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Identification
- Summary
Migalastat is an alpha-galactosidase A chaperone used for the treatment of Fabry disease in patients with an amenable galactosidase alpha gene (GLA) variant.
- Brand Names
- Galafold
- Generic Name
- Migalastat
- DrugBank Accession Number
- DB05018
- Background
Fabry disease is a rare, progressive genetic disorder characterized by a defective GLA gene that causes a deficiency in the enzyme alpha-Galactosidase A (alpha-Gal A). This enzyme is responsible for breaking down glycosphingolipid substrate that, when deficient in patients with Fabry disease, builds up in the blood vessels, the kidneys, the nerves, the heart, and other organs.5,6,3,4 In the U.S., it is estimated that more than 3,000 people are living with Fabry disease, and an estimated more than 50 percent of these diagnosed patients are currently untreated.3
Migalastat (approved and sold under Amicus Therapeutics' brand name Galafold) is subsequently an oral pharmacological chaperone of alpha-Gal A for the treatment of Fabry disease in adults who have amenable GLA variants.5,6,3,4 In these patients, migalastat works by stabilizing the body’s dysfunctional alpha-Gal A enzyme so that it can clear the accumulation of glycosphingolipid disease substrate.5,6,3,4 Globally, it is estimated that approximately 35 to 50 percent of Fabry patients may have amenable GLA variants that are treatable with migalastat. 3
Given the rarity of Fabry disease and the proportion of Fabry disease patients that could benefit from migalastat therapy, Amicus Therapeutics' brand name Galafold was approved using the Accelerated Approval pathway, under which the FDA may approve drugs for serious conditions where there is an unmet medical need and where a drug is shown to have certain effects that are reasonably likely to predict a clinical benefit to patients.5,6,3,4 A further study is required to verify and describe the clinical benefits of Galafold, and the sponsor will be conducting a confirmatory clinical trial of Galafold in adults with Fabry disease.5,6,3,4
Additionally, Galafold was also granted Priority Review designation, under which the FDA’s goal is to take action on an application within six months of application filing where the agency determines that the drug if approved, would provide a significant improvement in treating, diagnosing or preventing a serious condition over available therapies.5,6 Galafold also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.5,6
As of August 2018, migalastat under Amicus Therapeutics' brand name Galafold is currently approved in Australia, Canada, European Union, Israel, Japan, South Korea, Switzerland, and the United States.3
- Type
- Small Molecule
- Groups
- Approved, Investigational
- Structure
Structure for Migalastat (DB05018)
- Weight
- Average: 163.1717
Monoisotopic: 163.084457909 - Chemical Formula
- C6H13NO4
- Synonyms
- 1-Deoxygalactonojirimycin
- 1-Deoxygalactostatin
- Migalastat
Pharmacology
- Indication
Migalastat is approved by the FDA for the treatment of adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene (GLA) variant based on in vitro assay data.5
This indication is approved under accelerated approval based on a reduction in kidney interstitial capillary cell globotriaosylceramide (KIC GL-3) substrate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.5
Migalastat is also approved by the EMA and Health Canada to treat the same disease, although it is approved for both adults and adolescents aged 16 years and older in Europe.6,7
Reduce drug development failure ratesBuild, train, & validate machine-learning modelswith evidence-based and structured datasets.Build, train, & validate predictive machine-learning models with structured datasets.- Associated Conditions
Indication Type Indication Combined Product Details Approval Level Age Group Patient Characteristics Dose Form Treatment of Fabry disease •••••••••••• ••••• ••••••••••••••••••• ••••••• •••••••• ••••••• Treatment of Fabry disease •••••••••••• ••••• ••••••••••••••••••• ••••••• •••••••• ••••••• Treatment of Fabry disease •••••••••••• ••••••••••• ••••• ••••••••••••••••••• ••••••• •••••••• ••••••• - Contraindications & Blackbox Warnings
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In general, treatment in patients with migalastat in Phase 2 pharmacodynamic trials resulted in increases in endogenous alpha-galactosidase (alpha-Gal A) activity in white blood cells, as well as in skin and kidney for the majority of patients Label. In patients with amenable galactosidase alpha gene (GLA) mutations, globotriaosylceramide (GL-3) levels tended to decrease in the urine and in the kidney interstitial capillaries.6
In an in vitro assay (HEK-293 assay), Human Embryonic Kidney (HEK-293) cell lines were transfected with specific GLA variants (mutations) which produced mutant alpha-Gal A proteins. In the transfected cells, amenability of the GLA variants was assessed after a 5-day incubation with 10 micromol/L migalastat. A GLA variant was categorized as amenable if the resultant mutant alpha-Gal A activity (measured in the cell lysates) met two criteria: 1) it showed a relative increase of at least 20% compared to the pre-treatment alpha-Gal A activity, and 2) it showed an absolute increase of at least 3% of the wild-type (normal) alpha-Gal activity. The in vitro assay did not evaluate the trafficking of the mutant alpha-Gal A proteins into the lysosome or the dissociation of migalastat from the mutant alpha-Gal A proteins within the lysosome. Also, the in vitro assay did not test whether a GLA variant causes Fabry disease or not.5
- Mechanism of action
Fabry disease is a progressive X-linked lysosomal storage disorder that affects males and females Label. Fabry disease-causing mutations occur in the galactosidase alpha (GLA) gene and result in a deficiency of the lysosomal enzyme alpha-galactosidase A (alpha-Gal A) that is required for glycosphingolipid substrate (GL-3 and lyso-Gb3) metabolism Label. Reduced alpha-Gal A activity is, therefore, associated with the progressive accumulation of glycosphingolipid substrate in vulnerable organs and tissues, which ultimately leads to the morbidity and mortality associated with Fabry disease.6
Migalastat is a pharmacological chaperone that reversibly binds to the active site of the alpha-galactosidase A (alpha-Gal A) protein (encoded by the galactosidase alpha gene, GLA), which is deficient in Fabry disease. This binding stabilizes alpha-Gal A allowing its trafficking from the endoplasmic reticulum into the lysosome where it exerts its action. In the lysosome, at a lower pH and at a higher concentration of relevant substrates, migalastat dissociates from alpha-Gal A allowing it to break down the glycosphingolipids globotriaosylceramide (GL-3) and globotriaosylsphingosine (lyso-Gb3). Certain GLA variants (mutations) causing Fabry disease result in the production of abnormally folded and less stable forms of the alpha-Gal A protein which, however, retain enzymatic activity. Those GLA variants, referred to as amenable variants, produce alpha-Gal A proteins that may be stabilized by migalastat thereby restoring their trafficking to lysosomes and their intralysosomal activity.5
The GLA mutations that are amenable and not amenable to treatment with migalastat are regularly maintained and updated on online sites that are readily accessible by healthcare providers.5
Target Actions Organism AAlpha-galactosidase A stabilizationHumans - Absorption
With absorption occurring largely in the gut, the absolute bioavailability (AUC) for a single oral 150 mg migalastat hydrochloride dose or a single 2-hour 150 mg intravenous infusion was approximately 75% and Tmax was approximately 3 hours.2,6 Plasma migalastat exposure (AUC0-∞) and Cmax demonstrated dose-proportional increases at migalastat hydrochloride oral doses from 50 mg to 1,250 mg (doses from 0.5 to 8.3-fold of the approved recommended dosage).5,6
Migalastat administered with a high-fat meal (850 calories; 56% from fat), or 1 hour before a high-fat or light meal (507 calories; 30% from fat), or 1 hour after a light meal, resulted in significant reductions of 37% to 42% in mean total migalastat exposure (AUC0-∞) and reductions of 15% to 39% in mean peak migalastat exposure (Cmax) compared with the fasting state.5
- Volume of distribution
In healthy volunteers, the volume of distribution (Vz/F) of migalastat following ascending single oral doses (25-675 mg migalastat HCl) ranged from 77 to 133 L, indicating it is well distributed into tissues and greater than total body water (42 liters).6
- Protein binding
There was no detectable plasma protein binding following administration of [14C]-migalastat hydrochloride in the concentration range between 1 and 100 µM.5,6
- Metabolism
Based upon in vivo data, migalastat is a substrate for uridine diphosphate glucuronosyltransferase (otherwise known as UGT or UDPGT), being a minor elimination pathway.5
- Route of elimination
In a mass balance study in healthy male subjects, following oral administration of 123 mg [14C]-migalastat, approximately 77% of the total radiolabeled dose was recovered in urine and 20% of the total radiolabeled dose was recovered in feces with an overall total recovery of 98% within 96 hours post-dose. In urine, unchanged migalastat accounted for 80% of the radioactivity, which equates to 62% of the administered dose. In feces, unchanged migalastat was the only drug-related component. In plasma, unchanged migalastat accounted for approximately 77% of the plasma radioactivity, and three dehydrogenated O-glucuronide conjugated metabolites, M1 to M3, together accounted for approximately 13% of the plasma radioactivity, none of which comprised more than 6% of the radiolabeled dose. Approximately 9% of the total radioactivity in plasma was unassigned.5
- Half-life
The mean elimination half-life (t1/2) of migalastat ranges from approximately 3 to 5 hours for a single oral dose of 150 mg.6 For the dose of 123 mg, the mean elimination half-life was estimated to be 4 hours.5
- Clearance
Following ascending single oral doses (25-675 mg migalastat hydrochloride), no trends were found for clearance (CL/F). At the 150 mg dose, CL/F was approximately 11 to 14 L/hr, while at 123 mg, the apparent clearance was calculated to be 12.5 L/hr.6,5
- Adverse Effects
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The most common adverse reactions reported with migalastat (≥ 10%) during the 6-month placebo-controlled, double-blind phase of its Study 1 clinical studies were headache, nasopharyngitis, urinary tract infection, nausea, and pyrexia Label.
In case of overdose, general medical care is recommended Label. Headache and dizziness were the most common adverse reactions reported at doses of migalastat of up to 1250 mg and 2000 mg, respectively Label.
- Pathways
- Not Available
- Pharmacogenomic Effects/ADRs
- Not Available
Interactions
- Drug Interactions
- This information should not be interpreted without the help of a healthcare provider. If you believe you are experiencing an interaction, contact a healthcare provider immediately. The absence of an interaction does not necessarily mean no interactions exist.
Drug Interaction Integrate drug-drug
interactions in your softwareAbacavir Abacavir may decrease the excretion rate of Migalastat which could result in a higher serum level. Aceclofenac Aceclofenac may decrease the excretion rate of Migalastat which could result in a higher serum level. Acemetacin Acemetacin may decrease the excretion rate of Migalastat which could result in a higher serum level. Acetaminophen Acetaminophen may decrease the excretion rate of Migalastat which could result in a higher serum level. Acetazolamide Acetazolamide may increase the excretion rate of Migalastat which could result in a lower serum level and potentially a reduction in efficacy. - Food Interactions
- Avoid caffeine. Co-administration of GALAFOLD with caffeine decreases migalastat AUC and Cmax which may reduce its efficacy.
- Take at the same time every day.
- Take on an empty stomach. Avoid eating for at least two hours before and after taking migalastat.
Products
Drug product information from 10+ global regionsOur datasets provide approved product information including:dosage, form, labeller, route of administration, and marketing period.Access drug product information from over 10 global regions.- Product Ingredients
Ingredient UNII CAS InChI Key Migalastat hydrochloride CLY7M0XD20 75172-81-5 ZJIHMALTJRDNQI-OLALXQGDSA-N - International/Other Brands
- Amigal
- Brand Name Prescription Products
Name Dosage Strength Route Labeller Marketing Start Marketing End Region Image Galafold Capsule 123 mg Oral Amicus Therapeutics Canada Inc. 2018-01-15 Not applicable Canada 
Galafold Capsule 123 mg/1 Oral Amicus Therapeutics US, LLC 2018-08-10 Not applicable US 
Galafold Capsule 123 mg Oral Amicus Therapeutics Europe Limited 2020-12-22 Not applicable EU 
Categories
- ATC Codes
- A16AX14 — Migalastat
- Drug Categories
- Chemical TaxonomyProvided by Classyfire
- Description
- This compound belongs to the class of organic compounds known as piperidines. These are compounds containing a piperidine ring, which is a saturated aliphatic six-member ring with one nitrogen atom and five carbon atoms.
- Kingdom
- Organic compounds
- Super Class
- Organoheterocyclic compounds
- Class
- Piperidines
- Sub Class
- Not Available
- Direct Parent
- Piperidines
- Alternative Parents
- Secondary alcohols / 1,2-aminoalcohols / Polyols / Dialkylamines / Azacyclic compounds / Primary alcohols / Organopnictogen compounds / Hydrocarbon derivatives
- Substituents
- 1,2-aminoalcohol / Alcohol / Aliphatic heteromonocyclic compound / Amine / Azacycle / Hydrocarbon derivative / Organic nitrogen compound / Organic oxygen compound / Organonitrogen compound / Organooxygen compound
- Molecular Framework
- Aliphatic heteromonocyclic compounds
- External Descriptors
- Not Available
- Affected organisms
- Humans and other mammals
Chemical Identifiers
- UNII
- C4XNY919FW
- CAS number
- 108147-54-2
- InChI Key
- LXBIFEVIBLOUGU-DPYQTVNSSA-N
- InChI
- InChI=1S/C6H13NO4/c8-2-3-5(10)6(11)4(9)1-7-3/h3-11H,1-2H2/t3-,4+,5+,6-/m1/s1
- IUPAC Name
- (2R,3S,4R,5S)-2-(hydroxymethyl)piperidine-3,4,5-triol
- SMILES
- OC[C@H]1NC[C@H](O)[C@@H](O)[C@H]1O
References
- Synthesis Reference
Patent US20180208955A1: Process for the microbial synthesis of migalastat (https://patents.google.com/patent/US20180208955A1/en)
- General References
- Warnock DG, Bichet DG, Holida M, Goker-Alpan O, Nicholls K, Thomas M, Eyskens F, Shankar S, Adera M, Sitaraman S, Khanna R, Flanagan JJ, Wustman BA, Barth J, Barlow C, Valenzano KJ, Lockhart DJ, Boudes P, Johnson FK: Oral Migalastat HCl Leads to Greater Systemic Exposure and Tissue Levels of Active alpha-Galactosidase A in Fabry Patients when Co-Administered with Infused Agalsidase. PLoS One. 2015 Aug 7;10(8):e0134341. doi: 10.1371/journal.pone.0134341. eCollection 2015. [Article]
- Migalastat for the treatment of Fabry Disease: Sunder-Plassmann G., Schiffmann R., and Nicholls K. [Link]
- Amicus Therapeutics News Release: FDA Approves Galafold™ (migalastat) for the Treatment of Certain Adult Patients with Fabry Disease [Link]
- Drugs.com: FDA Approves Galafold (migalastat) for the Treatment of Fabry Disease [Link]
- FDA approved drug product: GALAFOLD® (migalastat) capsules, for oral use [Link]
- EMA Approved Drug Products: GALAFOLD (Migalastat) capsules for oral use [Link]
- Health Canada Approved Drug Proucts: GALAFOLD (Migalastat) capsules for oral use [Link]
- Patent US20180208955A1: Process for the microbial synthesis of migalastat [File]
- External Links
- PubChem Compound
- 176077
- PubChem Substance
- 347827704
- ChemSpider
- 153388
- BindingDB
- 50163440
- 2054252
- ChEBI
- 135923
- ChEMBL
- CHEMBL110458
- ZINC
- ZINC000001636704
- PDBe Ligand
- DGJ
- Wikipedia
- Migalastat
- PDB Entries
- 3s5y / 3thd / 3tv8 / 4cu8 / 4d1j / 4do5 / 4fns / 4ufm / 6euh / 6tsh … show 1 more
- FDA label
- Download (1.3 MB)
- MSDS
- Download (72.5 KB)
Clinical Trials
- Clinical Trials
Clinical Trial & Rare Diseases Add-on Data Package
Explore 4,000+ rare diseases, orphan drugs & condition pairs, clinical trial why stopped data, & more. Preview package Phase Status Purpose Conditions Count Start Date Why Stopped 100+ additional columns Unlock 175K+ rows when you subscribe.View sample dataNot Available Active Not Recruiting Not Available Fabry's Disease 1 somestatus stop reason just information to hide Not Available Approved for Marketing Not Available Fabry's Disease 1 somestatus stop reason just information to hide Not Available Recruiting Not Available Fabry's Disease 1 somestatus stop reason just information to hide 3 Active Not Recruiting Treatment Fabry's Disease 1 somestatus stop reason just information to hide 3 Completed Treatment Fabry's Disease 4 somestatus stop reason just information to hide
Pharmacoeconomics
- Manufacturers
- Not Available
- Packagers
- Not Available
- Dosage Forms
Form Route Strength Capsule Oral 123 mg Capsule Oral 123 mg/1 Capsule, coated Oral 150 mg - Prices
- Not Available
- Patents
Patent Number Pediatric Extension Approved Expires (estimated) Region US9987263 No 2018-06-05 2027-05-16 US US9000011 No 2015-04-07 2027-05-16 US US8592362 No 2013-11-26 2029-02-12 US US9095584 No 2015-08-04 2029-02-12 US US9480682 No 2016-11-01 2027-05-16 US US9999618 No 2018-06-19 2028-04-28 US US10076514 No 2018-09-18 2037-03-15 US US10251873 No 2019-04-09 2038-05-30 US US10383864 No 2019-08-20 2027-05-16 US US10471053 No 2019-11-12 2038-05-30 US US10406143 No 2019-09-10 2027-05-16 US US10525045 No 2020-01-07 2028-04-28 US US10792278 No 2020-10-06 2038-05-30 US US10792279 No 2020-10-06 2038-05-30 US US10813921 No 2020-10-27 2029-02-12 US US10857141 No 2020-12-08 2038-05-30 US US10849890 No 2020-12-01 2038-05-30 US US10849889 No 2020-12-01 2038-05-30 US US10857142 No 2020-12-08 2038-05-30 US US10874657 No 2020-12-29 2038-05-30 US US10874655 No 2020-12-29 2038-05-30 US US10874656 No 2020-12-29 2038-05-30 US US10799491 No 2020-10-13 2038-05-30 US US10806727 No 2020-10-20 2038-05-30 US US10925866 No 2021-02-23 2028-04-28 US US11033538 No 2021-06-15 2028-04-28 US USRE48608 No 2021-06-29 2029-02-12 US US11241422 No 2007-05-16 2027-05-16 US US11234972 No 2017-03-15 2037-03-15 US US11278538 No 2018-05-30 2038-05-30 US US11278537 No 2018-05-30 2038-05-30 US US11278536 No 2018-05-30 2038-05-30 US US11278539 No 2018-05-30 2038-05-30 US US11278540 No 2018-05-30 2038-05-30 US US11304940 No 2018-05-30 2038-05-30 US US11357761 No 2008-05-30 2028-05-30 US US11357762 No 2018-05-30 2038-05-30 US US11357763 No 2018-05-30 2038-05-30 US US11357784 No 2019-02-06 2039-02-06 US US11389437 No 2018-05-30 2038-05-30 US US11389436 No 2018-05-30 2038-05-30 US US11357764 No 2018-05-30 2038-05-30 US US11376244 No 2018-05-30 2038-05-30 US US11357765 No 2018-05-30 2038-05-30 US US11426396 No 2018-05-30 2038-05-30 US US11458128 No 2018-05-30 2038-05-30 US US11642334 No 2019-02-20 2039-02-20 US US11633388 No 2019-03-25 2039-03-25 US US11633387 No 2018-05-30 2038-05-30 US US11622962 No 2019-03-17 2039-03-17 US US11612593 No 2018-05-30 2038-05-30 US US11612594 No 2018-05-30 2038-05-30 US US11666564 No 2018-05-30 2038-05-30 US US11786516 No 2018-05-30 2038-05-30 US US11833164 No 2022-01-11 2042-01-11 US US11813255 No 2018-05-30 2038-05-30 US US11826360 No 2019-02-16 2039-02-16 US US11903938 No 2018-08-17 2038-08-17 US US12042490 No 2018-05-30 2038-05-30 US US12042489 No 2018-05-30 2038-05-30 US US12042488 No 2018-05-30 2038-05-30 US
Properties
- State
- Solid
- Experimental Properties
- Not Available
- Predicted Properties
Property Value Source Water Solubility 511.0 mg/mL ALOGPS logP -2.2 ALOGPS logP -2.9 Chemaxon logS 0.5 ALOGPS pKa (Strongest Acidic) 12.91 Chemaxon pKa (Strongest Basic) 8.06 Chemaxon Physiological Charge 1 Chemaxon Hydrogen Acceptor Count 5 Chemaxon Hydrogen Donor Count 5 Chemaxon Polar Surface Area 92.95 Å2 Chemaxon Rotatable Bond Count 1 Chemaxon Refractivity 36.57 m3·mol-1 Chemaxon Polarizability 15.86 Å3 Chemaxon Number of Rings 1 Chemaxon Bioavailability 1 Chemaxon Rule of Five Yes Chemaxon Ghose Filter No Chemaxon Veber's Rule No Chemaxon MDDR-like Rule No Chemaxon - Predicted ADMET Features
- Not Available
Spectra
- Mass Spec (NIST)
- Not Available
- Spectra
Spectrum Spectrum Type Splash Key Predicted GC-MS Spectrum - GC-MS Predicted GC-MS splash10-0feb-7900000000-536465f5fd772045b134 Predicted MS/MS Spectrum - 10V, Positive (Annotated) Predicted LC-MS/MS splash10-002b-0900000000-179be3503509a3b85d39 Predicted MS/MS Spectrum - 10V, Negative (Annotated) Predicted LC-MS/MS splash10-01q9-1900000000-158c07bd46d97cf5da76 Predicted MS/MS Spectrum - 20V, Positive (Annotated) Predicted LC-MS/MS splash10-004j-1900000000-54d3fe7d55f96a10c7ae Predicted MS/MS Spectrum - 20V, Negative (Annotated) Predicted LC-MS/MS splash10-03e9-3900000000-209067245351329bb22d Predicted MS/MS Spectrum - 40V, Positive (Annotated) Predicted LC-MS/MS splash10-0pbc-9400000000-030cbb5d966f8ee8d6ca Predicted MS/MS Spectrum - 40V, Negative (Annotated) Predicted LC-MS/MS splash10-0006-9100000000-de6e9c082c4064e26585 Predicted 1H NMR Spectrum 1D NMR Not Applicable Predicted 13C NMR Spectrum 1D NMR Not Applicable - Chromatographic Properties
Collision Cross Sections (CCS)
Adduct CCS Value (Å2) Source type Source [M-H]- 132.60118 predictedDeepCCS 1.0 (2019) [M+H]+ 134.99677 predictedDeepCCS 1.0 (2019) [M+Na]+ 142.0397 predictedDeepCCS 1.0 (2019)
Targets
- Kind
- Protein
- Organism
- Humans
- Pharmacological action
- Yes
- Actions
- Stabilization
- General Function
- Catalyzes the hydrolysis of glycosphingolipids and participates in their degradation in the lysosome
- Specific Function
- Alpha-galactosidase activity
- Gene Name
- GLA
- Uniprot ID
- P06280
- Uniprot Name
- Alpha-galactosidase A
- Molecular Weight
- 48766.34 Da
References
- FDA approved drug product: GALAFOLD® (migalastat) capsules, for oral use [Link]
Enzymes
- Kind
- Protein group
- Organism
- Humans
- Pharmacological action
- Unknown
- Actions
- Substrate
- General Function
- UDPGT is of major importance in the conjugation and subsequent elimination of potentially toxic xenobiotics and endogenous compounds. This isoform has specificity for phenols. Isoform 3 lacks transferase activity but acts as a negative regulator of isoform 1 (By similarity)
- Specific Function
- Enzyme binding
Components:
References
- FDA approved drug product: GALAFOLD® (migalastat) capsules, for oral use [Link]
Transporters
- Kind
- Protein
- Organism
- Humans
- Pharmacological action
- No
- Actions
- SubstrateInhibitor
- Curator comments
- Low affinity
- General Function
- Electrogenic Na(+)-coupled sugar symporter that actively transports D-glucose or D-galactose at the plasma membrane, with a Na(+) to sugar coupling ratio of 2:1. Transporter activity is driven by a transmembrane Na(+) electrochemical gradient set by the Na(+)/K(+) pump (PubMed:20980548, PubMed:34880492, PubMed:35077764, PubMed:8563765). Has a primary role in the transport of dietary monosaccharides from enterocytes to blood. Responsible for the absorption of D-glucose or D-galactose across the apical brush-border membrane of enterocytes, whereas basolateral exit is provided by GLUT2. Additionally, functions as a D-glucose sensor in enteroendocrine cells, triggering the secretion of the incretins GCG and GIP that control food intake and energy homeostasis (By similarity) (PubMed:8563765). Together with SGLT2, functions in reabsorption of D-glucose from glomerular filtrate, playing a nonredundant role in the S3 segment of the proximal tubules (By similarity). Transports D-glucose into endometrial epithelial cells, controlling glycogen synthesis and nutritional support for the embryo as well as the decidual transformation of endometrium prior to conception (PubMed:28974690). Acts as a water channel enabling passive water transport across the plasma membrane in response to the osmotic gradient created upon sugar and Na(+) uptake. Has high water conductivity, comparable to aquaporins, and therefore is expected to play an important role in transepithelial water permeability, especially in the small intestine
- Specific Function
- Alpha-glucoside transmembrane transporter activity
- Gene Name
- SLC5A1
- Uniprot ID
- P13866
- Uniprot Name
- Sodium/glucose cotransporter 1
- Molecular Weight
- 73497.275 Da
References
- FDA approved drug product: GALAFOLD® (migalastat) capsules, for oral use [Link]
Drug created at October 21, 2007 22:23 / Updated at September 15, 2024 04:53