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Medications for Primary Hyperoxaluria

Other names: Oxalosis; PH1

Primary hyperoxaluria type 1 (PH1) is an ultra-rare genetic disease characterized by oxalate overproduction.

PH1 is an autosomal recessive disorder of glyoxylate metabolism, where hepatic detoxification of glyoxylate is impaired due to mutation of the AGXT gene, which encodes the liver peroxisomal alanine-glyoxylate aminotransferase (AGT) enzyme, resulting in excessive oxalate production. The excess production of oxalate results in the deposition of calcium oxalate crystals in the kidneys and urinary tract and can lead to the formation of painful and recurrent kidney stones which can progress to kidney failure.

PH1 is associated with a progressive decline in kidney function, which exacerbates the disease as the excess oxalate can no longer be effectively excreted, resulting in subsequent accumulation and deposition of oxalate in bones, eyes, skin, and heart, leading to severe illness and death.

Management options for PH1 include hyperhydration, crystallization inhibitors and, in a minority of patients with a specific genotype, pyridoxine (vitamin B6). These measures help to delay inevitable progression to kidney failure.

Oxlumo (lumasiran) is the the first FDA approved therapy for the treatment of PH1. It works by reducing the hepatic levels of the GO enzyme, thereby depleting the substrate necessary for oxalate production.

Drugs used to treat Primary Hyperoxaluria

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

Drug name Rating Reviews Activity ? Rx/OTC Pregnancy CSA Alcohol
Oxlumo Rate Add review
Rx N

Generic name: lumasiran systemic

Drug class: miscellaneous metabolic agents

For consumers: dosage, side effects

For professionals: Prescribing Information

lumasiran Rate Add review
Rx N

Generic name: lumasiran systemic

Brand name:  Oxlumo

Drug class: miscellaneous metabolic agents

For consumers: dosage, side effects

For professionals: AHFS DI Monograph

Legend

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.
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.