SPINRAZA (Nusinersen)

On December 23, 2016, the FDA announced that it has approved Spinraza for SMA, making it the first-ever approved therapy for SMA.

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About Spinraza

SMA is caused by a mutation in the survival motor neuron gene 1 (SMN1). In a healthy person, this gene produces a protein—called survival motor neuron protein or SMN protein—that is critical to the function of the nerves that control our muscles. Without it, those nerve cells cannot properly function and eventually die, leading to debilitating and often fatal muscle weakness.

All patients with SMA have at least one copy of survival motor neuron gene 2 (SMN2), often referred to as the SMA "backup gene." Due to a splicing error, most of the SMN protein made by SMN2 is missing an important piece, called exon 7. Antisense drugs are small snippets of synthetic genetic material that bind to ribonucleic acid (RNA), so they can be used to fix splicing errors in genes such as SMN2. Spinraza is antisense oligonucleotide that targets SMN2, causing it to make more complete SMN protein.

Spinraza was first known as IONIS-SMNRx, then nusinersen.

From 2003 to 2006, Cure SMA provided the very first research funding needed to begin investigation into this therapeutic approach. We would like to thank and acknowledge Cold Spring Harbor Laboratory (CSHL) and the University of Massachusetts Medical School for generating critical intellectual property for the program that was licensed to Ionis Pharmaceuticals. We specifically thank Drs. Adrian Krainer, Yimin Hua and colleagues at CSHL for years of dedication to and hard work on the preclinical development of Spinraza for SMA, and Drs. Ravindra Singh and Elliot Androphy for their work funded by Cure SMA in originally identifying the ISSN1 gene sequence, which is the sequence targeted in Spinraza.

After the preclinical work and early clinical testing was complete, Ionis Pharmaceuticals partnered with Biogen to launch a number of clinical trials testing Spinraza.

On August 12, 2016, Biogen and Ionis announced that SpinrazaTM had met its primary endpoint in an interim analysis of ENDEAR, a double-blind, sham procedure-controlled trial testing Spinraza in infants with SMA type I. As a result, the trial was ended early and all participants were transitioned into SHINE, an open-label extension trial.

Also as a result of these findings, Biogen initiated a New Drug Application (NDA) with the FDA, as well as international regulatory filings. On October 28, the FDA announced that it had accepted the NDA for Spinraza with priority review.

On November 7, 2016, Biogen and Ionis announced that Spinraza had also met its primary endpoint in an interim analysis of CHERISH, a double-blind, sham procedure-controlled trial testing Spinraza in children with SMA type II. As a result, the trial was ended early and all participants were transitioned into SHINE, an open-label extension trial. Biogen also shared this new data with the FDA and other regulatory groups around the world.

On December 23, 2016, the FDA announced that it has approved Spinraza for SMA, making it the first-ever approved therapy for SMA.

Biogen is now responsible for all Spinraza development, regulatory and commercialization activities and costs.

Timeline of Events

  • 2003 - 2006: Cure SMA makes over $500,000 in seed grants to fund the therapeutic approach that led to Spinraza.
  • July 2010: Ionis (then known as Isis Pharmaceuticals) licenses the intellectual property to begin development of Spinraza.
  • December 2011: Ionis initiates a Phase 1 clinical trial of Spinraza.
  • January 2012: Biogen and Ionis enter into a partnership agreement to continue developing Spinraza.
  • April 2013: Ionis begins testing Spinraza in Phase 2 clinical trials.
  • August 2014: Ionis and Biogen launch ENDEAR.
  • November 2014: Ionis and Biogen launch CHERISH.
  • March 2015: Biogen and Ionis launch NURTURE, a Phase 2 trial testing Spinraza in infants genetically diagnosed with SMA but not yet showing symptoms.
  • August 12, 2016: Biogen and Ionis announce their intention to initiate regulatory filings for Spinraza, after the drug meets its primary endpoint in an interim analysis of ENDEAR.
  • September 26, 2016: Biogen and Ionis announce that they have completed their rolling NDA submission to the FDA and EMA.
  • October 28, 2016: Biogen and Ionis announce that the FDA has accepted their New Drug Application with priority review.
  • November 7, 2016: Biogen and Ionis announce that Spinraza also met its primary endpoint in an interim analysis of CHERISH.
  • December 23, 2016: The FDA announces that it has approved Spinraza for SMA.

Expanded Access Program - International

Expanded access for Spinraza is currently being offered at the following sites:

Australia, New South Wales
Sydney Children's Hospital
Randwick, New South Wales, Australia, 2031

Australia, Victoria
The Royal Children's Hospital Melbourne
Parkville, Victoria, Australia, 3052

Austria
Kaiser Franz Josef Spital
Vienna, Austria 1100 

Belgium
CHU Brugman
Brussels, Belgium, 1020

Canada, British Columbia
BC Children's Hospital
Vancouver, British Columbia, Canada, V6Z 2H3

Canada, Ontario
Children's Hospital at London Health Sciences Centre    
London, Ontario, Canada, N6A 5A5

Canada, Quebec
Montreal Children's Hospital
Montreal, Quebec, Canada, H4A 3J1

Finland
Turku University Central Hospital
Turku, Turku and Pori County, Finland, 20521

France
Hopitaux Universitaires de Strasbourg
Strasbourg, Alsace, France, 67091

Bicetre Hospital
Le Kremlin-Bicetre, Paris, France, 94270

Germany
Charite - Universitatsklinkum Berlin
Berlin, Brandenburg, Germany, 10117

DRK - Schwesternschaft Berlin
Berlin, Brandenburg, Germany, 12247

Universitätsklinikum Freidburg
Breisgau, Freidburg, Germany 79106

Universitatsklinikum GieBen und Marburg
Marburg, Hesse, Germany, 35043

Apotheke des Universitatsklinikum Munster
Munster, North Rhine-Westphalia, Germany, 48149

Universitätsklinikum Essen
Essen, Germany, 45147

Universitatsklinikum Hamburg-Eppendorft
Hamburg, Germany, 20246

Ireland
Temple Street Children's University Hospital
Dublin, Leinster, Ireland, Dublin 1 

Israel
Tel Aviv Sourasky Medical Center
Tel Aviv, Israel, 64239

Italy
Policlinico Agostino Gemelli
Rome, Latium, Italy, 00168

Ospedale Pediatrico Bambino Gesù
Vatican City, Rome, Lazio, Italy, 4 - 00165

Istituto Giannina Gaslini
Genova, Liguria, Italy, 16147

Norway
Kvinne og Barneklinikken, Oslo Universitetssy
Oslo, Norway, 0424

Poland
The Children's Memorial Health Institute
Warsaw, Poland, Al. Dzieci Polskich 20 

Portugal
Dona Estefania Hospital
Lisbon, Lisboa, Portugal, 1169-045 

Slovenia
University Medical Centre Ljubljana
Ljubljana, Slovenia, Zaloska 2 1000

Spain
Vall d'Hebron University Hospital
Barcelona, Spain, 08035 

Hospital Universitario La Paz
Madrid, Spain, 28046 

Sweden
The Queen Silvia Children's Hospital
Vitaminvägen, Gothenburg, Sweden, 2141685

United Kingdom
Royal Victoria Hospital
Belfast City, Belfast, United Kingdom, BT12 6BA

Ninewells Hospital
Dundee, Scotland, United Kingdom, DD1 9SY

The Newcastle upon Tyne Hospitals NHS Foundation Trust
Newcastle upon Tyne, Tyne and Wear, United Kingdom, NE7 7DN

More information on this program can be found at on the clinical trials registry.

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