DMD治療薬関連サイト

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核酸医薬来年日本でも相次ぎ承認へ―初の“国産”日本新薬の筋ジス薬も. AnsweresNews (2018/10/9)

Eteplirsen
株価と科学の関係
Eteplirsen続報
Exondys (eteplirsen): Assessment by EMA. REFUSED This medicine was refused authorisation for use in the European Union.

ビルトラルセンViltolarsen
Viltolarsen (NS-065/NCNP-01). Muscular Dystrophy News Today. This concise review says that a Phase 3 clinical trial (NCT04060199), called RACER53, is expected to conclude in late 2024.
ビルトラルセン（NS-065/NCNP-01）の作用機序（日本語の解説）
米国およびカナダでの第二相臨床試験結果（2018/10/3）

Golodirsen
Sarepta Therapeutics Receives Complete Response Letter from the US FDA for Golodirsen New Drug Application (August 19, 2019)
The CRL generally cites two concerns: the risk of infections related to intravenous infusion ports and renal toxicity seen in pre-clinical models of golodirsen and observed following administration of other antisense oligonucleotides. Renal toxicity with golodirsen was observed in pre-clinical models at doses that were ten-fold higher than the dose used in clinical studies. Renal toxicity was not observed in Study 4053-101, on which the application for golodirsen was based.

FDA, in Reversal, Approves Vyondys 53 to Treat Duchenne Patients with Exon 53 Mutations. Muscular Dystrophy News Today. (DECEMBER 13, 2019)
This accelerated approval comes less than four months after the FDA issued a complete response letter rejecting Sarepta Therapeutics’ application for approval due to safety concerns. Sarepta filed an appeal, and said it met with officials and resolved issues raised by the agency before resubmitting its request, which led to Thursday’s decision.

米FDA　DMD治療薬Vyondysを承認ミクスオンライン　2019/12/26

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