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Response to “Railroading at the FDA”

Nature Biotechnology volume 35, pages 207–209 (2017)Cite this article

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A Corrigendum to this article was published on 09 May 2017

This article has been updated

To the Editor:

We comment in response to the recent unsigned editorial published in your November issue regarding the drug Exondys 51 (ref. 1). This editorial, which was also published in Nature Medicine2, followed another critical editorial published in JAMA last October3. Aaron Kesselheim, one of the two co-authors of the JAMA editorial3, is a US Food and Drug Administration (FDA) advisor who voted against the drug approval, information that was provided appropriately as a conflict of interest.

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Change history

  • 30 March 2017

    In the version of this article initially published, F.M. was credited for giving input on study design and data analysis, which he did not. The error has been corrected in the HTML and PDF versions of the article.

References

  1. Anonymous. Nat. Biotechnol. 34, 1078 (2016).

  2. Anonymous. Nat. Med. 22, 1193 (2016).

  3. Kesselheim, A.S. & Avorn, J.A. JAMA 316, 2357–2358 (2016).

    Article  Google Scholar 

  4. Center for Drug Evaluation and Research. Application number 206488Orig1s000: summary review (Center for Drug Evaluation and Research) http://www.accessdata.fda.gov/drugsatfda_docs/nda/2016/206488_summary%20review_Redacted.pdf (16 September 2016).

  5. Kinali, M. et al. Lancet Neurol. 8, 918–928 (2009).

    Article  CAS  Google Scholar 

  6. Cirak, S. et al. Lancet 378, 595–605 (2011).

    Article  CAS  Google Scholar 

  7. Parent Project Muscular Dystrophy. Guidance for industry, Duchenne muscular dystrophy: developing drugs for treatment over the spectrum of disease. http://www.parentprojectmd.org/site/DocServer/Guidance_Document_Submission_-Duchenne_Muscular_Dystrop.pdf?docID=15283

  8. Arechavala-Gomeza, V. et al. Neuropathol. Appl. Neurobiol. 36, 265–274 (2010).

    Article  CAS  Google Scholar 

  9. Taylor, L.E., Kaminoh, Y.J., Rodesch, C.K. & Flanigan, K.M. Neuropathol. Appl. Neurobiol. 38, 591–601 (2012).

    Article  CAS  Google Scholar 

  10. Mendell, J.R. et al. Ann. Neurol. 74, 637–647 (2013).

    Article  CAS  Google Scholar 

  11. Dubowitz, V., Sewry, C.A. & Oldfors, A. Muscle Biopsy, a Practical Approach 3rd edn. 262 (Elsevier, 2013).

    Google Scholar 

  12. Sarepta Therapeutics. Peripheral and Central Nervous System Drugs Advisory Committee eteplirsen briefing document, NDA 206488 (FDA) http://www.fda.gov/downloads/advisorycommittees/committeesmeetingmaterials/drugs/peripheralandcentralnervoussystemdrugsadvisorycommittee/ucm497064.pdf (25 April 2016).

  13. Phelps, S.F. et al. Hum. Mol. Genet. 4, 1251–1258 (1995).

    Article  CAS  Google Scholar 

  14. Li, D., Yue, Y. & Duan, D. Am. J. Pathol. 172, 1332–1341 (2008).

    Article  CAS  Google Scholar 

  15. van Putten, M. et al. FASEB J. 27, 2484–2495 (2013).

    Article  CAS  Google Scholar 

  16. van Putten, M. et al. PLoS One 7, e31937 (2012).

    Article  CAS  Google Scholar 

  17. Hakim, C.H. & Duan, D. Muscle Nerve 46, 948–950 (2012).

    Article  CAS  Google Scholar 

  18. Ricotti, V. et al. J. Neurol. Neurosurg. Psychiatry 87, 149–155 (2016).

    Article  Google Scholar 

  19. Bello, L. et al. Neurology 87, 401–409 (2016).

    Article  Google Scholar 

  20. Anthony, K. et al. Neurology 83, 2062–2069 (2014).

    Article  Google Scholar 

Download references

Acknowledgements

F.M. is supported by the National Institute of Health Research Biomedical Research Centre at Great Ormond Street Hospital for Children NHS Foundation Trust and University College London.

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Authors and Affiliations

  1. Dubowitz Neuromuscular Centre, Great Ormond Street Hospital for Children, Great Ormond Street, London, UK

    Francesco Muntoni

  2. Centre for Comparative Genomics, Murdoch University, Perth, Western Australia, Australia

    Sue Fletcher & Steve Wilton

Authors
  1. Francesco Muntoni
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  2. Sue Fletcher
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  3. Steve Wilton
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Corresponding author

Correspondence to Steve Wilton.

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Competing interests

F.M. was involved in the original investigator-led and independently funded phase 1 and 2A studies that led to the development of Exondys 51. F.M. has received consulting fees from PTC Therapeutics, Sarepta Therapeutics, BioMarin, Roche, Biogen, Italfarmaco, Avexis, Pfizer, Trivorsan, Catabasis and Wave Therapeutics. S.F. and S.W. are named inventors on patents describing Exondys 51 and other exon skipping compounds, which are licensed to Sarepta Therapeutics by the University of Western Australia. S.F and S.W. are consultants to Sarepta Therapeutics.

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Muntoni, F., Fletcher, S. & Wilton, S. Response to “Railroading at the FDA”. Nat Biotechnol 35, 207–209 (2017). https://doi.org/10.1038/nbt.3819

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