User:Oceanflynn/sandbox/Spinraza a bibliography

Spinraza a bibliography

Spinraza, also known as nusinersen, ISIS-SMNRx or SMNRx, is "designed to boost the production of the protein SMN." and is being used to treat spinal muscular atrophy, a rare pediatric disease. SMNRx was discovered in a collaboration between Adrian Krainer at Cold Spring Harbor Laboratory and ENDEAR Isis Pharmaceuticals, Inc., now known as Ionis Pharmaceuticals. Early academic research, with a Cure SMA donation of $500,000, led to the SMNRx's development. Cure SMA, a patient advocacy group, invested $500,000 in early academic research that led to the development of Spinraza. 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," SMNRX benefited from the FDA's priority review

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

""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
"""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. 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."

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.

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." 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."

"In December 2016, Ionis' drug nusinersen (Spinraza) was approved by the FDA. It had been discovered in a collaboration between Adrian Krainer at Cold Spring Harbor Laboratory and Ionis (then Isis)  and preclinical work was done at University of Massachusetts.  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. The license to Biogen included licenses to intellectual property that Ionis had acquired from Cold Spring Harbor and University of Massachusetts.

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

Potential Categories
 * Healthcare industry
 * Health care brands
 * Businesspeople in the healthcare industry
 * Health care companies


 * Pharmaceutical companies

Health insuranceement‎ Pharmaceutical industry‎ Pharmaceuticals policy Health care industry trade groups‎ +**Pharmaceutical industry
 * Biopharmaceutical companies
 * Biopharmaceuticals
 * Specialty drugsSpecialty drugs, a recent classification of pharmaceuticals, are high-cost drugs that are often biologics.
 * [Category:Life sciences industry Life sciences industry]
 * FDA Priority review
 * Pharmaceuticals policy
 * Orphan drug companies

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. Transfer of products from Center for Biologics Evaluation and Research (CBER) to CDER was formalized in early 2004. This reorganization similarly merged the bureaus responsible for medical devices and radiation control into the Center for Devices and Radiological Health.
 * Portal Biotechnology
 * Category:Medical research institutes in the United States


 * Biomedical engineering Biomedicine Genomics Biopharmacology
 * History of biotechnology
 * Biotechnologists
 * DNA sequencing
 * Biotechnology products
 * Recombinant proteins
 * Regulators of biotechnology products


 * Biopharmaceuticals
 * Biopharmaceutical_companies
 * Biopharmaceutical law firms Therapeutic antibodies

***Category:Orphan drugs


 * WikiProject Genetics
 * Genetic disorder stubs
 * Genetic disorder


 * Genetic diseases and disorders
 * Geneticist and evolutionary biologist stubs
 * Moshe Szyf GlaxoSmithKline epigenetics "Szyf is an inventor of dozens of issued and patents in the application process, all the patents relate to “epigenetics” based therapeutics."