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Medications for Paroxysmal Nocturnal Hemoglobinuria

Other names: PNH

Common paroxysmal nocturnal hemoglobinuria medications are Solaris (eculizumab), Ultomiris (ravulizumab), and Empaveli (pegcetacoplan), which are used to reduce the breakdown of red blood cells.

Paroxysmal nocturnal hemoglobinuria is a rare, blood cell abnormality in which red blood cells break down earlier than normal. The red cells leak hemoglobin into the blood, which can pass into the urine.

Drugs used to treat Paroxysmal Nocturnal Hemoglobinuria

The following list of medications are in some way related to or used in the treatment of this condition.

Filter
Drug name Rating Reviews Activity ? Rx/OTC Pregnancy CSA Alcohol
Soliris 5.0 1 review for Soliris to treat Paroxysmal Nocturnal Hemoglobinuria
Rx C N
Generic name:
eculizumab systemic
Drug class:
selective immunosuppressants
For consumers:
dosage, interactions, side effects
For professionals:
Prescribing Information
Ultomiris Rate Add review
Rx N
Generic name:
ravulizumab systemic
Drug class:
selective immunosuppressants
For consumers:
dosage, interactions, side effects
For professionals:
Prescribing Information
eculizumab 5.0 1 review for eculizumab to treat Paroxysmal Nocturnal Hemoglobinuria
Rx C N
Generic name:
eculizumab systemic
Brand name:
Soliris
Drug class:
selective immunosuppressants
For consumers:
dosage, interactions, side effects
For professionals:
AHFS DI Monograph
Empaveli Rate Add review
Rx N
Generic name:
pegcetacoplan systemic
Drug class:
selective immunosuppressants
For consumers:
dosage, interactions, side effects
For professionals:
Prescribing Information
pegcetacoplan Rate Add review
Rx N
Generic name:
pegcetacoplan systemic
Brand name:
Empaveli
Drug class:
selective immunosuppressants
For consumers:
dosage, interactions, side effects
For professionals:
AHFS DI Monograph
ravulizumab Rate Add review
Rx N
Generic name:
ravulizumab systemic
Brand name:
Ultomiris
Drug class:
selective immunosuppressants
For consumers:
dosage, interactions, side effects
For professionals:
AHFS DI Monograph
Fabhalta Rate Add review
Rx N
Generic name:
iptacopan systemic
Drug class:
selective immunosuppressants
For consumers:
dosage, interactions,
For professionals:
Prescribing Information
iptacopan Rate Add review
Rx N
Generic name:
iptacopan systemic
Brand name:
Fabhalta
Drug class:
selective immunosuppressants
For consumers:
interactions,
For professionals:
AHFS DI Monograph

Frequently asked questions

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Rating For ratings, users were asked how effective they found the medicine while considering positive/adverse effects and ease of use (1 = not effective, 10 = most effective).
Activity Activity is based on recent site visitor activity relative to other medications in the list.
Rx Prescription only.
OTC Over-the-counter.
Rx/OTC Prescription or Over-the-counter.
Off-label This medication may not be approved by the FDA for the treatment of this condition.
EUA An Emergency Use Authorization (EUA) allows the FDA to authorize unapproved medical products or unapproved uses of approved medical products to be used in a declared public health emergency when there are no adequate, approved, and available alternatives.
Expanded Access Expanded Access is a potential pathway for a patient with a serious or immediately life-threatening disease or condition to gain access to an investigational medical product (drug, biologic, or medical device) for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available.
Pregnancy Category
A Adequate and well-controlled studies have failed to demonstrate a risk to the fetus in the first trimester of pregnancy (and there is no evidence of risk in later trimesters).
B Animal reproduction studies have failed to demonstrate a risk to the fetus and there are no adequate and well-controlled studies in pregnant women.
C Animal reproduction studies have shown an adverse effect on the fetus and there are no adequate and well-controlled studies in humans, but potential benefits may warrant use in pregnant women despite potential risks.
D There is positive evidence of human fetal risk based on adverse reaction data from investigational or marketing experience or studies in humans, but potential benefits may warrant use in pregnant women despite potential risks.
X Studies in animals or humans have demonstrated fetal abnormalities and/or there is positive evidence of human fetal risk based on adverse reaction data from investigational or marketing experience, and the risks involved in use in pregnant women clearly outweigh potential benefits.
N FDA has not classified the drug.
Controlled Substances Act (CSA) Schedule
M The drug has multiple schedules. The schedule may depend on the exact dosage form or strength of the medication.
U CSA Schedule is unknown.
N Is not subject to the Controlled Substances Act.
1 Has a high potential for abuse. Has no currently accepted medical use in treatment in the United States. There is a lack of accepted safety for use under medical supervision.
2 Has a high potential for abuse. Has a currently accepted medical use in treatment in the United States or a currently accepted medical use with severe restrictions. Abuse may lead to severe psychological or physical dependence.
3 Has a potential for abuse less than those in schedules 1 and 2. Has a currently accepted medical use in treatment in the United States. Abuse may lead to moderate or low physical dependence or high psychological dependence.
4 Has a low potential for abuse relative to those in schedule 3. It has a currently accepted medical use in treatment in the United States. Abuse may lead to limited physical dependence or psychological dependence relative to those in schedule 3.
5 Has a low potential for abuse relative to those in schedule 4. Has a currently accepted medical use in treatment in the United States. Abuse may lead to limited physical dependence or psychological dependence relative to those in schedule 4.
Alcohol
X Interacts with Alcohol.

Further information

Always consult your healthcare provider to ensure the information displayed on this page applies to your personal circumstances.