• Nusinersen (Spinraza ® ) has been available for reimbursement under hospital pricing approval since July 2019. (hse.ie)
  • The prescribing of nusinersen (Spinraza ® ) under hospital pricing approval is confined to consultant neurologists with experience in the diagnosis and management of Spinal Muscular Atrophy (SMA) in specialist centre(s) in Ireland, who have agreed to the terms of this MAP and have been approved by the HSE. (hse.ie)
  • A copy of the HSE-Managed Access Protocol for nusinersen (Spinraza ® ) and the application form for individual reimbursement approval can be found in the Related Files section below. (hse.ie)
  • Due to the complexity of the management of this rare disease, the Committee reiterates that it recommends that decisions to initiate or discontinue treatment with SPINRAZA (nusinersen) be taken at multidisciplinary review meetings in neuromuscular diseases reference and expertise centres. (has-sante.fr)
  • Biogen Inc. has announced new interim results from NURTURE, an ongoing open-label, single-arm efficacy and safety phase 2 study of Spinraza (nusinersen) in 25 presymptomatic infants with 5q SMA. (acnr.co.uk)
  • CAMBRIDGE, Mass. & CARLSBAD, Calif.--(BUSINESS WIRE) --Biogen (NASDAQ:BIIB) and Ionis Pharmaceuticals (NASDAQ:IONS) announced that SPINRAZATM (nusinersen), an investigational treatment for spinal muscular atrophy (SMA), met the primary endpoint at the interim analysis of CHERISH, the Phase 3 study evaluating SPINRAZA in later-onset (consistent with Type 2) SMA. (mda.org)
  • Today I received the fantastic news that non-ambulatory patients with Spinal Muscular Atrophy (SMA) type III will now be able to access nusinersen (Spinraza), a life-changing treatment, via NHS delivery services https://www.nice.org.uk/…/nice-announces-more-people… . (kerrymccarthy.org)
  • Spinraza (nusinersen) is an injection therapy widely approved for spinal muscular atrophy (SMA). (smanewstoday.com)
  • A pivotal factor in these improved outcomes has been the introduction of nusinersen (marketed as Spinraza), an injectable drug that targets the underlying cause of SMA. (ipharmacenter.com)
  • Genentech announced new two-year data of the JEWELFISH study, evaluating Evrysdi (risdiplam) in children pre-treated with other approved SMA treatments, including Spinraza (nusinersen) and Zolgensma (onasemnogene abeparvovec). (ipharmacenter.com)
  • This review assesses nusinersen (Spinraza), 4 mg/mL solution for intrathecal injection. (canjhealthtechnol.ca)
  • Biogen and Ionis have been awarded the prestigious 2017 Prix Galien USA Award for Best Biotechnology Product for SPINRAZA (nusinersen). (news-medical.net)
  • SPINRAZA® is the brand name for Nusinersen, a drug that was approved by the Food and Drug Administration (FDA) on December 23rd, 2016. (ufl.edu)
  • CAMBRIDGE, Mass.--( BUSINESS WIRE )-- Biogen (NASDAQ: BIIB) will present robust efficacy and safety data from Phase 2 and 3 SPINRAZA ® (nusinersen) studies at the Cure SMA 2017 Annual SMA Conference in Orlando, Fl, June 29 - July 2, 2017. (biogen.com)
  • One medicine, Nusinersen (or Spinraza™), is given through a spinal tap . (kidshealth.org)
  • Then Biogen discovered the wildly successful Spinraza (Nusinersen), which treats spinal muscular atrophy (SMA), a rare genetic disease that is fatal if untreated. (genengnews.com)
  • In December 2016, nusinersen (Spinraza) became the first FDA approved treatment for SMA1. (rarediseases.org)
  • Nusinersen (Spinraza) is approved by the Food and Drug Administration (FDA) for use in children and adults. (healthline.com)
  • SPINRAZA ® also known as nusinersen is indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients. (reachmd.com)
  • Over 50 countries have approved Spinraza (nusinersen), but not all have made it available to the patients who need it. (everyone.org)
  • Nusinersen, also called Spinraza and made by Biogen, is the first treatment that targets the underlying cause of spinal muscular atrophy (SMA). (england.nhs.uk)
  • Perspectives on Spinraza (Nusinersen) Treatment Study: Views of Individuals and Parents of Children Diagnosed with Spinal Muscular Atrophy. (cdc.gov)
  • These results, along with our successful trial in infantile-onset SMA, reinforce the potential of SPINRAZA to benefit a broad range of SMA patients," said Michael Ehlers, M.D., Ph.D., executive vice president, head of Research and Development at Biogen. (mda.org)
  • Biogen is preparing for the potential launch of SPINRAZA in the U.S. possibly as early as the end of 2016 or the first quarter of 2017. (mda.org)
  • The National Institute for Health and Care Excellence (NICE) has held talks over whether to recommend Spinraza, developed by pharmaceutical firm Biogen, having last year said the cost was too high. (mirror.co.uk)
  • Ionis first developed theantisense oligonucleotide drug, but Biogen exercised its option to commercialize Spinraza in August after positive results were announced from a late-stage clinical study. (foxbusiness.com)
  • Ionis has licensed Spinraza to Biogen BIIB. (yahoo.com)
  • Biogen is responsible for commercializing Spinraza, approved for treating spinal muscular atrophy, or SMA, worldwide. (yahoo.com)
  • Spinraza product sales (recorded by Biogen) were hurt by lower new patients starts in the United States, currency headwinds and unfavorable channel dynamics in the first quarter. (yahoo.com)
  • In a statement, Biogen Chief Executive Michel Vounatsos touted "continued strong worldwide growth for Spinraza. (wbur.org)
  • The existence of Biogen Inc. in the United States, high disposable income, and the increase in the uptake of Spinraza are a few more variables that helped to contribute to the greatest revenue share. (emailwire.com)
  • SPINRAZA is a first-line treatment in pre-symptomatic infants and children with genetically diagnosed 5q spinal muscular atrophy with 2 to 3 copies of the SMN2 gene. (has-sante.fr)
  • They conducted the trial to evaluate the safety and efficacy of Spinraza for the treatment of individuals with spinal muscular atrophy (or SMA). (marketrealist.com)
  • Spinraza is administered directly into the spinal canal - called an intrathecal injection - at a recommended dose of 12 mg for all individuals, regardless of SMA type or patient age or body weight. (smanewstoday.com)
  • SPINRAZA ® is injected into the spinal fluid in the lower back by doing a lumbar puncture. (ufl.edu)
  • The breadth of data presented reinforces the significant and clinically meaningful efficacy of SPINRAZA on the achievement of motor milestones and measures of motor function across a broad range of individuals with spinal muscular atrophy (SMA), as well as on survival endpoints in infantile-onset SMA. (biogen.com)
  • A great example is spinal muscular atrophy drug Spinraza. (foxbusiness.com)
  • The Cambridge pharmaceutical company said Thursday that revenue from Spinraza, which treats spinal muscular atrophy (SMA), totaled $543 million in the fourth quarter. (wbur.org)
  • Spinraza is indicated for treatment of spinal muscular atrophy in "pediatric and adult" patients, but study subjects were infants less than or equal to 7 months of age at the time of first dose. (pharmexec.com)
  • Spinraza, a medicine for spinal muscular atrophy (SMA), was once priced at a whopping 700,000 yuan. (news.cn)
  • Several Ionis antisense drugs have been commercially approved, including the U.S. Food and Drug Administration (FDA)-approved SPINRAZA, a therapy for spinal muscular atrophy. (scienceblog.com)
  • The patient populations for Biogen's Spinraza and Novartis' Zolgensma are overlapping but not identical, meaning the rivals don't compete for every prescription. (wbur.org)
  • Still, with a record list price of $2.1 million, Zolgensma can generate revenue in bigger chunks than Spinraza, which bears a sticker price of $750,000 in the first year of treatment and $375,000 annually thereafter. (wbur.org)
  • Two treatments, including the first-ever drug approved for the condition by the U.S. Food and Drug Administration (FDA) in 2016 called Spinraza, and Zolgensma, a gene therapy approved by the drug agency this month, are rapidly changing the trajectory for children with Arabella's condition. (neurosciencenews.com)
  • In their next appointment, Leigh presented both Spinraza and Zolgensma as options for Arabella. (neurosciencenews.com)
  • This makes Risdiplam the third SMA medicine available in Ireland, alongside Zolgensma and Spinraza. (mdi.ie)
  • SPINRAZA demonstrated a favorable safety profile in the study. (mda.org)
  • SPINRAZA demonstrated a favorable benefit-risk profile, with commonly reported adverse events consistent with those expected in the general SMA population or related to a lumbar puncture procedure. (biogen.com)
  • Spinraza was approved by the U.S. Food and Drug Administration (FDA) in December 2016 to treat children and adults with SMA, marking the first approval of a disease-modifying therapy for the condition. (smanewstoday.com)
  • since the nation's approval of Spinraza in December 2016. (emailwire.com)
  • SPINRAZA Intrathecal injection 12mg" is a prescription medicine (Rx) in Japan. (synapse.inc)
  • Certificate of Origin (CoO), Patient Information Leaflet (PIL) and Summary of Product Characteristics (SmPC) of "SPINRAZA Intrathecal injection 12mg" are available. (synapse.inc)
  • Search on-going Clinical Trials of "SPINRAZA Intrathecal injection 12mg" on International Clinical Trials Registry Platform (ICTRP) Search Portal provided by WHO. (synapse.inc)
  • Safety data involving the intrathecal administration of SPINRAZA showed the incidence and nature of the most common lumbar puncture-related adverse events were similar in children with later-onset SMA with or without scoliosis in the clinical studies. (biogen.com)
  • Spinraza is an antisense oligonucleotide, or ASO - a type of RNA-based molecule that's designed to increase SMN levels by binding to SMN2 's mRNA molecule and correcting its splicing. (smanewstoday.com)
  • SPINRAZA ® uses an antisense oligonucleotide, which is a synthetic material that replaces the missing protein in SMN 2 . (ufl.edu)
  • These data further validate the potential of SPINRAZA as a treatment for patients with SMA," said B. Lynne Parshall, chief operating officer of Ionis Pharmaceuticals. (mda.org)
  • Ionis would also reap rewards if Spinraza storms out of the gate. (foxbusiness.com)
  • National health plan reimbursement for Spinraza is available in nearly 30 European nations, and in countries like Australia, which also includes coverage for presymptomatic infants . (smanewstoday.com)
  • In the ENDEAR trial, Spinraza also achieved the pre-specified primary endpoint by demonstrating statistically significant 47% reduction in risk of death or permanent assisted ventilation. (marketrealist.com)
  • And despite the small decline in the fourth quarter, Spinraza revenue increased 22% in 2019, overall. (wbur.org)
  • In 4Q17, Biogen's (BIIB) Spinraza generated revenues of $363 million, which reflected 34% quarter-over-quarter growth. (marketrealist.com)
  • The analysis of the ENDEAR trial demonstrated that 51% of infants receiving Spinraza therapy had motor milestone responses, including full head control, ability to roll over, and free sitting and standing, compared to 0% for untreated infants. (marketrealist.com)
  • In an analysis of the Phase 3 ENDEAR end of study results, a greater proportion of infants with SMA on permanent ventilation treated with SPINRAZA demonstrated clinical benefits compared to untreated infants. (biogen.com)
  • In addition, further results from the interim analysis of the Phase 2 NURTURE study highlight the clinically meaningful efficacy of SPINRAZA on event-free survival, measures of motor function and achievement of motor milestones when administered to infants with genetically-diagnosed SMA before symptom onset. (biogen.com)
  • The additional NURTURE data extends this finding by showing substantial improvements in motor milestones, generally consistent with normal development among infants with SMA who have yet to manifest symptoms before they were treated with SPINRAZA. (biogen.com)
  • End of study data from both the Phase 3 ENDEAR and CHERISH studies further demonstrate that earlier SPINRAZA treatment in individuals with SMA may lead to improved outcomes. (biogen.com)
  • As part of our mission to make a meaningful difference in the lives of those affected by SMA, we continue to collect and evaluate data to provide a deeper understanding of the impact of SPINRAZA across SMA populations and share those results with the SMA community. (biogen.com)
  • New SPINRAZA data continue to reinforce the positive results seen in clinical studies and in my own practice," said Thomas Crawford, M.D., co-director, Muscular Dystrophy Association Clinic at Johns Hopkins Medicine. (biogen.com)
  • The analysis found that children receiving SPINRAZA experienced a highly statistically significant improvement in motor function compared to those who did not receive treatment. (mda.org)
  • From baseline to 15 months of treatment, patients who received SPINRAZA achieved a mean improvement of 4.0 points in the HFMSE, while patients who were not on treatment declined by a mean of 1.9 points. (mda.org)
  • The U.S. Food and Drug Administration (FDA) recently accepted the company's New Drug Application (NDA) for SPINRAZA as a treatment for SMA and communicated they plan to act early on the NDA under an expedited review. (mda.org)
  • SMA360° insurance and financial assistance programs are designed to help you understand your insurance benefits for SPINRAZA ® and find an affordable way to start treatment and continue as prescribed by your child's physician. (ufl.edu)
  • Data presented at the Cure SMA 2017 Annual SMA Conference further demonstrate the significant impact of SPINRAZA and the benefits of early treatment initiation. (biogen.com)
  • The SPINRAZA clinical development program demonstrates the impact of early treatment. (biogen.com)
  • SPINRAZA ® is the first approved therapy shown in clinical trials to change the expected course of SMA for some individuals, increasing survival, motor milestone achievement, and motor function. (ufl.edu)
  • In 3Q17 and 4Q16, Spinraza generated revenues of $271 million and $5 million, respectively. (marketrealist.com)
  • In 4Q17, in the US market and outside the US market (international markets), Spinraza generated revenues of $218 million and $144 million, respectively, compared to $198 million and $73 million, respectively, in 3Q17, which reflected 10% and 97% growth, respectively, on a quarter-over-quarter basis. (marketrealist.com)
  • Commercial revenues from Spinraza royalties were $50 million, down 7.4% year over. (yahoo.com)
  • CHERISH is a fifteen-month study investigating SPINRAZA in 126 non-ambulatory patients with later-onset SMA (consistent with Type 2), including patients with the onset of signs and symptoms at greater than 6 months and an age of 2 to 12 years at screening. (mda.org)
  • With the positive interim analysis, the CHERISH study will be stopped and participants will be able to transition into the SHINE open-label extension study to receive SPINRAZA. (mda.org)
  • How is SPINRAZA ® given to patients? (ufl.edu)
  • We will make regulators around the globe aware of this data and will continue working closely with them to bring SPINRAZA to families affected by SMA as quickly as possible. (mda.org)
  • Children with the most extreme form of SMA - Type 1 - rarely live beyond the age of two, and calls are mounting for the drug, called Spinraza, to be approved before it is too late for them. (mirror.co.uk)
  • Spinraza brought in almost $1B in sales in its first year alone. (genengnews.com)
  • The revenue growth of Spinraza could boost the share price of the iShares Nasdaq Biotechnology (IBB) . (marketrealist.com)
  • Recently, a tear-jerking video showing how a national health insurance negotiator helped cut down the bidding price of Spinraza from over 53,680 yuan (8,422 U.S. dollars) per dose to 33,000 yuan went viral online and was lauded by netizens. (news.cn)
  • Since then, I helped bring Jake's fight for access to Spinraza to Parliament for Rare Diseases Day 2020, and his case has seen coverage from local and national news outlets, in order to raise much-needed awareness. (kerrymccarthy.org)
  • Analysts project that Spinraza could reach peak sales between $1 billion and $2 billion. (foxbusiness.com)
  • The Green Party welcomes the news that Pharmac will fully fund Spinraza for people under the age of 18. (greens.org.nz)
  • We are grateful to all the families and clinicians who have participated in all of the SPINRAZA studies. (mda.org)
  • We found out about Spinraza in 2015, and it felt like a Christmas present for us all. (mirror.co.uk)