Ataluren (PTC124)

    WARNING: This product is for research use only, not for human or veterinary use.

MedKoo CAT#: 205786

CAS#: 775304-57-9

Description: Ataluren, also known as PTC124, is an investigational new drug designed to enable the formation of a functioning protein in patients with genetic disorders due to a nonsense mutation. A nonsense mutation is an alteration in the genetic code that prematurely halts the synthesis of an essential protein. Ataluren is currently being investigated for use in patients with nonsense mutation Duchenne/Becker muscular dystrophy (nmDBMD) and cystic fibrosis (nmCF)

Chemical Structure

Ataluren (PTC124)
CAS# 775304-57-9

Theoretical Analysis

MedKoo Cat#: 205786
Name: Ataluren (PTC124)
CAS#: 775304-57-9
Chemical Formula: C15H9FN2O3
Exact Mass: 284.05972
Molecular Weight: 284.24196
Elemental Analysis: C, 63.38; H, 3.19; F, 6.68; N, 9.86; O, 16.89

Size Price Shipping out time Quantity
100mg USD 550 2 Weeks
200mg USD 950 2 Weeks
500mg USD 1450 2 Weeks
1g USD 2450 2 Weeks
2g USD 3850 2 Weeks
5g USD 5950 2 Weeks
Inquire bulk and customized quantity

Pricing updated 2021-03-02. Prices are subject to change without notice.

Ataluren, purity > 98%, is in stock. Current shipping out time is about 2 weeks after order is received. CoA, QC data and MSDS documents are available in one week after order is received.

Synonym: PTC124; PTC 124; PTC-124; Ataluren.

IUPAC/Chemical Name: 3-[5-(2-fluorophenyl)-1,2,4-oxadiazol-3-yl]benzoic acid


InChi Code: InChI=1S/C15H9FN2O3/c16-12-7-2-1-6-11(12)14-17-13(18-21-14)9-4-3-5-10(8-9)15(19)20/h1-8H,(H,19,20)


Solid powder

>98% (or refer to the Certificate of Analysis)

Shipping Condition:
Shipped under ambient temperature as non-hazardous chemical. This product is stable enough for a few weeks during ordinary shipping and time spent in Customs.

Storage Condition:
Dry, dark and at 0 - 4 C for short term (days to weeks) or -20 C for long term (months to years).

Soluble in DMSO, not in water

Shelf Life:
>5 years if stored properly

Drug Formulation:
This drug may be formulated in DMSO

Stock Solution Storage:
0 - 4 C for short term (days to weeks), or -20 C for long term (months).

HS Tariff Code:

Handling Instructions:

Preparing Stock Solutions

The following data is based on the product molecular weight 284.24196 Batch specific molecular weights may vary from batch to batch due to the degree of hydration, which will affect the solvent volumes required to prepare stock solutions.

Select a batch to recalculate based on the batch molecular weight:
Concentration / Solvent Volume / Mass 1 mg 5 mg 10 mg
1 mM 1.15 mL 5.76 mL 11.51 mL
5 mM 0.23 mL 1.15 mL 2.3 mL
10 mM 0.12 mL 0.58 mL 1.15 mL
50 mM 0.02 mL 0.12 mL 0.23 mL

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1: Koopmann TT, Verkerk AO, Bezzina CR, de Bakker JM, Wilde AA. The Chemical Compound PTC124 Does Not Affect Cellular Electrophysiology of Cardiac Ventricular Myocytes. Cardiovasc Drugs Ther. 2012 Feb;26(1):41-5. PubMed PMID: 22065308.

2: Sarkar C, Zhang Z, Mukherjee AB. Stop codon read-through with PTC124 induces palmitoyl-protein thioesterase-1 activity, reduces thioester load and suppresses apoptosis in cultured cells from INCL patients. Mol Genet Metab. 2011 Nov;104(3):338-45. Epub 2011 Jun 13. PubMed PMID: 21704547; PubMed Central PMCID: PMC3220191.

3: Tan L, Narayan SB, Chen J, Meyers GD, Bennett MJ. PTC124 improves readthrough and increases enzymatic activity of the CPT1A R160X nonsense mutation. J Inherit Metab Dis. 2011 Apr;34(2):443-7. Epub 2011 Jan 21. PubMed PMID: 21253826.

4: Goldmann T, Overlack N, Wolfrum U, Nagel-Wolfrum K. PTC124-mediated translational readthrough of a nonsense mutation causing Usher syndrome type 1C. Hum Gene Ther. 2011 May;22(5):537-47. Epub 2011 Mar 25. PubMed PMID: 21235327.

5: Wilschanski M, Miller LL, Shoseyov D, Blau H, Rivlin J, Aviram M, Cohen M, Armoni S, Yaakov Y, Pugatsch T, Cohen-Cymberknoh M, Miller NL, Reha A, Northcutt VJ, Hirawat S, Donnelly K, Elfring GL, Ajayi T, Kerem E. Chronic ataluren (PTC124) treatment of nonsense mutation cystic fibrosis. Eur Respir J. 2011 Jul;38(1):59-69. Epub 2011 Jan 13. Erratum in: Eur Respir J. 2011 Oct;38(4):996. Pugatch, T [corrected to Pugatsch, T]. PubMed PMID: 21233271.

6: Sermet-Gaudelus I, Boeck KD, Casimir GJ, Vermeulen F, Leal T, Mogenet A, Roussel D, Fritsch J, Hanssens L, Hirawat S, Miller NL, Constantine S, Reha A, Ajayi T, Elfring GL, Miller LL. Ataluren (PTC124) induces cystic fibrosis transmembrane conductance regulator protein expression and activity in children with nonsense mutation cystic fibrosis. Am J Respir Crit Care Med. 2010 Nov 15;182(10):1262-72. Epub 2010 Jul 9. PubMed PMID: 20622033.

7: Finkel RS. Read-through strategies for suppression of nonsense mutations in Duchenne/ Becker muscular dystrophy: aminoglycosides and ataluren (PTC124). J Child Neurol. 2010 Sep;25(9):1158-64. Epub 2010 Jun 2. Review. PubMed PMID: 20519671.

8: Auld DS, Lovell S, Thorne N, Lea WA, Maloney DJ, Shen M, Rai G, Battaile KP, Thomas CJ, Simeonov A, Hanzlik RP, Inglese J. Molecular basis for the high-affinity binding and stabilization of firefly luciferase by PTC124. Proc Natl Acad Sci U S A. 2010 Mar 16;107(11):4878-83. Epub 2010 Mar 1. PubMed PMID: 20194791; PubMed Central PMCID: PMC2841876.

9: Peltz SW, Welch EM, Jacobson A, Trotta CR, Naryshkin N, Sweeney HL, Bedwell DM. Nonsense suppression activity of PTC124 (ataluren). Proc Natl Acad Sci U S A. 2009 Jun 23;106(25):E64; author reply E65. Epub 2009 Jun 8. PubMed PMID: 19506240; PubMed Central PMCID: PMC2700894.

10: Goodier JL, Mayer J. PTC124 for cystic fibrosis. Lancet. 2009 Apr 25;373(9673):1426; author reply 1426-7. PubMed PMID: 19394530.

Additional Information