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2159-33452712017FebNucleic acid therapeuticsNucleic Acid TherFDA Approves Eteplirsen for Duchenne Muscular Dystrophy: The Next Chapter in the Eteplirsen Saga.131-310.1089/nat.2016.0657Aartsma-RusAnnemiekeA1 Leiden University Medical Center , Leiden, the Netherlands .KriegArthur MAM2 Checkmate Pharmaceuticals , Cambridge, Massachusetts.engJournal Article20161208
United StatesNucleic Acid Ther1015627582159-33370Dystrophin0Morpholinos0Oligonucleotides0PRO051AIW6036FASeteplirsenIMAdolescentChildClinical Trials as TopicDose-Response Relationship, DrugDrug ApprovalDystrophingeneticsExonsHumansMorpholinostherapeutic useMuscular Dystrophy, Duchennedrug therapyeconomicsOligonucleotidestherapeutic useTime FactorsTreatment OutcomeUnited StatesUnited States Food and Drug AdministrationWalk TestYoung Adultoligonucleotideregulatorysplicing modulationtherapyA.A.R. report being employed by LUMC, which has patents on exon skipping technology. As a coinventor of some of these patents, A.A.R. may receive a share of potential royalties. A.A.R. reports being an ad hoc consultant for BioMarin, PTC, BMS, and Summit. Remuneration for these activities and speaker honoraria for BioMarin and PTC-organized satellite symposia go to LUMC. A.M.K. was previously employed as Sarepta's CSO and has been a consultant to companies working on RNA and DMD therapeutics.201612960201842160201612960201721ppublish27929755PMC531246010.1089/nat.2016.0657www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm521263.htm Goemans NM, Tulinius M, van den Hauwe M, Kroksmark AK, Buyse G, Wilson RJ, van Deutekom JC, de Kimpe SJ, Lourbakos A, and Campion G. (2016). Long-term efficacy, safety, and pharmacokinetics of drisapersen in Duchenne muscular dystrophy: results from an open-label extension study. PLoS One 11:e0161955.PMC501019127588424http://investors.bmrn.com/releasedetail.cfm?ReleaseID=973536 Mendell JR, Rodino-Klapac LR, Sahenk Z, Roush K, Bird L, Lowes LP, Alfano L, Gomez AM, Lewis S, et al. ; Eteplirsen Study Group. (2013). Eteplirsen for the treatment of Duchenne muscular dystrophy. Ann Neurol 74:637–64723907995www.fda.gov/downloads/advisorycommittees/committeesmeetingmaterials/drugs/peripheralandcentralnervoussystemdrugsadvisorycommittee/ucm481912.pdf www.fda.gov/downloads/advisorycommittees/committeesmeetingmaterials/drugs/peripheralandcentralnervoussystemdrugsadvisorycommittee/ucm497064.pdf Mendell JR, Goemans N, Lowes LP, Alfano LN, Berry K, Shao J, Kaye EM, and Mercuri E; Eteplirsen Study Group and Telethon Foundation DMD Italian Network. 2016). Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy. Ann Neurol 79:257–271PMC506475326573217https://clinicaltrials.gov/ct2/show/NCT02255552 Presented by Prof. Francesco Muntoni at the Sarepta sponsored satellite symposium of the World Muscle Society, October 2016, Granada, Spainwww.accessdata.fda.gov/drugsatfda_docs/nda/2016/206488_summary%20review_Redacted.pdf www.cdmd.ucla.edu/files/view/FDA_ETEPLIRSEN_LETTER_02242016.pdf www.srnnews.com/anthem-says-will-not-cover-first-fda-approved-duchenne-drug www.drugs.com/nda/eteplirsen_130725.html Straub V, Balabanov P, Bushby K, Ensini M, Goemans N, De Luca A, Pereda A, Hemmings R, Campion G, et al. (2016). Stakeholder cooperation to overcome challenges in orphan medicine development: the example of Duchenne muscular dystrophy. Lancet Neurol 15:882–89027302365