Global InformationEzutromid information
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| Formula | C19H15NO3S |
| Molar mass | 337.39 g·mol−1 |
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Ezutromid is an orally administered small molecule utrophin modulator involved in a Phase 2 clinical trial produced by Summit Therapeutics for the treatment of Duchenne muscular dystrophy (DMD).[1][2] DMD is a fatal x-linked recessive disease affecting approximately 1 in 5000 males and is a designated orphan disease by the FDA and European Medicines Agency.[3] Approximately 1/3 of the children obtain DMD as a result of spontaneous mutation in the dystrophin gene and have no family history of the disease.[3] Dystrophin is a vital component of mature muscle function, and therefore DMD patients have multifarious forms of defunct or deficient dystrophin proteins that all manifest symptomatically as muscle necrosis and eventually organ failure.[3][4] Ezutromid is theorized to maintain utrophin, a protein functionally and structurally similar to dystrophin that precedes and is replaced by dystrophin during development.[3][5] Utrophin and dystrophin are reciprocally expressed, and are found in different locations in a mature muscle cell.[4][6] However, in dystrophin-deficient patients, utrophin was found to be upregulated and is theorized to replace dystrophin in order to maintain muscle fibers.[7] Ezutromid is projected to have the potential to treat all patients suffering with DMD as it maintains the production of utrophin to counteract the lack of dystrophin to retard muscle degeneration.[7][8] Both the FDA and European Medicines Agency has given ezutromid an orphan drug designation.[5][9] The FDA Office of Orphan Products and Development offers an Orphan Drug Designation program (ODD) that allows drugs aimed to treat diseases that affect less than 200,000 people in the U.S. monetary incentives such as a period of market exclusivity, tax incentives, and expedited approval processes.[5][10]
The Phase 2 clinical trial was ended in 2018 and the medication discontinued after it failed to show any benefit in slowing the disease.[11]
- ^ "About Summit Therapeutics - Summit". Summit. Retrieved 2016-11-14.
- ^ Clinical trial number NCT02858362 for "PoC Study to Assess Activity and Safety of SMT C1100 (Ezutromid) in Boys With DMD" at ClinicalTrials.gov
- ^ a b c d "Duchenne Muscular Dystrophy - Summit". Summit. Archived from the original on 2016-11-15. Retrieved 2016-11-14.
- ^ a b Ricotti V, Spinty S, Roper H, Hughes I, Tejura B, Robinson N, et al. (2016-01-01). "Safety, Tolerability, and Pharmacokinetics of SMT C1100, a 2-Arylbenzoxazole Utrophin Modulator, following Single- and Multiple-Dose Administration to Pediatric Patients with Duchenne Muscular Dystrophy". PLOS ONE. 11 (4): e0152840. Bibcode:2016PLoSO..1152840R. doi:10.1371/journal.pone.0152840. PMC 4824384. PMID 27055247.
- ^ a b c "Potential DMD Therapy, Ezutromid, Shows Promise in Upgraded Form". Retrieved 2016-11-14.
- ^ Janghra N, Morgan JE, Sewry CA, Wilson FX, Davies KE, Muntoni F, Tinsley J (2016-03-14). "Correlation of Utrophin Levels with the Dystrophin Protein Complex and Muscle Fibre Regeneration in Duchenne and Becker Muscular Dystrophy Muscle Biopsies". PLOS ONE. 11 (3): e0150818. Bibcode:2016PLoSO..1150818J. doi:10.1371/journal.pone.0150818. PMC 4790853. PMID 26974331.
- ^ a b "Home - Summit". Summit. Retrieved 2016-11-14.
- ^ Werther CA (2016). Ezutromid Has the Potential to Treat All Duchenne Patients; Initiating Coverage With a Buy. H.C. Wainwright & Co. pp. 1–29.
- ^ "Search Orphan Drug Designations and Approvals". www.accessdata.fda.gov. Retrieved 2016-11-14.
- ^ Office of the Commissioner. "Developing Products for Rare Diseases & Conditions". www.fda.gov. Retrieved 2016-11-14.
- ^ Inacio P (2018-06-29). "Summit Therapeutics Ends Development of Ezutromid Therapy for DMD After Trial Failure". Muscular Dystrophy News. Retrieved 2019-11-17.