Tezacaftor

Identification

Summary

Tezacaftoris a medication used to treat homozygous or heterozygous F508del mutation cystic fibrosis.

Brand Names

Symdeko, Trikafta (100 Mg / 50 Mg / 75 Mg; 150 Mg)

Generic Name

Tezacaftor

DrugBank Accession Number

DB11712

Background

Tezacaftor is a drug of the cystic fibrosis transmembrane conductance regulator (CFTR) potentiator class.¹⁰It was developed by Vertex Pharmaceuticals and FDA approved in combination withivacaftorto manage cystic fibrosis.¹⁴This drug was approved by the FDA on February 12, 2018.¹⁵

Cystic Fibrosis is an autosomal recessive disorder caused by one of several different mutations in the gene for the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein, an ion channel involved in the transport of chloride and sodium ions across cell membranes. CFTR is active in epithelial cells of organs such as of the lungs, pancreas, liver, digestive system, and reproductive tract. Alterations in the CFTR gene result in altered production, misfolding, or function of the protein and consequently abnormal fluid and ion transport across cell membranes.^5,6As a result, CF patients produce thick, sticky mucus that clogs the ducts of organs where it is produced making patients more susceptible to complications such as infections, lung damage, pancreatic insufficiency, and malnutrition.⁷

Type

Small Molecule

Groups

Approved, Investigational

Structure

3D

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MOL SDF 3D-SDF PDB SMILES InChI

Similar Structures

Structure for Tezacaftor (DB11712)

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Weight

Average: 520.505
Monoisotopic: 520.182121086

Chemical Formula

C₂₆H₂₇F₃N₂O₆

Synonyms

• Tezacaftor

External IDs

• VX 661
• VX-661
• VX661

Pharmacology

Indication

Tezacaftor is combined with ivacaftor in one product for the treatment of cystic fibrosis (CF) in patients aged 12 years or older with two copies of theF508delgene mutation or at least one mutation in the CFTR gene that is responsive to this drug.¹⁴

Tezacaftor, when used in combination with ivacaftor andelexacaftorin the product Trikafta, is also indicated for the treatment of CF in patients 12 years of age and older that have at least oneF508delmutation in the CFTR gene.¹⁹

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Associated Conditions

• Cystic Fibrosis (CF)

Contraindications & Blackbox Warnings

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Pharmacodynamics

Clinical studies have shown a significant decrease in sweat chloride and an increase in the forced expiratory volume (FEV), a measure of lung function, following Tevacaftor/Ivacaftor therapy.¹Phase 3 clinical studies have shown that a significant increase in forced expiratory volume was attained at 4 and 8 weeks after initiating this drug. The above effects lead to improvement of the respiratory symptoms of cystic fibrosis. Tezacaftor does not induce clinically significant QT prolongation.²When given with ivacaftor, tezacaftor can lead to liver transaminase elevations.¹⁴Testing of transaminases (ALT and AST) levels should occur before starting this combination every 3 months during the first year of treatment, and every year afterwards. Patients with a history of transaminase elevations should be monitored more frequently.¹⁴

Mechanism of action

The transport of charged ions across cell membranes is normally achieved through the actions of the cystic fibrosis transmembrane regulator (CFTR) protein. This protein acts as a channel and allows for the passage of chloride and sodium. This process affects the movement of water in and out of the tissues and impacts the production of mucus that lubricates and protects certain organs and body tissues, including the lungs. In theF508delCFTR突变的检测基因,一个氨基酸是删除ed at the position 508, therefore, the CFTR channel function is compromised, resulting in thickened mucus secretions.¹⁰CFTR correctors such as tezacaftor aim to repair F508del cellular misprocessing.⁹This is done by modulating the position of the CFTR protein on the cell surface to the correct position, allowing for adequate ion channel formation and increased in water and salt movement through the cell membrane.¹⁰The concomitant use of ivacaftor is intended to maintain an open channel, increasing the transport of chloride, reducing thick mucus production.¹³

Target	Actions	Organism
ACystic fibrosis transmembrane conductance regulator	activator	Humans

Absorption

The Cmax, Tmax and AUC of tezacaftor, when administered with ivacaftor, are 5.95 mcg/ml, 2-6 h, and 84.5 mcg.h/ml respectively.¹⁴Exposure of tezacaftor/ivacaftor increases 3-fold when it is administered with a high-fat meal.¹⁴

Volume of distribution

The apparent volume of distribution of tezacaftor was 271 L in a study of patients in the fed state who received 100 mg of tezacaftor every 12 hours.¹⁴

Protein binding

Tezacaftor is approximately 99% bound to plasma proteins, mainly albumin.¹⁴

Metabolism

Tezacaftor is metabolized extensively in humans by the action of CYP3A4 and CYP3A5. There are three main circulating metabolites; M1, M2, and M5. The M1 is an active metabolite with similar activity to the parent drug, tezacaftor. The M2 metabolite is significantly less active and M5 is considered an inactive metabolite.^4,14An additional circulating metabolite, M3, corresponding to the glucuronide form of tezacaftor.¹⁴

Hover over products below to view reaction partners

• Tezacaftor
  • M1 Metabolite, Tezacaftor+M2 Metabolite, Tezacaftor+M5 metabolite, Tezacaftor
  • M3 Metabolite, Tezacaftor

Route of elimination

After oral administration, the majority of tezacaftor dose (72%) is found excreted in the feces either unchanged or as its metabolite, M2. About 14% of the administered dose is found excreted in the urine as the metabolite, M2. It was noted that less than 1% of the administered dose is excreted unchanged in the urine and thus, renal excretion is not the major elimination pathway.¹⁴

Half-life

The apparent half-life of tezacaftor is approximately 57.2 hours.^4,18

Clearance

The apparent clearance of tezacaftor has been measured at 1.31 L/h for patients in the fed state during a clinical trial.³

Adverse Effects

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Toxicity

The LD50 of an oral dose in rats is >2000 mg/kg.¹⁷

Overdose symptoms may include dizziness and diarrhea. There have been no reports to this date of tezacaftor overdose, but the highest dose of 450 mg every 12 hours commonly resulted in reports of dizziness and diarrhea. No antidote exists for treating an overdose with this drug. General supportive measures should be undertaken along with monitoring of vital signs and close monitoring of clinical status.¹⁴

Pathways

Not Available

Pharmacogenomic Effects/ADRsBrowse all" title="" id="snp-actions-info" class="drug-info-popup" href="javascript:void(0);">

Not Available

Interactions

Drug InteractionsLearn More" title="" id="structured-interactions-info" class="drug-info-popup" href="javascript:void(0);">

这些信息不应该被解释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.

• Approved
• Vet approved
• Nutraceutical
• Illicit
• Withdrawn
• Investigational
• Experimental
• All Drugs

Drug	Interaction
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Abametapir	的血清浓度Tezacaftor可以分辨ased when it is combined with Abametapir.
Abatacept	The metabolism of Tezacaftor can be increased when combined with Abatacept.
Abemaciclib	The serum concentration of Abemaciclib can be increased when it is combined with Tezacaftor.
Abrocitinib	的血清浓度Tezacaftor可以分辨ased when it is combined with Abrocitinib.
Acalabrutinib	The metabolism of Tezacaftor can be decreased when combined with Acalabrutinib.
Adagrasib	的血清浓度Tezacaftor可以分辨ased when it is combined with Adagrasib.
Adalimumab	The metabolism of Tezacaftor can be increased when combined with Adalimumab.
Afatinib	的血清浓度Tezacaftor可以分辨ased when it is combined with Afatinib.
Alfentanil	The metabolism of Tezacaftor can be decreased when combined with Alfentanil.
Alprazolam	The metabolism of Tezacaftor can be decreased when combined with Alprazolam.

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Food Interactions

• Avoid grapefruit products. Grapefruit inhibits CYP3A metabolism, which may increase the serum concentration of tezacaftor.
• Avoid St. John's Wort. This herb induces CYP3A metabolism and may reduce serum levels of tezacaftor.
• Take with food. Tezacaftor should be taken with fat-containing food to increase exposure.

Products

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Mixture Products

Name	Ingredients	Dosage	Route	Labeller	Marketing Start	Marketing End	Region	Image
Kaftrio	Tezacaftor(25 mg)+Elexacaftor(50 mg)+Ivacaftor(37.5 mg)	Tablet, film coated	Oral	Vertex Pharmaceuticals (Ireland) Limited	2022-05-04	Not applicable
Kaftrio	Tezacaftor(50 mg)+Elexacaftor(100 mg)+Ivacaftor(75 mg)	Tablet, film coated	Oral	Vertex Pharmaceuticals (Ireland) Limited	2020-12-15	Not applicable
Symdeko	Tezacaftor(100 mg)+Ivacaftor(150 mg)+Ivacaftor(150 mg)	Tablet	Oral	Vertex Pharmaceuticals Incorporated	2018-06-28	Not applicable
Symdeko	Tezacaftor(100 mg/1)+Ivacaftor(150 mg/1)+Ivacaftor(150 mg/1)	Kit; Tablet, film coated	Oral	Vertex Pharmaceuticals Incorporated	2018-02-12	Not applicable
Symdeko	Tezacaftor(50 mg/1)+Ivacaftor(75 mg/1)+Ivacaftor(75 mg/1)	Kit; Tablet, film coated	Oral	Vertex Pharmaceuticals Incorporated	2019-06-21	Not applicable
Symkevi	Tezacaftor(100 mg)+Ivacaftor(150 mg)	Tablet, film coated	Oral	Vertex Pharmaceuticals (Ireland) Limited	2021-01-12	Not applicable
Symkevi	Tezacaftor(50 mg)+Ivacaftor(75 mg)	Tablet, film coated	Oral	Vertex Pharmaceuticals (Ireland) Limited	2021-01-28	Not applicable
Trikafta	Tezacaftor(25 mg)+Elexacaftor(50 mg)+Ivacaftor(37.5 mg)+Ivacaftor(75 mg)	Tablet	Oral	Vertex Pharmaceuticals Incorporated	2022-04-22	Not applicable
Trikafta	Tezacaftor(25 mg/1)+Elexacaftor(50 mg/1)+Ivacaftor(37.5 mg/1)+Ivacaftor(75 mg/1)	Kit; Tablet, film coated	Oral	Vertex Pharmaceuticals Incorporated	2021-06-08	Not applicable
Trikafta	Tezacaftor(50 mg/1)+Elexacaftor(100 mg/1)+Ivacaftor(75 mg/1)+Ivacaftor(150 mg/1)	Kit; Tablet, film coated	Oral	Vertex Pharmaceuticals Incorporated	2019-10-21	Not applicable

Categories

ATC Codes

R07AX32 — Ivacaftor, tezacaftor and elexacaftor

• R07AX — Other respiratory system products
• R07A — OTHER RESPIRATORY SYSTEM PRODUCTS
• R07 — OTHER RESPIRATORY SYSTEM PRODUCTS
• R — RESPIRATORY SYSTEM

R07AX31 — Ivacaftor and tezacaftor

• R07AX — Other respiratory system products
• R07A — OTHER RESPIRATORY SYSTEM PRODUCTS
• R07 — OTHER RESPIRATORY SYSTEM PRODUCTS
• R — RESPIRATORY SYSTEM

Drug Categories

• BCRP/ABCG2 Substrates
• 细胞色素P-450 CYP3A Substrates
• 细胞色素P-450 CYP3A4 Substrates
• 细胞色素P-450 CYP3A5 Substrates
• 细胞色素P-450 Substrates
• Dioxoles
• Heterocyclic Compounds, Fused-Ring
• P-glycoprotein inhibitors
• P-glycoprotein substrates

Chemical TaxonomyProvided byClassyfire

Description

This compound belongs to the class of organic compounds known as n-alkylindoles. These are compounds containing an indole moiety that carries an alkyl chain at the 1-position.

Kingdom

Organic compounds

Super Class

Organoheterocyclic compounds

Class

Indoles and derivatives

Sub Class

N-alkylindoles

Direct Parent

N-alkylindoles

Alternative Parents

Indoles/Benzodioxoles/N-arylamides/Substituted pyrroles/Aryl fluorides/Benzenoids/Cyclopropanecarboxylic acids and derivatives/Heteroaromatic compounds/Secondary carboxylic acid amides/二级醇 /1,2-diols/Oxacyclic compounds/Azacyclic compounds/Carbonyl compounds/Hydrocarbon derivatives/Organic oxides/Organofluorides/Alkyl fluorides/Primary alcohols

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Substituents

1,2-diol/Alcohol/Alkyl fluoride/Alkyl halide/Aromatic heteropolycyclic compound/Aryl fluoride/Aryl halide/Azacycle/Benzenoid/Benzodioxole /Carbonyl group/Carboxamide group/Carboxylic acid derivative/Cyclopropanecarboxylic acid or derivatives/Heteroaromatic compound/Hydrocarbon derivative/Indole/N-alkylindole/N-arylamide/Organic nitrogen compound/Organic oxide/Organic oxygen compound/Organofluoride/Organohalogen compound/Organonitrogen compound/Organooxygen compound/Oxacycle/Primary alcohol/Pyrrole/Secondary alcohol/Secondary carboxylic acid amide/Substituted pyrrole

show 22 more

Molecular Framework

Aromatic heteropolycyclic compounds

External Descriptors

Not Available

Affected organisms

• Humans and other mammals

Chemical Identifiers

UNII

8RW88Y506K

CAS number

1152311-62-0

InChI Key

MJUVRTYWUMPBTR-MRXNPFEDSA-N

InChI

InChI=1S/C26H27F3N2O6/c1-24(2,13-33)22-8-14-7-18(17(27)10-19(14)31(22)11-16(34)12-32)30-23(35)25(5-6-25)15-3-4-20-21(9-15)37-26(28,29)36-20/h3-4,7-10,16,32-34H,5-6,11-13H2,1-2H3,(H,30,35)/t16-/m1/s1

IUPAC Name

1-(2,2-difluoro-2H-1,3-benzodioxol-5-yl)-N-{1-[(2R)-2,3-dihydroxypropyl]-6-fluoro-2-(1-hydroxy-2-methylpropan-2-yl)-1H-indol-5-yl}cyclopropane-1-carboxamide

SMILES

CC(C)(CO)C1=CC2=CC(NC(=O)C3(CC3)C3=CC=C4OC(F)(F)OC4=C3)=C(F)C=C2N1C[C@@H](O)CO

References

General References

1. Donaldson SH, Pilewski JM, Griese M, Cooke J, Viswanathan L, Tullis E, Davies JC, Lekstrom-Himes JA, Wang LT: Tezacaftor/Ivacaftor in Subjects with Cystic Fibrosis and F508del/F508del-CFTR or F508del/G551D-CFTR. Am J Respir Crit Care Med. 2018 Jan 15;197(2):214-224. doi: 10.1164/rccm.201704-0717OC. [Article]
2. Taylor-Cousar杰,Munck McKone EF, van der EntCK, Moeller A, Simard C, Wang LT, Ingenito EP, McKee C, Lu Y, Lekstrom-Himes J, Elborn JS: Tezacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del. N Engl J Med. 2017 Nov 23;377(21):2013-2023. doi: 10.1056/NEJMoa1709846. Epub 2017 Nov 3. [Article]
3. Rowe SM, Daines C, Ringshausen FC, Kerem E, Wilson J, Tullis E, Nair N, Simard C, Han L, Ingenito EP, McKee C, Lekstrom-Himes J, Davies JC: Tezacaftor-Ivacaftor in Residual-Function Heterozygotes with Cystic Fibrosis. N Engl J Med. 2017 Nov 23;377(21):2024-2035. doi: 10.1056/NEJMoa1709847. Epub 2017 Nov 3. [Article]
4. Garg V, Shen J, Li C, Agarwal S, Gebre A, Robertson S, Huang J, Han L, Jiang L, Stephan K, Wang LT, Lekstrom-Himes J: Pharmacokinetic and Drug-Drug Interaction Profiles of the Combination of Tezacaftor/Ivacaftor. Clin Transl Sci. 2019 May;12(3):267-275. doi: 10.1111/cts.12610. Epub 2019 Jan 29. [Article]
5. Saint-Criq V, Gray MA: Role of CFTR in epithelial physiology. Cell Mol Life Sci. 2017 Jan;74(1):93-115. doi: 10.1007/s00018-016-2391-y. Epub 2016 Oct 6. [Article]
6. Kunzelmann K, Mall M: Pharmacotherapy of the ion transport defect in cystic fibrosis: role of purinergic receptor agonists and other potential therapeutics. Am J Respir Med. 2003;2(4):299-309. [Article]
7. Fraser-Pitt D, O'Neil D: Cystic fibrosis - a multiorgan protein misfolding disease. Future Sci OA. 2015 Sep 1;1(2):FSO57. doi: 10.4155/fso.15.57. eCollection 2015 Sep. [Article]
8. Fohner AE, McDonagh EM, Clancy JP, Whirl Carrillo M, Altman RB, Klein TE: PharmGKB summary: ivacaftor pathway, pharmacokinetics/pharmacodynamics. Pharmacogenet Genomics. 2017 Jan;27(1):39-42. doi: 10.1097/FPC.0000000000000246. [Article]
9. Rowe SM, Verkman AS: Cystic fibrosis transmembrane regulator correctors and potentiators. Cold Spring Harb Perspect Med. 2013 Jul 1;3(7). pii: 3/7/a009761. doi: 10.1101/cshperspect.a009761. [Article]
10. Cystic fibrosis news [Link]
11. Vertex news [Link]
12. Vertex news [Link]
13. Vertex news [Link]
14. Symdeko FDA label [Link]
15. FDA Approved Drug Products: Apadaz (benzhydrocodone and acetaminophen) tablets [Link]
16. NIH Medline Tezacaftor/Ivacaftor [Link]
17. Pharmacology review, FDA [Link]
18. Monograph, Tezacaftor/Ivacaftor [Link]
19. FDA Approved Drug Products: Trikafta (elexacaftor, tezacaftor and ivacaftor tablets; ivacaftor tablets) [Link]

External Links

KEGG Drug

D11041

PubChem Compound

46199646

PubChem Substance

347828077

ChemSpider

28637762

BindingDB

281054

RxNav

1999382

ChEMBL

CHEMBL3544914

ZINC

ZINC000068206930

PDBe Ligand

CV6

Wikipedia

Tezacaftor

PDB Entries

7sv7/8eiq

FDA label

Download (459 KB)

MSDS

Download (27.1 KB)

Clinical Trials

Clinical TrialsLearn More" title="" id="clinical-trials-info" class="drug-info-popup" href="javascript:void(0);">

Phase	Status	Purpose	Conditions	Count
4	Recruiting	Other	Adherence, Medication/Cystic Fibrosis (CF)/Cystic Fibrosis Gastrointestinal Disease/Cystic Fibrosis in Children/Cystic Fibrosis Liver Disease	1
4	Recruiting	Other	Drug Drug Interaction (DDI)	1
4	Recruiting	Treatment	Cystic Fibrosis (CF)	1
3	Active Not Recruiting	Treatment	Cystic Fibrosis (CF)	6
3	Completed	Treatment	Cystic Fibrosis (CF)	24
3	Enrolling by Invitation	Treatment	Cystic Fibrosis (CF)	3
3	Recruiting	Treatment	Cystic Fibrosis (CF)	1
3	Terminated	Treatment	Cystic Fibrosis (CF)	2
2	Completed	Other	Coronavirus Disease 2019 (COVID‑19)/Cystic Fibrosis (CF)	1
2	Completed	Treatment	Cystic Fibrosis (CF)	8

Pharmacoeconomics

Manufacturers

Not Available

Packagers

Not Available

Dosage Forms

Form	Route	Strength
Tablet	Oral
Tablet, film coated	Oral
Kit; tablet, film coated	Oral

Prices

Not Available

Patents

Patent Number	Pediatric Extension	Approved	Expires (estimated)	Region
US8324242	No	2012-12-04	2027-04-18
US8354427	No	2013-01-15	2026-07-06
US8754224	No	2014-06-17	2026-12-28
US8410274	No	2013-04-02	2026-12-28
US7495103	No	2009-02-24	2027-05-20
US9670163	No	2017-06-06	2026-12-28
US8415387	No	2013-04-09	2027-11-12
US9012496	No	2015-04-21	2033-07-15
US8598181	No	2013-12-03	2027-05-01
US8629162	No	2014-01-14	2025-06-24
US8623905	No	2014-01-07	2027-05-01
US7645789	No	2010-01-12	2027-05-01
US7776905	No	2010-08-17	2027-06-03
US9931334	No	2018-04-03	2026-12-28
US9974781	No	2018-05-22	2027-04-09
US10022352	No	2018-07-17	2027-04-09
US10058546	No	2018-08-28	2033-07-15
US10081621	No	2018-09-25	2031-03-25
US10206877	No	2019-02-19	2035-04-14
US10239867	No	2019-03-26	2027-04-09
US10646481	No	2020-05-12	2029-08-13
US10758534	No	2020-09-01	2035-10-06
US10793547	No	2020-10-06	2037-12-08
US11179367	No	2021-11-23	2037-12-08
US11426407	No	2015-10-06	2035-10-06
US11453655	No	2017-12-08	2037-12-08
US11517564	No	2017-12-08	2037-12-08
US11564916	No	2009-08-13	2029-08-13

Properties

State

Solid

Experimental Properties

Property	Value	Source
water solubility	Insoluble in water	https://www.medkoo.com/products/4790
logP	99	https://www.medkoo.com/uploads/product/Tezacaftor__VX-661_/safety/MSDS-VX661.pdf
pKa	13.99, 0.19	https://www.ebi.ac.uk/chembl/compound_report_card/CHEMBL3544914/

Predicted Properties

Property	Value	Source
Water Solubility	0.0124 mg/mL	ALOGPS
logP	2.97	ALOGPS
logP	4.03	Chemaxon
logS	-4.6	ALOGPS
pKa (Strongest Acidic)	11.54	Chemaxon
pKa (Strongest Basic)	-2.8	Chemaxon
Physiological Charge	0	Chemaxon
Hydrogen Acceptor Count	6	Chemaxon
Hydrogen Donor Count	4	Chemaxon
Polar Surface Area	113.18 Å2	Chemaxon
Rotatable Bond Count	8	Chemaxon
Refractivity	125.53 m3·mol-1	Chemaxon
Polarizability	51.79 Å3	Chemaxon
Number of Rings	5	Chemaxon
Bioavailability	1	Chemaxon
Rule of Five	No	Chemaxon
Ghose Filter	No	Chemaxon
Veber's Rule	No	Chemaxon
MDDR-like Rule	Yes	Chemaxon

Predicted ADMET Features

Not Available

Spectra

Mass Spec (NIST)

Not Available

Spectra

Spectrum	Spectrum Type	Splash Key
Predicted MS/MS Spectrum - 10V, Positive (Annotated)	Predicted LC-MS/MS	Not Available
Predicted MS/MS Spectrum - 20V, Positive (Annotated)	Predicted LC-MS/MS	Not Available
Predicted MS/MS Spectrum - 40V, Positive (Annotated)	Predicted LC-MS/MS	Not Available
Predicted MS/MS Spectrum - 10V, Negative (Annotated)	Predicted LC-MS/MS	Not Available
Predicted MS/MS Spectrum - 20V, Negative (Annotated)	Predicted LC-MS/MS	Not Available
Predicted MS/MS Spectrum - 40V, Negative (Annotated)	Predicted LC-MS/MS	Not Available

Targets

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Use our structured and evidence-based datasets tounlock new
insights and accelerate drug research.

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Details 1.Cystic fibrosis transmembrane conductance regulator

Kind

Protein

Organism

Humans

Pharmacological action

Yes

Actions

Activator

General Function

Pdz domain binding

Specific Function

Involved in the transport of chloride ions. May regulate bicarbonate secretion and salvage in epithelial cells by regulating the SLC4A7 transporter. Can inhibit the chloride channel activity of ANO...

Gene Name

CFTR

Uniprot ID

P13569

Uniprot Name

Cystic fibrosis transmembrane conductance regulator

分子量

168139.895 Da

References

1. Donaldson SH, Pilewski JM, Griese M, Cooke J, Viswanathan L, Tullis E, Davies JC, Lekstrom-Himes JA, Wang LT: Tezacaftor/Ivacaftor in Subjects with Cystic Fibrosis and F508del/F508del-CFTR or F508del/G551D-CFTR. Am J Respir Crit Care Med. 2018 Jan 15;197(2):214-224. doi: 10.1164/rccm.201704-0717OC. [Article]
2. Taylor-Cousar杰,Munck McKone EF, van der EntCK, Moeller A, Simard C, Wang LT, Ingenito EP, McKee C, Lu Y, Lekstrom-Himes J, Elborn JS: Tezacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del. N Engl J Med. 2017 Nov 23;377(21):2013-2023. doi: 10.1056/NEJMoa1709846. Epub 2017 Nov 3. [Article]
3. Saint-Criq V, Gray MA: Role of CFTR in epithelial physiology. Cell Mol Life Sci. 2017 Jan;74(1):93-115. doi: 10.1007/s00018-016-2391-y. Epub 2016 Oct 6. [Article]
4. Cystic fibrosis news [Link]
5. Symdeko FDA label [Link]

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Drug created at October 20, 2016 20:41 / Updated at March 04, 2021 09:26