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Medications for X-Linked Hypophosphatemia

About X-Linked Hypophosphatemia: 

X-linked hypophosphatemia (XLH) is a genetic disorder which occurs when there are mutations to a gene on the X chromosome.  The gene is called PHEX (Phosphate Regulating Endopeptidase Homolog X-Linked) gene and has a in role keeping a proper balance of phosphate in the body.

This gene mutation causes an increase of a protein called fibroblast growth factor 23 (FGF23) which decreases the kidneys’ ability to reabsorb phosphate into blood stream and causes an increase of phosphate in the urine (phosphaturia) and therefore this results in a decrease of phosphate blood levels (hypophosphatemia).

As phosphate is important for normal bone and teeth formation the symptoms of  X-linked hypophosphatemia include bone pain, skeletal deformities, short stature, dental abscesses, cavities and enamel problems.

Drugs Used to Treat X-Linked Hypophosphatemia

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

Drug name Rx / OTC Pregnancy CSA Alcohol Reviews Rating Popularity
Crysvita N Add review Rate

Generic name: burosumab systemic

Drug class: miscellaneous metabolic agents

For consumers: dosage, interactions, side effects

For professionals: AHFS DI Monograph, Prescribing Information

burosumab N Add review Rate

Generic name: burosumab systemic

Brand name:  Crysvita

Drug class: miscellaneous metabolic agents

For consumers: dosage, interactions,

For professionals: A-Z Drug Facts, AHFS DI Monograph

Legend

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.
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
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.

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Further information

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

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