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Golodirsen: First Approval.

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  • 1Springer Nature, Private Bag 65901, Mairangi Bay, Auckland, 0754, New Zealand. dru@adis.com.

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Golodirsen is a new treatment for Duchenne muscular dystrophy (DMD) that works by helping skip exon 53. It received US approval in 2019 for specific DMD patients based on positive clinical trial results.

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Area of Science:

  • Pharmacology
  • Genetics
  • Neurology

Background:

  • Duchenne muscular dystrophy (DMD) is a progressive genetic disorder.
  • Current treatments for DMD are limited.
  • Golodirsen offers a novel therapeutic approach for DMD.

Purpose of the Study:

  • To summarize the development of golodirsen.
  • To highlight the milestones leading to its first approval for DMD.

Main Methods:

  • Golodirsen is an antisense oligonucleotide of the phosphorodiamidate morpholino oligomer (PMO) subclass.
  • It is designed to induce exon 53 skipping in the DMD gene.
  • Development involved phase I/II clinical trials.

Main Results:

  • Golodirsen received its first global approval in the USA in December 2019.
  • Approval was for the treatment of DMD in patients with specific DMD gene mutations amenable to exon 53 skipping.
  • Positive results from a phase I/II clinical trial supported the approval.

Conclusions:

  • Golodirsen represents a significant advancement in DMD treatment.
  • The drug's development highlights the potential of exon skipping therapies.
  • Golodirsen is currently in phase III clinical development worldwide.