Sarepta therapeutics announces fda approval of amondys 45™ (casimersen) injection for the treatment of duchenne muscular dystrophy (dmd) in patients amenable to skipping exon 45

-- amondys 45 is sarepta's third rna exon-skipping treatment for dmd approved in the u.s. ---- commercial distribution of amondys 45 in the u.s. will commence immediately ---- information for patients and clinicians is available at www.sareptassist.com -- cambridge, mass., feb. 25, 2021 (globe newswire) --  sarepta therapeutics, inc. (nasdaq:srpt), the leader in precision genetic medicine for rare diseases, today announced that the u.s. food and drug administration (fda) has approved amondys 45 (casimersen). amondys 45 is an antisense oligonucleotide from sarepta's phosphorodiamidate morpholino oligomer (pmo) platform, indicated for the treatment of duchenne muscular dystrophy (dmd) in patients with a confirmed mutation amenable to exon 45 skipping. this indication is based on a statistically significant increase in dystrophin production in skeletal muscle observed in patients treated with amondys 45, which is reasonably likely to predict clinical benefit for those patients who are exon 45 amenable. consistent with the accelerated approval pathway, the continued approval of amondys 45 may be contingent on confirmation of a clinical benefit in confirmatory trials.
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