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Spinraza a bibliography

Spinraza, also known as nusinersen, ISIS-SMNRx or SMNRx, is "designed to boost the production of the protein SMN."[1] and is being used to treat spinal muscular atrophy, a rare pediatric disease.[1] SMNRx was discovered in a collaboration between Adrian Krainer at Cold Spring Harbor Laboratory and ENDEAR Isis Pharmaceuticals, Inc., now known as Ionis Pharmaceuticals.[2][3][4][5] Early academic research, with a Cure SMA donation of $500,000, led to the SMNRx's development.[6] Cure SMA, a patient advocacy group, invested $500,000 in early academic research that led to the development of Spinraza. [6] Because it is treating an orphan drug that is also a specialty drug, A strategic alliance between Biogen IdecBiogen, which is licensing Spinraza from Ionis Pharmaceuticals, helped SMNRx to advance quickly from preclinical trial through late-stage studies within just a few years. Following the successful 2015 clinical trial, involving 82 infants in which "40 percent of babies on the drug reached milestones such as sitting, crawling and walking,"[6] SMNRX benefited from the FDA's priority review[6]

Development

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Cure SMA, Cure SMA, a patient advocacy group, invested $500,000 in early academic research that led to the development of Spinraza. [6]a patient advocacy group that invested $500,000 in early academic research that led to the development of Spinraza. [6]

"We believe the Spinraza pricing decision is likely to invite a storm of criticism, up to and including presidential tweets."

— Geoffrey C. Porges, an analyst for Leerink Partners in a note to investors on December 30, 2016

Clinical trials

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""The successful advancement of ISIS-SMNRx from a preclinical drug candidate to late-stage studies within just a few years reflects the effectiveness of our strategic alliance with Biogen Idec..."

— August 2014

ENDEAR Isis Pharmaceuticals, Inc. Now Ionis Pharmaceuticals, began "the initiation of a pivotal Phase III study evaluating ISIS-SMNRx in infants with spinal muscular atrophy (SMA)" in the fall of 2014.[7][1] ENDEAR was a "Phase III study of ISIS-SMNRx. ENDEAR was "a randomized, double-blind, sham-procedure controlled thirteen month study in approximately 110 infants diagnosed with SMA and was "the first ever Phase III trial for a new drug developed to treat the underlying cause of SMA in the US, with original funding from Cure SMA."[7][8][9][10]

There was a clinical trial with 82 infants with spinal muscular atrophy, starting in ??, "40 percent of babies on the drug reached milestones such as sitting, crawling and walking." As a result of this trial, "The F.D.A. approved the drug months ahead of time and, because the drug treats a rare pediatric disease, Priority review (FDA), granted Biogen a special voucher that it can use to gain priority review of a future drug that would not otherwise qualify for the program." One of the investigators of the trial was Dr. John Brandsema of the Children’s Hospital of Philadelphia.[6]

In December 2016 the F.D.A. approved the According to the New York Times, Spinraza, "will be among the most expensive drugs in the world", with an estimated cost $750,000 in the first year of treatment and "about $375,000 annually after that."[6] Peak annual sales of nusinersen "could reach around $1.5 billion, triggering up to $150 million in additional milestone payments and a royalty percentage up to the mid-teens."[11]

"In December 2016, Ionis' drug nusinersen (Spinraza) was approved by the FDA.[12] It had been discovered in a collaboration between Adrian Krainer at Cold Spring Harbor Laboratory and Ionis (then Isis)[13][14][4][15] and preclinical work was done at University of Massachusetts.[16] The drug was initially developed by Ionis, which partnered with Biogen on development starting in 2012, and in 2015 Biogen acquired an exclusive license to the drug for a $75 million license fee, milestone payments up to $150M, and tiered royalties up to the mid-teens; Biogen also paid for all development subsequent to taking the license.[17] The license to Biogen included licenses to intellectual property that Ionis had acquired from Cold Spring Harbor and University of Massachusetts.[18]

Mary K. Schroth, who "has previously worked as a paid consultant to Biogen, which is selling the drug" is now promoting the drug.[6][19]

Potential Categories

Health insuranceement‎ Pharmaceutical industry‎ Pharmaceuticals policy Health care industry trade groups‎

  • [Category:Life sciences industry Life sciences industry]

+**Pharmaceutical industry

In 1982, when the beginning of the biotechnology revolution blurred the line between a drug and a biologic or a device and a biologic, the Bureau of Drugs was merged with the FDA's Bureau of Biologics to form the Center for Drugs and Biologics during an agency-wide reorganization under Commissioner Arthur Hayes.[23] Transfer of products from Center for Biologics Evaluation and Research (CBER) to CDER was formalized in early 2004.[23] This reorganization similarly merged the bureaus responsible for medical devices and radiation control into the Center for Devices and Radiological Health. 


***Category:Orphan drugs


References

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  1. ^ a b c http://www.fiercebiotech.com/r-d/isis-rolls-into-phase-iii-an-orphan-drug-as-it-waits-on-biogen
  2. ^ Garber, K (11 October 2016). "Big win possible for Ionis/Biogen antisense drug in muscular atrophy". Nature biotechnology. 34 (10): 1002–1003. doi:10.1038/nbt1016-1002. PMID 27727217.
  3. ^ Wadman, Meredith (23 December 2016). "Updated: FDA approves drug that rescues babies with fatal neurodegenerative disease". Science.
  4. ^ a b Offord, Catherine (December 1, 2016). "Oligonucleotide Therapeutics Near Approval". The Scientist.
  5. ^ Tarr, Peter (24 December 2016). "CSHL FDA approval of life-saving SMA drug is hailed by its researcher-inventor at CSHL". Cold Spring Harbor Laboratory.
  6. ^ a b c d e f g h i Katie Thomas (December 30, 2016), Costly Drug for Fatal Muscular Disease Wins F.D.A. Approval, New York Times, retrieved December 31, 2016 {{citation}}: Italic or bold markup not allowed in: |publisher= (help)
  7. ^ a b http://www.curesma.org/news/isis-begins-phase-iii.html Isis Begins Phase III Clinical Trial BY CURE SMA | PUBLISHED ON SEPTEMBER 22, 2014
  8. ^ https://vector.childrenshospital.org/2015/12/rallying-a-backup-gene-could-boost-strength-in-spinal-muscular-atrophy/
  9. ^ http://www.curesma.org/news/isis-begins-phase-iii.html
  10. ^ http://www.ema.europa.eu/docs/en_GB/document_library/Other/2016/12/WC500217553.pdf
  11. ^ Cory Renauer (October, 2016), Better Buy: Anavex Life Sciences Corp. vs. Ionis Pharmaceuticals, Inc., retrieved December 31, 2016 {{citation}}: Check date values in: |date= (help)
  12. ^ Grant, Charley (2016-12-27). "Surprise Drug Approval Is Holiday Gift for Biogen". Wall Street Journal. ISSN 0099-9660. Retrieved 2016-12-27.
  13. ^ Garber, K (11 October 2016). "Big win possible for Ionis/Biogen antisense drug in muscular atrophy". Nature biotechnology. 34 (10): 1002–1003. doi:10.1038/nbt1016-1002. PMID 27727217.
  14. ^ Wadman, Meredith (23 December 2016). "Updated: FDA approves drug that rescues babies with fatal neurodegenerative disease". Science.
  15. ^ Tarr, Peter (24 December 2016). "CSHL FDA approval of life-saving SMA drug is hailed by its researcher-inventor at CSHL". Cold Spring Harbor Laboratory.
  16. ^ "Therapeutic Approaches". www.curesma.org. Cure SMA. Retrieved 1 January 2017.
  17. ^ "Biogen Shells Out $75M to Develop Ionis' Nusinersen after Positive Phase III Results", Genetic Engineering News, August 1, 2016
  18. ^ "Press release: Biogen and Ionis Pharmaceuticals Report Nusinersen Meets Primary Endpoint at Interim Analysis of Phase 3 ENDEAR Study in Infantile-Onset Spinal Muscular Atrophy". Biogen. August 1, 2016.
  19. ^ http://www.uwhealth.org/findadoctor/profile/mary-k-schroth-md/7433
  20. ^ a b Gleason PP, Alexander GC, Starner CI, Ritter ST, Van Houten HK, Gunderson BW, Shah ND (Sep 2013). "Health plan utilization and costs of specialty drugs within 4 chronic conditions". Journal of Managed Care Pharmacy. 19 (7): 542–8. doi:10.18553/jmcp.2013.19.7.542. PMID 23964615.
  21. ^ Thomas, Kate; Pollack, Andrew (15 July 2015). "Specialty Pharmacies Proliferate, Along With Questions". Sinking Spring, Pa.: New York Times. Retrieved 5 October 2015.
  22. ^ Murphy, Chad O. "Specialty Pharmacy Managed Care Strategies" (PDF). Retrieved 24 September 2015.
  23. ^ a b Reid, Ken. "CBER and Center for Drug Evaluation and Research (CDER) have long history of being lumped together and split up." Bioresearch Monitoring Alert Sept. 2002. page 4.
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