЛЕННОГО ДЕЙСТВИЯ И ИХ ПРИМЕНЕНИЕ
Japan	2013531030
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	122022000034	Germany		PRODUCT NAME: PEGCETACOPLAN; REGISTRATION NO/DATE: EU/1/21/1595 20211213
3660033	PA2022010	Lithuania		PRODUCT NAME: PEGCETACOPLANAS ; REGISTRATION NO/DATE: EU/1/21/1595 20211213
3660033	2022C/522	Belgium		PRODUCT NAME: PEGCETACOPLAN; AUTHORISATION NUMBER AND DATE: EU/1/21/1595 20211214
3660033	CR 2022 00023	Denmark		PRODUCT NAME: PEGCETACOPLAN; REG. NO/DATE: EU/1/21/1595 20211214
3660033	2022017	Norway		PRODUCT NAME: PEGCETACOPLAN; REG. NO/DATE: EU/1/21/1595 20211216
3660033	301178	Netherlands		PRODUCT NAME: PEGCETACOPLAN; REGISTRATION NO/DATE: EU/1/21/1595 20211214

Pegcetacoplan Market Analysis and Financial Projection Experimental

Market Dynamics and Financial Trajectory for Pegcetacoplan

Introduction to Pegcetacoplan

Pegcetacoplan, marketed by Apellis Pharmaceuticals as Empaveli for certain blood disorders and as Syfovre for eye conditions, is a synthetic cyclic peptide conjugated to a polyethylene glycol polymer. It functions as a targeted C3 inhibitor, regulating excessive complement activation that leads to various serious diseases[4].

Clinical Indications and Approvals

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Pegcetacoplan is approved to treat adult patients with PNH, a rare blood disorder. It has shown significant efficacy in reducing the symptoms associated with this condition[4].

Geographic Atrophy

In early 2023, the FDA approved pegcetacoplan (Syfovre) for the treatment of geographic atrophy, an eye condition. Despite initial strong sales in the U.S., the drug faced setbacks due to concerns over a rare but serious side effect and a recommendation for rejection by European regulators, although Apellis has requested a re-examination[1].

Kidney Diseases

Recent trials have demonstrated the drug's potential in treating kidney diseases such as C3 glomerulopathy (C3G) and primary immunoglobulin A (IgA) nephropathy. The positive 6-month Phase 3 VALIANT results have prompted Apellis to plan an FDA filing for these indications in early 2025[2].

Market Competition

Comparison with Iptacopan

Pegcetacoplan faces competition from iptacopan (Fabhalta), another drug approved for PNH and IgA nephropathy. Analysts suggest that the strong data from pegcetacoplan's trials may force investors to reconsider their expectations regarding market share between these two drugs[1].

Emerging Therapies

The market for PNH and other related diseases is expected to see the launch of late-stage emerging therapies, which could significantly impact the dominance of pegcetacoplan. Companies are developing novel approaches to treat these conditions, posing potential competition[4].

Financial Performance

Revenue Growth

In the third quarter of 2024, Apellis generated $196.8 million in revenues, with $176.6 million coming from U.S. net product sales. Syfovre (pegcetacoplan injection) contributed $152.0 million, showing a 7% quarter-over-quarter growth in demand[2].

Cost of Sales and Operating Expenses

The cost of sales increased to $33.6 million in the third quarter of 2024, compared to $22.4 million in the same period of 2023. Operating expenses, including research and development and selling, general, and administrative costs, totaled $244.1 million for the quarter[2].

Cash and Cash Equivalents

As of September 30, 2024, Apellis had $396.9 million in cash and cash equivalents. The company projects that its current revenues and cash reserves will be sufficient to fund operations until it reaches positive cash flow[2].

Regulatory Milestones

FDA Feedback and Future Filings

Following positive feedback from the FDA, Apellis is on track to file a supplemental New Drug Application (sNDA) seeking approval for pegcetacoplan in C3G and primary IC-MPGN in early 2025[2].

European Regulatory Status

Despite the European Medicines Agency's (EMA) recommendation to reject Syfovre, Apellis has initiated a re-examination process. A final decision is expected in the fourth quarter of 2024[3].

Clinical Trials and Data

Phase 3 VALIANT Results

The 6-month Phase 3 VALIANT results showed significant efficacy of pegcetacoplan in reducing urine protein levels by 68% compared to placebo. This data is crucial for the upcoming FDA filing[1].

Phase 2 NOBLE Study

Positive top-line data from the Phase 2 NOBLE study, investigating pegcetacoplan in post-transplant recurrence of primary IC-MPGN and C3G, further supports the drug's potential in kidney diseases[5].

Analyst Insights

Market Share Reconsideration

Analysts like Steven Seedhouse from Raymond James suggest that the strong data from pegcetacoplan's trials may force investors to reconsider their expectations regarding its market share versus iptacopan[1].

Financial Projections

Given the positive clinical data and growing demand for Syfovre, analysts are optimistic about Apellis's financial trajectory. The company's ability to manage costs and drive revenue growth is seen as a key factor in its future success[2].

Market Forecasts

Sales Projections

The market for pegcetacoplan is expected to expand significantly from 2023 to 2032, driven by increasing healthcare spending and the drug's efficacy in treating various conditions. Forecasted sales data indicate a growing market size that will allow drug manufacturers to penetrate deeper into the market[4].

Competitive Landscape

The launch of late-stage emerging therapies will significantly impact the market dynamics. Companies are focusing on novel approaches to treat diseases, which could challenge pegcetacoplan's dominance but also present opportunities for growth and innovation[4].

Conclusion

Pegcetacoplan, with its diverse clinical indications and strong clinical trial data, is poised for significant market impact. Despite facing competition and regulatory challenges, the drug's financial trajectory looks promising, driven by growing demand and expanding market opportunities.

Key Takeaways

• Clinical Efficacy: Pegcetacoplan has shown strong efficacy in treating PNH, geographic atrophy, and potential in kidney diseases.
• Market Competition: The drug faces competition from iptacopan and emerging therapies.
• Financial Performance: Apellis has seen revenue growth and manages its costs effectively.
• Regulatory Milestones: Upcoming FDA filings and re-examination in Europe are critical.
• Market Forecasts: The market is expected to expand significantly from 2023 to 2032.

FAQs

Q: What is pegcetacoplan used for?

Pegcetacoplan is used to treat paroxysmal nocturnal hemoglobinuria (PNH), geographic atrophy, and is being investigated for use in kidney diseases such as C3 glomerulopathy (C3G) and primary immunoglobulin A (IgA) nephropathy.

Q: What are the key competitors for pegcetacoplan?

The key competitor for pegcetacoplan is iptacopan (Fabhalta), which is also approved for PNH and IgA nephropathy.

Q: How has the financial performance of Apellis been with pegcetacoplan?

Apellis has generated significant revenues from pegcetacoplan, with $196.8 million in revenues in the third quarter of 2024, and the company projects sufficient cash reserves to fund operations until reaching positive cash flow.

Q: What are the regulatory challenges facing pegcetacoplan in Europe?

Pegcetacoplan (Syfovre) faced a recommendation for rejection by European regulators, but Apellis has initiated a re-examination process with a final decision expected in the fourth quarter of 2024.

Q: What are the market forecasts for pegcetacoplan?

The market for pegcetacoplan is expected to expand significantly from 2023 to 2032, driven by increasing healthcare spending and the drug's efficacy in treating various conditions.

Sources

1. Yahoo Finance: Apellis plans FDA filing after drug success in kidney trial.
2. Apellis Pharmaceuticals: Apellis Pharmaceuticals Reports Third Quarter 2024 Financial Results.
3. Apellis Pharmaceuticals: Apellis Pharmaceuticals Reports Second Quarter 2024 Financial Results.
4. GlobeNewswire: EMPAVELI (Pegcetacoplan) Drug Insight and Market Forecasts.
5. Apellis Pharmaceuticals: Apellis Pharmaceuticals Reports Third Quarter 2023 Financial Results.