Bkemv (eculizumab-aeeb) / Amgen - LARVOL DELTA

A Cost Minimization and Budget Impact Analysis of an Eculizumab Biosimilar, ABP 959, From the Spanish Healthcare Perspective (ISPOR 2025) - "OBJECTIVES: To estimate the economic consequences of adopting ABP 959, an eculizumab biosimilar, for treating paroxysmal nocturnal haemoglobinuria (PNH) and atypical haemolytic uremic syndrome (aHUS). The CMA and BIA both demonstrated substantial estimated cost savings with ABP 959 compared with originator eculizumab and ravulizumab. The savings in drug acquisition costs offset the higher administration costs from more frequent administrations of ABP 959 compared with ravulizumab. These results support the rapid adoption of ABP 959 in treating PNH and aHUS."

HEOR • Atypical Hemolytic Uremic Syndrome • Complement-mediated Rare Disorders • Paroxysmal Nocturnal Hemoglobinuria

Comparative clinical efficacy and safety of biosimilar ABP 959 and eculizumab reference product in patients with paroxysmal nocturnal hemoglobinuria. (PubMed, Am J Hematol) - P3 | "ABP 959 is a biosimilar to the eculizumab reference product (RP), which is approved for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH). The results of this study in patients with PNH, along with previously demonstrated similarity of analytical, nonclinical, and clinical pharmacokinetics and pharmacodynamics in healthy volunteers support a demonstration of no clinically meaningful differences between ABP 959 and eculizumab RP. Clinical Trial Registration: NCT03818607."

Clinical • Journal • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

EVALUATION OF HEREDITARY FRUCTOSE TOLERANCE IN PREVALENT PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA, HEMOLYTIC UREMIC SYNDROME, OR MYASTHENIA GRAVIS (EHA 2024) - "Inclusion of sorbitol in ABP 959, an eculizumabbiosimilar, enhances the stability of ABP 959 when frozen, but may raise concern for patients with hereditaryfructose intolerance (HFI) who cannot metabolize sorbitol. By analyzing decades of patient medical records and claims data from the US, Germany and the UK, thelikelihood for a patient of having both HFI and eculizumab indicated conditions is very low. If HFI is suspected,a detailed symptom history should be collected prior to initiating treatment with biosimilar ABP 959."

Clinical • Atypical Hemolytic Uremic Syndrome • CNS Disorders • Complement-mediated Rare Disorders • Hematological Disorders • Myasthenia Gravis • Nephrology • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

FDA Approves First Interchangeable Biosimilar for Two Rare Diseases (PRNewswire) - "Today, the U.S. Food and Drug Administration approved Bkemv (eculizumab-aeeb) as the first interchangeable biosimilar to Soliris (eculizumab) to treat certain rare diseases. Bkemv is approved for the following treatment indications, which are also currently approved for Soliris: (i) the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH) to reduce hemolysis; and (ii) the treatment of patients with atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy."

FDA approval • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

Efficacy of Parallel and Crossover Analysis As Well As Pharmacokinetic Similarity Were Confirmed between ABP 959 and Eculizumab Reference Product in Patients with PNH (ASH 2023) - "Introduction: ABP 959, a biosimilar to eculizumab reference product (RP), binds to the human complement component 5 protein to inhibit terminal complement activation. Similarity in clinical efficacy in both parallel and crossover comparisons as determined by hemolysis in patients with PNH was established between ABP 959 and eculizumab RP. Additionally, analyses of serum total and unbound PK concentrations for patients with PNH further demonstrated PK similarity between ABP 959 and eculizumab RP. The results of this study, along with previously demonstrated analytical, non-clinical, clinical PK/PD in healthy adults, and efficacy and safety evaluations in PNH patients, further support a demonstration of no clinically meaningful differences between ABP 959 and eculizumab RP."

Clinical • PK/PD data • Atypical Hemolytic Uremic Syndrome • CNS Disorders • Complement-mediated Rare Disorders • Hematological Disorders • Myasthenia Gravis • Nephrology • Neuromyelitis Optica Spectrum Disorder • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

Functional similarity of ABP 959 and eculizumab in simulated serum models of aHUS and NMOSD. (PubMed, Ann Hematol) - "This work presents a full comparison of the effect of C5 inhibition across five complement functional assays. Using this approach to confirm functional similarity of ABP 959 with eculizumab RP contributes to the TOE for biosimilarity and provides support for extrapolation based on inhibition of C5 function to other rare disease indications approved for eculizumab RP."

Journal • Atypical Hemolytic Uremic Syndrome • CNS Disorders • Complement-mediated Rare Disorders • Hematological Disorders • Myasthenia Gravis • Nephrology • Neuromyelitis Optica Spectrum Disorder • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

SENSITIVITY AND SUBGROUP ANALYSES FURTHER SUPPORT CLINICAL SIMILARITY IN EFFICACY BETWEEN ABP 959 AND ECULIZUMAB REFERENCE PRODUCT IN PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) (EHA 2023) - "Background: ABP 959, a biosimilar to eculizumab reference product (RP), binds to the human complement C5 protein to inhibit progression of the complement cascades. These additional analyses support the findings of the key primary efficacy analysis between ABP 959 and RP inpatients with PNH. The results of this study, along with previously demonstrated analytical, non-clinical, and clinical similarity, validate the robustness of similarity in clinical efficacy and are consistent with the conclusion of no clinically meaningful differences between ABP 959 and eculizumab RP. Paroxysmal nocturnal hemoglobinuria (PNH), PNH"

Clinical • Atypical Hemolytic Uremic Syndrome • CNS Disorders • Complement-mediated Rare Disorders • Gastroenterology • Hematological Disorders • Hepatology • Infectious Disease • Myasthenia Gravis • Nephrology • Neuromyelitis Optica Spectrum Disorder • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases • Respiratory Diseases

Efficacy and Safety of Biosimilar ABP 959 Compared with Reference Eculizumab in Adults with PNH (BSH 2023) - "Introduction : ABP 959, a biosimilar to eculizumab reference product (RP), binds to the human complement component 5 protein to inhibit the progression of the complement cascades. Additionally, PK, immunogenicity, and safety were similar between ABP 959 and RP. The results of this study, along with previously demonstrated analytical, non-clinical, PK, and pharmacodynamics similarity, support a demonstration of no clinically meaningful differences between ABP 959 and RP."

Clinical • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases • HP

Efficacy and Safety of Biosimilar Candidate ABP 959 As Compared with Eculizumab Reference Product in Paroxysmal Nocturnal Hemoglobinuria (ASH 2022) - "Similarity in clinical efficacy was established between ABP 959 and eculizumab RP in the primary endpoint of hemolysis, measured by LDH at week 27. Additionally, comparable safety, PK, and immunogenicity results support the conclusion of clinical similarity between ABP 959 and eculizumab RP."

Clinical • Atypical Hemolytic Uremic Syndrome • CNS Disorders • Complement-mediated Rare Disorders • Fatigue • Hematological Disorders • Myasthenia Gravis • Nephrology • Neuromyelitis Optica Spectrum Disorder • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases • HP

DAHLIA: A Study Evaluating the Efficacy and Safety of ABP 959 Compared With Eculizumab in Adult Participants With PNH (clinicaltrials.gov) - P3 | N=42 | Completed | Sponsor: Amgen | Active, not recruiting ➔ Completed

Trial completion • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases • HP

PHARMACOKINETIC AND PHARMACODYNAMIC SIMILARITY OF ABP 959 AND ECULIZUMAB REFERENCE PRODUCT: UNBOUND ECULIZUMAB AND CH50 FROM THE RANDOMIZED, DOUBLE-BLIND, SINGLE-DOSE STUDY IN HEALTHY VOLUNTEERS (EHA 2022) - "Background ABP 959 is being developed as a biosimilar to eculizumab reference product (RP). Conclusion These additional clinical pharmacology results demonstrate bioequivalence of ABP 959 to eculizumab RP with regard to unbound eculizumab and CH50. These results further support the PK/PD similarity of ABP 959 with eculizumab RP."

Clinical • PK/PD data • Atypical Hemolytic Uremic Syndrome • CNS Disorders • Complement-mediated Rare Disorders • Hematological Disorders • Myasthenia Gravis • Nephrology • Neuromyelitis Optica Spectrum Disorder • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

DAHLIA: A Study Evaluating the Efficacy and Safety of ABP 959 Compared With Eculizumab in Adult Participants With PNH (clinicaltrials.gov) - P3 | N=42 | Active, not recruiting | Sponsor: Amgen | Trial primary completion date: Jan 2022 ➔ Jul 2022

Trial primary completion date • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases • HP

Analytical Similarity Assessment of ABP 959 in Comparison with Eculizumab Reference Product. (PubMed, BioDrugs) - "Based on these results, it can be concluded that ABP 959 is analytically similar to eculizumab RP."

Journal • Atypical Hemolytic Uremic Syndrome • CNS Disorders • Complement-mediated Rare Disorders • Hematological Disorders • Myasthenia Gravis • Nephrology • Neuromyelitis Optica Spectrum Disorder • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

DAHLIA: A Study Evaluating the Efficacy and Safety of ABP 959 Compared With Eculizumab in Adult Participants With PNH (clinicaltrials.gov) - P3; N=42; Active, not recruiting; Sponsor: Amgen; Recruiting ➔ Active, not recruiting

Clinical • Enrollment closed • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases • HP

DAHLIA: A Study Evaluating the Efficacy and Safety of ABP 959 Compared With Eculizumab in Adult Participants With PNH (clinicaltrials.gov) - P3; N=40; Recruiting; Sponsor: Amgen; Trial completion date: Aug 2021 ➔ Mar 2022; Trial primary completion date: Feb 2021 ➔ Sep 2021

Clinical • Trial completion date • Trial primary completion date

A Randomized, Double-Blind, Single-Dose, Three-Arm, Parallel Group Study to Determine Pharmacokinetic Similarity of ABP 959 and Eculizumab (Soliris ) in Healthy Male Subjects. (PubMed, Eur J Haematol) - "This study demonstrated PK and PD similarity of ABP 959 to eculizumab RP; safety and immunogenicity profiles were also similar."

Clinical • Journal • PK/PD data

DAHLIA: A Study Evaluating the Efficacy and Safety of ABP 959 Compared With Eculizumab in Adult Participants With PNH (clinicaltrials.gov) - P3; N=40; Recruiting; Sponsor: Amgen; Trial primary completion date: Nov 2020 ➔ Feb 2021

Trial primary completion date • HP

Relationship between Pharmacokinetics and Antidrug Antibody Status of ABP 959, a Biosimilar Candidate to Eculizumab: Results from a Pharmacokinetic Similarity Study (ASH 2019) - "The incidence of ADAs was similar between ABP 959 and eculizumab RP and did not impact the overall PK similarity assessment."

PK/PD data

PHARMACOKINETIC AND PHARMACODYNAMIC SIMILARITY OF ABP 959 WITH ECULIZUMAB: RESULTS FROM A RANDOMIZED, DOUBLE-BLIND, SINGLE-DOSE, PARALLEL-GROUP STUDY IN HEALTHY SUBJECTS (EHA 2019) - "No subjects developed neutralizing antibodies.Conclusion This phase 1 study demonstrated PK and PD bioequivalence of ABP 959 to eculizumab RP. Safety and immunogenicity profiles were similar between ABP 959 and eculizumab RP."

Clinical • PK/PD data

DAHLIA: A Study Evaluating the Efficacy and Safety of ABP 959 Compared With Eculizumab in Adult Participants With PNH (clinicaltrials.gov) - P3; N=40; Recruiting; Sponsor: Amgen; Not yet recruiting ➔ Recruiting

Clinical • Enrollment open

DAHLIA: A Study Evaluating the Efficacy and Safety of ABP 959 Compared With Eculizumab in Adult Participants With PNH (clinicaltrials.gov) - P3; N=40; Not yet recruiting; Sponsor: Amgen; Trial primary completion date: Feb 2021 ➔ Jul 2020

Clinical • Trial primary completion date

DAHLIA: A Study Evaluating the Efficacy and Safety of ABP 959 Compared With Eculizumab in Adult Participants With PNH (clinicaltrials.gov) - P3; N=40; Not yet recruiting; Sponsor: Amgen

Clinical • New P3 trial