Biogen’s SPINRAZA® (nusinersen) Data Show Earlier Treatment Initiation May Lead to Improved Motor Function Across a Broad Population of People Living with Spinal Muscular Atrophy
Biogen (NASDAQ: BIIB) presented new data demonstrating that earlier initiation of treatment with SPINRAZA® (nusinersen) may improve motor function outcomes in infants and children with spinal muscular atrophy (SMA). Results continued to reinforce the favorable efficacy and safety profile of SPINRAZA. The data were shared at the 22nd International Annual Congress of the World Muscle Society in Saint Malo, France (October 3-7, 2017).
A new analysis from the Phase 3 ENDEAR study showed infants with SMA who initiated treatment earlier in the disease (shorter disease duration) demonstrated greater benefit and improvement in motor function outcomes.
As measured by the Hammersmith Infant Neurological Examination (HINE), significant differences in motor milestone responders were observed between infants treated with SPINRAZA compared to untreated infants with disease duration less than or equal to 12 weeks (75% vs. 0%; P<.0001) and those with disease duration greater than 12 weeks (32% vs. 0%; P=.0026). There was also a significant benefit in event-free survival in infants treated with SPINRAZA with disease duration less than or equal to 12 weeks (P=.0004).
“These studies contribute to a growing body of evidence that SPINRAZA can make a meaningful difference in the lives of people with SMA regardless of their age or stage of the disease,” said Alfred Sandrock, M.D., Ph.D., executive vice president and chief medical officer at Biogen. “Across studies, we continue to see evidence that earlier initiation of treatment with SPINRAZA can lead to improved clinical and functional outcomes.”
Interim analyses were also presented from the Phase 2 EMBRACE study which was designed to assess the efficacy and safety of SPINRAZA in individuals with infantile- and later-onset SMA who were ineligible for the two earlier pivotal studies.
The EMBRACE interim analysis showed a larger proportion of infants and children treated with SPINRAZA were HINE motor milestone responders compared to those who were untreated. Results from the interim analysis also supported the dosing regimen of four loading doses in the first two months, followed by the administration of SPINRAZA every four months thereafter, for individuals with infantile- and later-onset SMA.
In the ENDEAR and EMBRACE studies SPINRAZA demonstrated a favorable benefit-risk profile. Safety data involving the intrathecal administration of SPINRAZA showed the incidence and nature of the most common lumbar puncture-related adverse events in the clinical studies were similar in children with later-onset SMA with or without scoliosis.
For more information about SPINRAZA and prescribing information in the United States, please visit www.SPINRAZA.com. Prescribing information in the European Union is available at http://www.ema.europa.eu/ema/.
About ENDEAR and EMBRACE
ENDEAR is a randomized, double-blind, sham-procedure controlled 13-month study in patients with infantile-onset SMA. The end of study efficacy analysis included all patients (n=121) who had their final study visit after the interim analysis (n=78) and had the opportunity to attend the six-month study visit assessment. The Hammersmith Infant Neurological Examination (HINE) is a reliable and clinically validated tool to assess motor milestone achievement in infants with SMA.
EMBRACE is a Phase 2, multicenter, randomized, double-blind, sham-procedure controlled 14-month study of SPINRAZA in infants and children not eligible to participate in ENDEAR (symptom onset less than or equal to six months, less than or equal to seven months of age at screening; 2 SMN2 copies) or CHERISH (symptom onset age greater than six months, age 2-12 years at screening).
SPINRAZA Program Status
SPINRAZA is the first approved medicine for the treatment of SMA and is currently approved in the United States, the European Union, Brazil, Japan and Canada. Biogen has submitted regulatory filings in additional countries and plans to initiate additional filings in other countries.
Globally, in 2016, in response to the urgent need for treatment for the most severely affected individuals living with SMA, Biogen sponsored one of the largest, pre-approval Expanded Access Programs (EAP) in rare disease, free of charge.
Biogen licensed the global rights to develop, manufacture and commercialize SPINRAZA from Ionis Pharmaceuticals (NASDAQ: IONS), a leader in antisense therapeutics. Biogen and Ionis conducted an innovative clinical development program that moved SPINRAZA from its first dose in humans in 2011 to its first regulatory approval in five years.
Spinal muscular atrophy (SMA) is characterized by loss of motor neurons in the spinal cord and lower brain stem, resulting in severe and progressive muscular atrophy and weakness. Ultimately, individuals with the most severe type of SMA can become paralyzed and have difficulty performing the basic functions of life, like breathing and swallowing.
Due to a loss of, or defect in, the SMN1 gene, people with SMA do not produce enough SMN protein, which is critical for the maintenance of motor neurons. The severity of SMA correlates with the amount of SMN protein. People with Type 1 SMA, the form that requires the most intensive and supportive care, produce very little SMN protein and do not achieve the ability to sit without support or live beyond two years without respiratory support. People with Type 2 and Type 3 SMA produce greater amounts of SMN protein and have less severe, but still life-altering forms of SMA.
SPINRAZA is being developed globally for the treatment of SMA.
SPINRAZA is an antisense oligonucleotide (ASO), using Ionis Pharmaceuticals’ proprietary antisense technology, that is designed to treat SMA caused by mutations or deletions in the SMN1 gene located in chromosome 5q that leads to SMN protein deficiency. SPINRAZA alters the splicing of SMN2 pre-mRNA in order to increase production of full-length SMN protein.6 ASOs are short synthetic strings of nucleotides designed to selectively bind to target RNA and regulate gene expression. Through use of this technology, SPINRAZA has the potential to increase the amount of full-length SMN protein in individuals with SMA.
SPINRAZA must be administered via intrathecal injection, which delivers therapies directly to the cerebrospinal fluid (CSF) around the spinal cord,7 where motor neurons degenerate in individuals with SMA due to insufficient levels of survival motor neuron (SMN) protein.8
SPINRAZA demonstrated a favorable benefit-risk profile. The most common adverse reactions reported for SPINRAZA were upper respiratory infection, lower respiratory infection and constipation. Serious adverse reactions of atelectasis were more frequent in SPINRAZA-treated patients. Coagulation abnormalities and thrombocytopenia, including acute severe thrombocytopenia, have been observed after administration of some antisense oligonucleotides. Individuals may be at increased risk of bleeding complications. Renal toxicity has been observed after administration of some antisense oligonucleotides. SPINRAZA is present in and excreted by the kidney.
Through cutting-edge science and medicine, Biogen discovers, develops and delivers innovative therapies worldwide for people living with serious neurological and neurodegenerative diseases. Founded in 1978, Biogen is a pioneer in biotechnology and today the Company has the leading portfolio of medicines to treat multiple sclerosis, has introduced the first and only approved treatment for spinal muscular atrophy and is at the forefront of neurology research for conditions including Alzheimer’s disease, Parkinson’s disease and amyotrophic lateral sclerosis. Biogen also manufactures and commercializes biosimilars of advanced biologics. For more information, please visit www.biogen.com. Follow us on social media – Twitter, LinkedIn, Facebook, YouTube.
Biogen Safe Harbor
This press release contains forward-looking statements, including statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 relating to the potential benefits, safety and efficacy of SPINRAZA, the results of certain real-world data, the status of current regulatory filings, plans for additional regulatory filings in other jurisdictions, planning and timing for commercial launch, and availability of patient access and reimbursement pathways, which may vary on a country-by-country basis. These forward-looking statements may be accompanied by words such as “aim,” “anticipate,” “believe,” “could,” “estimate,” “except,” “forecast,” “intend,” “may,” “plan,” “potential,” “possible,” “will” and other words and terms of similar meaning. You should not place undue reliance on these statements or the scientific data presented. Drug development and commercialization involve a high degree of risk. These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements, including without limitation uncertainty of success in commercialization of SPINRAZA, which may be impacted by, among other things, the level of preparedness of healthcare providers to treat patients, difficulties in obtaining or changes in the availability of reimbursement for SPINRAZA, the effectiveness of sales and marketing efforts, problems with the manufacturing process for SPINRAZA, the occurrence of adverse safety events, unexpected concerns that may arise from additional data or analysis; regulatory authorities may require additional information or further studies, or may fail to approve or may delay approval of Biogen’s drug candidates or expansion of product labeling; or Biogen may encounter other unexpected hurdles which may be impacted by, among other things, the occurrence of adverse safety events, failure to obtain regulatory approvals in certain jurisdictions, failure to obtain regulatory approvals in other jurisdictions, failure to protect intellectual property and other proprietary rights; product liability claims; or third party collaboration risks. The foregoing sets forth many, but not all, of the factors that could cause actual results to differ from our expectations in any forward-looking statement. Investors should consider this cautionary statement, as well as the risk factors identified in Biogen’s most recent annual or quarterly report and in other reports Biogen has filed with the U.S. Securities and Exchange Commission. These statements are based on our current beliefs and expectations and speak only as of the date of this press release. We do not undertake any obligation to publicly update any forward-looking statements, whether as a result of new information, future developments or otherwise.
1. Darras B, Markowitz J, Monani U, De Vivo D. Chapter 8 - Spinal Muscular Atrophies. In: Vivo BTD, ed. Neuromuscular Disorders of Infancy, Childhood, and Adolescence (Second Edition). San Diego: Academic Press; 2015:117-145.
2. Lefebvre S, Burglen L, Reboullet S, et al. Identification and characterization of a spinal muscular atrophy-determining gene. Cell.1995;80(1):155-165.
3. Mailman MD, Heinz JW, Papp AC, et al. Molecular analysis of spinal muscular atrophy and modification of the phenotype by SMN2. Genet Med. 2002;4(1):20-26.
4. Monani UR, Lorson CL, Parsons DW, et al. A single nucleotide difference that alters splicing patterns distinguishes the SMA gene SMN1 from the copy gene SMN2. Hum Mol Genet. 1999;8(7):1177-1183.
5. Peeters K, Chamova T, Jordanova A. Clinical and genetic diversity of SMN1-negative proximal spinal muscular atrophies. Brain.2014;137(Pt 11):2879-2896.
6. Hua Y, Sahashi K, Hung G, Rigo F, Passini MA, Bennett CF, Krainer AR. Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model. Genes Dev. 2010 Aug 1; 24(15):16344-44.
7. Evers MM, Toonen LJ, van Roon-Mom WM. Antisense oligonucleotides in therapy for neurodegenerative disorders. Adv Drug Deliv Rev. 2015;87:90-103.
8. Lunn MR, Wang CH. Spinal muscular atrophy. Lancet. 2008;371(9630):2120-2133.
For more than 50 years, Business Wire has been the global leader in press release distribution and regulatory disclosure.
Tilaa tiedotteet sähköpostiisi
Haluatko tietää asioista ensimmäisten joukossa? Kun tilaat mediatiedotteemme, saat ne sähköpostiisi välittömästi julkaisuhetkellä. Tilauksen voit halutessasi perua milloin tahansa.
Lue lisää julkaisijalta Business Wire
CHMP Grants Positive Opinion for New Indication of Clovis Oncology’s Rubraca®▼ (rucaparib) Tablets as Maintenance Treatment for Women with Relapsed Ovarian Cancer13.12.2018 20:46 | Tiedote
Clovis Oncology, Inc. (NASDAQ: CLVS) today announced that the European Union’s (EU) European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending an additional indication to include rucaparib as monotherapy for the maintenance treatment of adult patients with platinum-sensitive relapsed high-grade epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in response (complete or partial) to platinum-based chemotherapy. EC approval is anticipated in the first quarter of 2019. Once approved, Rubraca’s indication will expand beyond its initial Marketing Authorization in Europe granted in May 2018 for adult patients with platinum-sensitive, relapsed or progressive, BRCA mutated (germline and/or somatic), high-grade epithelial ovarian, fallopian tube, or primary peritoneal cancer, who have been treated with two or more prior lines of platinum-based chemotherapy, and who are unable to tolerate further platinum
Dole Food Company Sells Fresh Cut Business in Sweden and Finland13.12.2018 20:00 | Tiedote
Dole Food Company, Inc., announced today an agreement to sell Saba Fresh Cuts AB and Saba Fresh Cuts OY to BAMA International. Saba Fresh Cuts AB—with production facility in Helsingborg Sweden—and Saba Fresh Cuts OY—with production facility in Espoo Finland—are producers of washed and ready-to-eat salads. The companies prepare and sell various fresh-cut salads and vegetables to the retail and foodservice industry in Sweden, Denmark, Finland, Germany and the Baltic States. The sale of Saba Fresh Cuts AB was a necessary condition of the European Commission’s approval of the investment by Total Produce into Dole in July 2018. “I am very pleased with the outcome of this sale in meeting the EU’s requirement,” said Mr. Johan Linden, President and Chief Executive Officer of Dole Food Company, “and I am confident that Saba Fresh Cuts will be in good hands under BAMA’s ownership.” "We are pleased to announce this agreement with Dole for Saba Fresh Cuts. Convenience products and ready-to-eat sal
Thales and Gemalto Are Granted Regulatory Clearance by the Commerce Commission in New Zealand13.12.2018 19:00 | Tiedote
Regulatory News: Reference is made to the joint press release by Thales (Euronext Paris: HO) and Gemalto (Euronext Amsterdam and Paris: GTO) dated 27 March 2018 in relation to the launch of the recommended all-cash offer by Thales for all the issued and outstanding shares of Gemalto (the “Offer”), the publication of the Offer Document, and the joint press release of Thales and Gemalto dated 10 August 2018 in relation to the further extension of the Acceptance Period. Terms not defined in this press release will have the meaning as set forth in the Offer Document. Thales and Gemalto announce today that they have received merger control Regulatory Clearance from the Commerce Commission in New Zealand, following Thales’s commitment to divest its general purpose hardware security modules (GP HSM) business globally1 to a suitable purchaser. This clearance is effective immediately. Together with the antitrust clearances obtained in China, Israel, South Africa, Turkey, and for the European Un
Hurricane Electric Becomes First Internet Backbone to Connect to 200 Internet Exchanges around the Globe13.12.2018 18:37 | Tiedote
Hurricane Electric, the world’s largest IPv6-native Internet backbone, today announced that it is the first Internet backbone in the world to connect to over 200 unique Internet exchanges. As an early leader in the switch to IPv6, Hurricane Electric now connects to the largest number of networks on the planet for both IPv6 and IPv4 and has been at the forefront of providing high-speed Internet connectivity to organizations throughout the world using:100GE (100 Gigabit Ethernet), 10GE (10 Gigabit Ethernet), and GigE (1 Gigabit Ethernet) connections. As a result of the improved connectivity with these 200 exchanges, customers receive the benefit of lower latency, higher throughput and increased fault tolerance. This reliable connectivity is especially important as global IP traffic continues to increase – Cisco’s Visual Networking Index: Forecast and Trends, 2017–2022 predicts that annual global IP traffic will reach 4.8 zettabytes (ZB) per year by 2022, or 396 exabytes (EB) per month, t
The World's Largest Heritage and Culture Festival to Begin With the Slogan Janadria 33 Faithfulness and Loyalty13.12.2018 18:15 | Tiedote
With the support and under the patronage of the Custodian of the Two Holy Mosques King Salman bin Abdulaziz, the 33rd Edition of the National Festival of Heritage and Culture will be held in Janadria for 21 days from Thursday 20th December 2018 to Wednesday 9th January 2019. Organized by the Saudi Ministry of National Guard, the festival is part of the efforts to intensify attention to Saudi heritage, expose it regionally and globally and to promote dialogue among cultures and civilizations on the level of people and nations. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20181213005472/en/ The Custodian of the Two Holy Mosques King Salman bin Abdulaziz (Photo: AETOSWire) Janadria is the largest Saudi cultural festival that simulates the history, present and future of Saudi Arabia and the largest event of its kind in the region. Since 1985, it has witnessed extensive Saudi participation, a growing Arab and Gulf presence and an
PPG to Invest in Automated Dispense Cell Manufacturing for Automotive Parts and Accessories13.12.2018 17:32 | Tiedote
PPG (NYSE: PPG) today announced that it is increasing its investment in automated dispense cell manufacturing of paint and related coatings for the automotive parts and accessories decorative (APAD) space. Financial details are not being disclosed. Dispense cell technology enables PPG to dramatically accelerate production and approval of paint batches for vehicle original equipment manufacturers (OEMs) and component suppliers. The technology allows coatings components to be automatically drawn from storage vats in the correct proportions and mixed directly in their shipping containers, vastly simplifying and increasing the efficiency of the coatings manufacturing process. PPG recently launched an APAD dispense cell operation at its San Juan del Rio, Mexico, coatings facility and will begin production on an additional cell in 2019 in Sriperumbudur, India. These cells are in addition to PPG’s recent APAD investments in Germany and Russia and the dispense cells already online at the Compa
Uutishuoneessa voit lukea tiedotteitamme ja muuta julkaisemaamme materiaalia. Löydät sieltä niin yhteyshenkilöidemme tiedot kuin vapaasti julkaistavissa olevia kuvia ja videoita. Uutishuoneessa voit nähdä myös sosiaalisen median sisältöjä. Kaikki STT Infossa julkaistu materiaali on vapaasti median käytettävissä.Tutustu uutishuoneeseemme