FGF23

Chr 12ADAR

fibroblast growth factor 23

Also known as: ADHR, FGFN, HFTC2, HPDR2, HYPF, PHPTC

FGF23 encodes a hormone that regulates phosphate homeostasis by inhibiting renal phosphate reabsorption and controlling vitamin D metabolism. Mutations cause autosomal dominant hypophosphatemic rickets or autosomal recessive hyperphosphatemic familial tumoral calcinosis, representing opposite ends of the phosphate regulation spectrum. The gene shows low constraint to loss-of-function variants (pLI 0.03, LOEUF 1.28), consistent with both loss-of-function and gain-of-function alleles causing distinct phosphate disorders.

Summary from RefSeq, OMIM, UniProt
Research Assistant →

Primary Disease Associations & Inheritance

Hypophosphatemic rickets, autosomal dominantMIM #193100
AD
Tumoral calcinosis, hyperphosphatemic, familial, 2MIM #617993
AR
12
Active trials
350
Pubs (1 yr)
88
P/LP submissions
15%
P/LP missense
1.28
LOEUF
Multiple*
Mechanism· predicted
Clinical SummaryFGF23
Population Constraint (gnomAD)
Low constraint (pLI 0.03) — loss-of-function variants are relatively tolerated in the population.
📋
ClinVar Variants
73 unique Pathogenic / Likely Pathogenic· 155 VUS of 319 total submissions
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Clinical Trials
12 active or recruiting trials — potential therapeutic options may be available
📖
GeneReview available — FGF23
Authoritative clinical overview · Recommended first read
Open GeneReview ↗

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Tolerant — LoF & missense variants common in population
LoF Constraint
1.28LOEUF
pLI 0.031
Z-score 1.14
OE 0.50 (0.231.28)
Tolerant

Highly tolerant — LoF variants common in population

Missense Constraint
0.40Z-score
OE missense 0.91 (0.801.04)
150 obs / 164.3 exp
Tolerant

Mild missense constraint

Observed / Expected Ratios
LoF OE0.50 (0.231.28)
00.351.4
Missense OE0.91 (0.801.04)
00.61.4
Synonymous OE0.78
01.21.6
LoF obs/exp: 3 / 6.0Missense obs/exp: 150 / 164.3Syn Z: 1.49
DN
0.6842th %ile
GOF
0.6737th %ile
LOF
0.3357th %ile

This gene has evidence for multiple mechanisms of pathogenicity (gain-of-function and dominant-negative). The Badonyi & Marsh model scores dominant-negative highest among its predictions, but genomic evidence (constraint, ClinVar variant spectrum, and literature) most strongly supports gain-of-function. Different variants in this gene may act through different mechanisms — interpret in context of the specific variant.

GOFprediction above median · 1 literature citation
DNprediction above median

Note: In-silico variant effect predictors (SIFT, PolyPhen, REVEL, CADD) may underestimate pathogenicity of missense variants in genes with GOF or DN mechanisms. Consider functional evidence and clinical context.

Literature Evidence

GOFAutosomal dominant hypophosphatemic rickets(ADHR)is caused by gain-of-function mutations in FGF23 that prevent its proteolytic cleavage.PMID:26813504

Predictions from Badonyi M, Marsh JA. PLoS ONE. 2024;19(8):e0307312.

ClinVar Variant Classifications

319 submitted variants in ClinVar

Classification Summary

Pathogenic61
Likely Pathogenic12
VUS155
Likely Benign58
Benign18
Conflicting14
61
Pathogenic
12
Likely Pathogenic
155
VUS
58
Likely Benign
18
Benign
14
Conflicting

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
0
6
55
0
61
Likely Pathogenic
1
5
6
0
12
VUS
3
106
41
5
155
Likely Benign
0
2
19
37
58
Benign
0
1
17
0
18
Conflicting
14
Total412013842318

LoF = frameshift, stop gained/lost, canonical splice · Counts from ClinVar esearch · Updated hourly

View in ClinVar →

Protein Context — Lollipop Plot

FGF23 · protein map & ClinVar variants

Showing all ClinVar variants across the protein. Search a specific variant to highlight its position.

Clinical Trials

Active and recruiting trials from ClinicalTrials.gov

Healthy Adults

Safety and Efficacy of Klotho and Follistatin Gene Therapy

RECRUITING
NCT07285629Phase EARLY_PHASE1MinicircleStarted 2025-12-16
Follistatin and klotho gene therapy
Healthy Adults

Safety and Efficacy of Injectable Klotho Plasmid Gene Therapy in Humans

RECRUITING
NCT07216781Phase PHASE1MinicircleStarted 2025-10-06
Injectable Plasmid Klotho Gene Therapy
X-Linked Hypophosphatemic Rickets

Effect of Burosumab on the Inflammatory Profile of Patients With X-linked Hypophosphatemic Rickets FLAM-XLH

NOT YET RECRUITING
NCT06248632Hospices Civils de LyonStarted 2024-04-01
Expression of inflammatory markers (Il6, Il8, Il1β, CXCL1, CCL2, CXCR3, Il1R, Il6R)
Hyperparathyroidism, PrimaryOsteoporosisParathyroid Neoplasms

The Bone-parathyroid Crosstalk in Primary Hyperparathyroidism

NOT YET RECRUITING
NCT05556499I.R.C.C.S Ospedale Galeazzi-Sant'AmbrogioStarted 2022-10
Biomarker assays
Obesity, Morbid

Different Limb Lengths in Gastric Bypass Surgery (SLIM) - Part 3: Metabolism and Inflammation

RECRUITING
NCT05471037Phase NAUniversity Hospital, Basel, SwitzerlandStarted 2022-08-01
Long Biliopancreatic Limb LRYGBShort Biliopancreatic Limb LRYGB
Kidney Disease, ChronicKidney Failure Chronic

Physical Exercise and Biomolecular Analysis to Reduce Uremic Toxins in Chronic Kidney Disease: An Exploratory Study

ENROLLING BY INVITATION
NCT06910475Phase NACatholic University of BrasíliaStarted 2025-04-01
Resistance trainingEndurance trainingConcurrent training
Fibrous Dysplasia of BoneMcCune Albright Syndrome

PAINDYS_Characterizing Pain in Fibrous Dysplasia of Bone/McCune-Albright Syndrome: an Exploratory Pilot Study

NOT YET RECRUITING
NCT07476768Phase NAUniversity Hospital, Clermont-FerrandStarted 2026-03-01
Visit 1 at the center
Ischemic Heart DiseaseChronic Coronary SyndromeOptical Coherence Tomography (OCT)

Evaluating the Role of IGF-1 and S-Klotho In Plaque Phenotype and Vulnerability: the VISION Study.

ACTIVE NOT RECRUITING
NCT06522074Fondazione Policlinico Universitario Agostino Gemelli IRCCSStarted 2024-10-15
Coronary angiography with OCT
Acute Coronary Syndromes (ACS)Iron DeficienciesElderly (People Aged 65 or More)

Effect of Intravenous Iron on Quality of Life in Older Patients With Acute Coronary Syndrome

RECRUITING
NCT07476859Phase PHASE4Fundación para la Investigación del Hospital Clínico de ValenciaStarted 2026-03-05
Ferric Carboxymaltose Injection [Ferinject]
Phosphate DiabetesX-linked Hypophosphatemia

Phosphorus-31 Spectroscopy in Phosphate Diabetes

NOT YET RECRUITING
NCT06921720Phase NAHospices Civils de LyonStarted 2025-05-01
intra-muscular ATP values in phosphate diabetesintra-muscular phosphate values in phosphate diabetes
Heart Failure

Acute Reno-Cardiac Action of Dapagliflozin In Advanced Heart Failure Patients on Heart Transplant Waiting List

RECRUITING
NCT06868797Phase NACentral Hospital, Nancy, FranceStarted 2025-08-29
Biological sample for the measurement of suPAR levels.
FGFR2 Gene Fusion/RearrangementOther Solid Tumors, Adult

A Study of Lirafugratinib in Non-CCA Solid Tumors With FGFR2 Fusion or Rearrangement

NOT YET RECRUITING
NCT07359820Phase PHASE2Elevar TherapeuticsStarted 2026-02
Lirafugratinib
Clinical Literature
Open Research Assistant →
Key Publications
Landmark & review papers · by relevance
PubMed
Rickets.
Carpenter TO et al.·Nat Rev Dis Primers
2017Review
Rickets guidance: part I-diagnostic workup.
Haffner D et al.·Pediatr Nephrol
2022Review
Approach to Hypophosphatemic Rickets.
Ackah SA et al.·J Clin Endocrinol Metab
2022
Klotho in Clinical Nephrology: Diagnostic and Therapeutic Implications.
Neyra JA et al.·Clin J Am Soc Nephrol
2020Review
Top 5 results · since 2015Search PubMed ↗