Dosage and administration of anti-C5 antibodies for treatment of paroxysmal nocturnal hemoglobinuria (PNH) in pediatric patients
Inventors
PAYTON, Lori • ROTTINGHAUS, Scott T. • Pradhan, Rajendra • Ortiz, Stephan • OGAWA, Masayo • Gao, Xiang
Assignees
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Publication Number
US-12404320-B2
Publication Date
2025-09-02
Expiration Date
Abstract
Provided are methods for clinical treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) in pediatric patients using an anti-C5 antibody, or antigen binding fragment thereof.
Core Innovation
The invention relates to a method of treating a human pediatric patient with Paroxysmal Nocturnal Hemoglobinuria (PNH) by administering an anti-C5 antibody. The anti-C5 antibody comprises heavy and light chains with amino acid sequences shown in SEQ ID NOs:14 and 11, respectively, and is administered intravenously as part of a pediatric treatment regimen intended to inhibit complement activity associated with PNH.
The method includes administering the anti-C5 antibody once on Day 1 followed by maintenance dosing on Day 15 and every four weeks thereafter or every eight weeks thereafter. The specific Day 1 and Day 15 follow-on doses are weight-based using defined pediatric weight bands, providing an anti-C5 dosing schedule across pediatric patients and supporting continued administration after the initial dose.
The disclosure specifies associated antibody properties and treatment-related assessment approaches. The antibody is described with binding to human C5 and includes defined binding affinity constraints at specified pH and temperature conditions, and serum pharmacokinetic monitoring such as a serum trough concentration threshold. Treatment outcomes and response are evaluated using complement inhibition and hemolysis related measures, including lactate dehydrogenase (LDH) and other clinical laboratory and quality of life assessments.
Claims Coverage
The partial set provides one independent claim directed to an anti-C5 antibody treatment method for pediatric PNH, with main inventive features centered on specific heavy/light chain sequence-defined antibody administration and a weight-based intravenous Day 1 and Day 15 maintenance dosing schedule with either every-4-weeks or every-8-weeks follow-on dosing.
Sequence-defined anti-C5 antibody for pediatric PNH treatment
Administering to a human pediatric patient with Paroxysmal Nocturnal Hemoglobinuria (PNH) an anti-C5 antibody comprising heavy and light chains comprising the amino acid sequences shown in SEQ ID NOs:14 and 11, respectively.
Intravenous weight-based Day 1 and maintenance dosing regimen
Administering the anti-C5 antibody intravenously once on Day 1 using weight-based doses, and administering on Day 15 and every four weeks thereafter or on Day 15 and every eight weeks thereafter using weight-based follow-on doses.
Complement inhibition and hemolysis/clinical response assessment
Further comprising determining whether a treated patient shows terminal complement inhibition and reduced hemolysis by lactate dehydrogenase (LDH) levels versus baseline and/or at least one therapeutic effect versus baseline.
The coverage is anchored in a specific sequence-defined anti-C5 antibody and a pediatric weight-band intravenous regimen with Day 1 loading and Day 15 maintenance given on either a four-week or eight-week schedule. The claim-derived refinements further extend to monitoring thresholds and to evaluating complement inhibition and hemolysis-related clinical laboratory outcomes versus baseline.
Stated Advantages
Not explicitly described in patent.
Documented Applications
Not explicitly described in patent.
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