Paroxysmal Cold Hemoglobinuria Treatments

Find Paroxysmal Cold Hemoglobinuria Treatments

Generic Name

Pegcetacoplan

Brand Names

Empaveli, Syfovre

FDA approval date: May 14, 2021

Classification: Complement Inhibitor

Form: Injection

What is Empaveli (Pegcetacoplan)?

EMPAVELI is a complement inhibitor indicated: for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria .

Approved To Treat

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Related Clinical Trials

Integration of Lesion-tailored, Fundus-controlled Perimetry Into Routine Clinical Care for Patients With Geographic Atrophy (GA) Receiving Pegcetacoplan Treatment in Accordance With the Approved Label.

Enrollment Status: Recruiting

Publish Date: June 17, 2026

Summary: The goal of this observational study is to evaluate changes in retinal sensitivity over time in patients with geographic atrophy due to age related macular degeneration who are receiving pegcetacoplan as part of routine clinical care. The study aims to determine whether lesion tailored fundus-controlled perimetry can reliably measure functional changes near areas of atrophy and whether this testin...

Safety, Tolerability, Pharmacokinetics, and Exploratory Efficacy of ABBV-6628 in Subjects With Geographic Atrophy Secondary to Age-Related Macular Degeneration

Enrollment Status: Recruiting

Publish Date: June 15, 2026

Intervention Type: Drug

Study Phase: Phase 1/Phase 2

Summary: Age-related macular degeneration (AMD) is the abnormal growth of new blood vessels in the light-sensitive tissue at the back of the eye called the retina. Geographic Atrophy (GA) is an advanced form of dry AMD. The purpose of this study is to assess the adverse events and how intravitreal ABBV-6628 moves through the body of adult participants with secondary to age-related macular degeneration ABBV...

A Phase I/II Study of Complement Inhibition in Pancreatic Ductal Adenocarcinoma

Enrollment Status: Recruiting

Publish Date: June 08, 2026

Intervention Type: Procedure, Drug

Study Phase: Phase 1/Phase 2

Summary: This phase I/II trial tests the effect of pegcetacoplan in combination with oxaliplatin, irinotecan, leucovorin, and fluorouracil (mFOLFIRINOX) in treating patients with pancreatic ductal adenocarcinoma (PDAC) that has spread from where it first started (primary site) to other places in the body (metastatic). Pegcetacoplan works by targeting the immune complement process, a part of the immune syst...

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Related Latest Advances

Real-world pegcetacoplan treatment of paroxysmal nocturnal hemoglobinuria patients with an unsatisfactory response to prior therapies.

Journal: Hematology (Amsterdam, Netherlands)

Published: June 11, 2026

Short-Term Intraocular Pressure Trends After Intravitreal Pegcetacoplan Injection.

Journal: Journal of vitreoretinal diseases

Published: May 26, 2026

Complement Inhibition in Geographic Atrophy: Clinical Evidence for Pegcetacoplan and Avacincaptad Pegol.

Journal: Cureus

Published: May 19, 2026

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Brand Information

Empaveli (Pegcetacoplan)

WARNING: SERIOUS INFECTIONS CAUSED BY ENCAPSULATED BACTERIA

EMPAVELI, a complement inhibitor, increases the risk of serious infections, especially those caused by encapsulated bacteria, such as

Complete or update vaccination for encapsulated bacteria at least 2 weeks prior to the first dose of EMPAVELI, unless the risks of delaying therapy with EMPAVELI outweigh the risk of developing a serious infection. Comply with the most current Advisory Committee on Immunization Practices (ACIP) recommendations for vaccinations against encapsulated bacteria in patients receiving a complement inhibitor.
Patients receiving EMPAVELI are at increased risk for invasive disease caused by encapsulated bacteria, even if they develop antibodies following vaccination. Monitor patients for early signs and symptoms of serious infections and evaluate immediately if infection is suspected.

Because of the risk of serious infections caused by encapsulated bacteria, EMPAVELI is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the EMPAVELI REMS

1DOSAGE FORMS AND STRENGTHS

Injection: 1,080 mg/20 mL (54 mg/mL) clear, colorless to slightly yellowish solution in a single-dose vial.

2CONTRAINDICATIONS

EMPAVELI is contraindicated:

in patients with hypersensitivity to pegcetacoplan or to any of the excipients
for initiation in patients with unresolved serious infection caused by encapsulated bacteria including

3ADVERSE REACTIONS

The following clinically significant adverse reactions are discussed in greater detail in other sections of the labeling:

Serious Infections Caused by Encapsulated Bacteria
Infusion-Related Reactions

3.1Clinical Trials Experience

Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice.

3.2Postmarketing Experience

The following adverse reactions have been identified during post-approval use of EMPAVELI. Because these reactions are reported voluntarily from a population of uncertain size, it is not always possible to reliably estimate their frequency or establish a causal relationship to EMPAVELI exposure.

Anaphylaxis and urticaria

4DESCRIPTION

EMPAVELI contains pegcetacoplan, a complement inhibitor. Pegcetacoplan is a symmetrical molecule comprised of two identical pentadecapeptides covalently bound to the ends of a linear 40-kiloDalton (kDa) PEG molecule. The peptide portions of pegcetacoplan contain 1-methyl-L-tryptophan (Trp(Me)) in position 4 and amino(ethoxyethoxy)acetic acid (AEEA) in position 14.

The molecular weight of pegcetacoplan is approximately 43.5 kDa. The molecular formula is C

EMPAVELI injection is a sterile, clear, colorless to slightly yellowish aqueous solution for subcutaneous use and is supplied in a 20-mL single-dose vial. Each 1 mL of solution contains 54 mg of pegcetacoplan, 41 mg of sorbitol, 0.384 mg of glacial acetic acid, 0.490 mg of sodium acetate trihydrate, and Water for Injection USP. EMPAVELI may also contain sodium hydroxide and/or additional glacial acetic acid for adjustment to a target pH of 5.

5PATIENT COUNSELING INFORMATION

Advise the patient to read the FDA-approved patient labeling (Medication Guide and Instructions for Use).

6PRINCIPAL DISPLAY PANEL - 1,080 mg/20 mL Vial Carton

NDC 73606-010-01

EMPAVELI

1,080 mg/20 mL

For Subcutaneous Infusion Only

Dispense the enclosed

One 20 mL Single-Dose Vial.

Rx only

PRINCIPAL DISPLAY PANEL - 1,080 mg/20 mL Vial Carton

7PRINCIPAL DISPLAY PANEL - 20 mL Injector Carton

EMPAVELI

20 mL

Single-use only

QTY 1

REF 30129903

Rx Only

PRINCIPAL DISPLAY PANEL - 20 mL Injector Carton