Medications for Paroxysmal Nocturnal Hemoglobinuria (PNH)
These are drugs that have been approved by the US Food and Drug Administration (FDA), meaning they have been determined to be safe and effective for use in Paroxysmal Nocturnal Hemoglobinuria (PNH).
Found 6 Approved Drugs for Paroxysmal Nocturnal Hemoglobinuria (PNH)
Eculizumab
Brand Names
Soliris, BKEMV, Epysqli
Eculizumab
Brand Names
Soliris, BKEMV, Epysqli
Form: Injection
Method of administration: Intravenous
FDA approval date: April 02, 2007
Classification: Complement Inhibitor
EPYSQLI is a complement inhibitor indicated for: the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH) to reduce hemolysis.
Ultomiris
Generic Name
Ravulizumab
Ultomiris
Generic Name
Ravulizumab
Form: Solution
Method of administration: Intravenous
FDA approval date: December 21, 2018
Classification: Complement Inhibitor
ULTOMIRIS is indicated for: the treatment of adult and pediatric patients one month of age and older with paroxysmal nocturnal hemoglobinuria (PNH). the treatment of adults and pediatric patients one month of age and older with atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy (TMA). ULTOMIRIS is a complement inhibitor indicated for: the treatment of adult and pediatric patients one month of age and older with paroxysmal nocturnal hemoglobinuria (PNH) ( 1 ). the treatment of adults and pediatric patients one month of age and older with atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy (TMA) ( 1 ). Limitations of Use: ULTOMIRIS is not indicated for the treatment of patients with Shiga toxin E. coli related hemolytic uremic syndrome (STEC-HUS). Limitations of Use: ULTOMIRIS is not indicated for the treatment of patients with Shiga toxin E. coli related hemolytic uremic syndrome (STEC-HUS).
Pegcetacoplan
Brand Names
Empaveli, Syfovre
Pegcetacoplan
Brand Names
Empaveli, Syfovre
Form: Injection
Method of administration: Subcutaneous, Intravitreal
FDA approval date: May 14, 2021
Classification: Complement Inhibitor
EMPAVELI™ is indicated for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH). EMPAVELI is a complement inhibitor indicated for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH). ( 1 )
Fabhalta
Generic Name
Iptacopan
Fabhalta
Generic Name
Iptacopan
Form: Capsule
Method of administration: Oral
FDA approval date: December 05, 2023
Classification: Complement Factor B Inhibitor
FABHALTA is a complement factor B inhibitor, indicated for: the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH).
Piasky
Generic Name
Crovalimab
Piasky
Generic Name
Crovalimab
Form: Injection
Method of administration: Subcutaneous
FDA approval date: June 20, 2024
PIASKY is indicated for the treatment of adult and pediatric patients 13 years and older with paroxysmal nocturnal hemoglobinuria (PNH) and body weight of at least 40 kg. PIASKY is a complement C5 inhibitor indicated for the treatment of adult and pediatric patients 13 years and older with paroxysmal nocturnal hemoglobinuria (PNH) and body weight of at least 40 kg ( 1 )
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