Global InformationOmigapil information
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| Other names | TCH346, CGP3466B |
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| Formula | C19H17NO |
| Molar mass | 275.351 g·mol−1 |
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Omigapil (TCH346 or CGP3466) is a drug that was developed by Novartis and tested in clinical trials for its ability to help treat Parkinson's disease (PD)[1] and amyotrophic lateral sclerosis (ALS).[2] The development for PD and ALS have been terminated due to lack of benefit, but Santhera Pharmaceuticals bought the compound for development for the treatment of congenital muscular dystrophy (CMD).[3][4][5][6]
Omigapil was first synthesized at Ciba-Geigy, Basel, Switzerland. Santhera Pharmaceuticals has since taken over production of omigapil and preclinical trials for CMD. In May 2008, omigapil was granted orphan designation to commence clinical trials for.[7] Pharmacokinetic trials are scheduled to commence enrollment in the second half of 2012 to determine the appropriate pharmacokinetic profile of the drug for children with laminin-α2-deficient congenital muscular dystrophy (MDC1A) and collagen VI related myopathy. Santhera Pharmaceuticals will use the phase 1 clinical trial to determine if the drug is safe and acts with the same pharmacokinetic profile in children as it does in adults. The impending clinical trial will take place in the United States at the National Institute of Neurological Disorders and Stroke/National Institute of Health(NNDCS/NINDS) (Bethesda, Maryland) and in the United Kingdom at Great Ormond Street Hospital (UCL).[8]
- ^ Olanow CW, Schapira AH, LeWitt PA, Kieburtz K, Sauer D, Olivieri G, et al. (December 2006). "TCH346 as a neuroprotective drug in Parkinson's disease: a double-blind, randomised, controlled trial". The Lancet. Neurology. 5 (12): 1013–1020. doi:10.1016/S1474-4422(06)70602-0. PMID 17110281. S2CID 1562331.
- ^ Clinical trial number NCT00036413 for "A 12-Week, Multicenter, Safety and Dose-Ranging Study of 3 Oral Doses of TCH346 in Patients With Amyotrophic Lateral Sclerosis" at ClinicalTrials.gov
- ^ "Santhera to Test Compound in CMD". Archived from the original on 2011-10-25. Retrieved 2011-09-18.
- ^ Meinen S, Lin S, Thurnherr R, Erb M, Meier T, Rüegg MA (August 2011). "Apoptosis inhibitors and mini-agrin have additive benefits in congenital muscular dystrophy mice". EMBO Molecular Medicine. 3 (8): 465–479. doi:10.1002/emmm.201100151. PMC 3377088. PMID 21674808.
- ^ Olanow CW, Schapira AH, LeWitt PA, Kieburtz K, Sauer D, Olivieri G, et al. (December 2006). "TCH346 as a neuroprotective drug in Parkinson's disease: a double-blind, randomised, controlled trial". The Lancet. Neurology. 5 (12): 1013–1020. doi:10.1016/S1474-4422(06)70602-0. PMID 17110281. S2CID 1562331.
- ^ "Santhera Pharmaceuticals: Development of SNT-317 (INN: omigapil) in CMD and other neuromuscular diseases" (PDF). Archived from the original (PDF) on 2016-03-04. Retrieved 2012-05-07.
- ^ Committee for Orphan Medicinal Products. "Public summary of opinion on orphan designation: Omigapil maleate for the congenital muscular dystrophy with merosin (laminin alpha 2) deficiency" (PDF). EMA/COMP/204694/2008 Rev.1.
- ^ Muscular Dystrophy Campaign. "New collaboration to support omigapil clinical trial for congenital muscular dystrophy". Archived from the original on 5 May 2012. Retrieved 13 May 2012.