Biogen Completes Rolling Submission of New Drug Application to FDA for Nusinersen as a Treatment for Spinal Muscular Atrophy
Biogen (NASDAQ: BIIB) and Ionis (NASDAQ:IONS) today announced that Biogen has completed the rolling submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for the approval of nusinersen, an investigational treatment for spinal muscular atrophy (SMA). Biogen has also applied for Priority Review which, if granted, would shorten the review period of nusinersen following the Agency’s acceptance of the NDA.
This Smart News Release features multimedia. View the full release here: http://www.businesswire.com/news/home/20160926005499/en/
“Since announcing the positive results of the ENDEAR interim analysis in infantile-onset SMA last month, we have heard from many families expressing their excitement about nusinersen. Their stories continue to inspire us and they are in the forefront of our minds as we work to support the FDA’s review of nusinersen,” noted Alfred Sandrock, M.D., Ph.D., executive vice president and chief medical officer at Biogen. “We appreciate the FDA’s collaboration with us during the application process, and we look forward to continuing this productive dialogue, with the goal of rapidly bringing the first treatment for SMA to as many patients as possible.”
In addition to the NDA filing with FDA, Biogen plans to submit a Marketing Authorization Application (MAA) for nusinersen to the European Medicines Agency (EMA) in the coming weeks. The EMA’s Committee for Medicinal Products for Human Use (CHMP) recently granted Accelerated Assessment to nusinersen, which can reduce the standard review time. Biogen will initiate regulatory filings in other countries in the coming months.
“Our ability to advance the nusinersen program as quickly as we have is largely due to the tremendous contributions of the entire SMA community, from the patients and families who participated in the clinical trials to the doctors, nurses and advocates who work tirelessly on their behalf,” said B. Lynne Parshall, chief operating officer at Ionis Pharmaceuticals. “We are deeply appreciative of their unwavering commitment to finding a treatment for SMA and today’s milestone is truly a collective achievement.”
The regulatory submissions are comprised of results from the pre-specified interim analysis of ENDEAR, the controlled Phase 3 study evaluating nusinersen in infantile-onset (most likely to develop Type 1) SMA, as well as all other clinical and preclinical data currently available, which includes open-label data in other patients types. The ENDEAR interim analysis demonstrated that infants receiving nusinersen experienced a statistically significant improvement in the achievement of motor milestones compared to those who did not receive treatment. Biogen anticipates hearing from regulatory authorities regarding the acceptance and validation of these submissions within the next couple of months.
The Nusinersen Clinical Trial Program
The nusinersen clinical trial program is comprised of two controlled studies, ENDEAR and CHERISH. ENDEAR was designed as a thirteen-month study investigating nusinersen in 122 patients with infantile-onset SMA; the onset of signs and symptoms of SMA less than or equal to 6 months of age and age less than or equal to 7 months at screening. Based on the results of the pre-specified interim analysis, the ENDEAR study will be stopped; patients who elect to are currently being transitioned to the SHINE open-label study where they will all receive nusinersen. Results from the ENDEAR interim analysis will be presented at future medical congresses.
CHERISH is a fifteen-month study investigating nusinersen in 126 non-ambulatory patients with later-onset SMA, consistent with Type 2; onset of signs and symptoms greater than 6 months and age 2 to 12 years at screening. CHERISH was fully enrolled in May 2016 and remains ongoing.
Additionally, the SHINE open-label extension study, for patients who previously participated in ENDEAR and CHERISH, is open and is intended to evaluate the long-term safety and tolerability of nusinersen.
Two additional Phase 2 studies, EMBRACE and NURTURE, were designed to collect additional data on nusinersen. The EMBRACE study is designed to collect additional data on a small subset of patients with infantile or later-onset SMA who do not meet the age and other criteria of ENDEAR or CHERISH. Due to the evidence demonstrated in the infantile-onset SMA (most likely to develop Type 1) population, the sham arm of the EMBRACE study is being stopped and patients are being given the option to receive nusinersen through an open-label extension study. NURTURE is an open-label, ongoing study in pre-symptomatic infants who are less than or equal to 6 weeks of age at time of first dose to determine if treatment before symptoms begin would prevent or delay onset of SMA symptoms.
About SMA 1-5
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 survival motor neuron (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 most severe life-threatening form, produce very little SMN protein and do not achieve the ability to sit without support or live beyond 2 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.
Currently, there is no approved treatment for SMA.
To support awareness and education in SMA, Biogen is launching Together in SMA in the United States, a program created to provide informational materials and resources to the SMA community. Learn more at TogetherinSMA.com.
Nusinersen is an investigational, potentially disease-modifying therapy6 for the treatment of SMA. Nusinersen is an antisense oligonucleotide (ASO) that is designed to alter the splicing of pre-mRNA from the SMN2 gene in order to increase production of fully functional SMN protein. SMN2 is a gene that is nearly identical to SMN1.7
ASOs are short synthetic strings of nucleotides designed to selectively bind to target RNA and regulate gene expression. Through use of this technology, nusinersen has the potential to increase the amount of functional SMN protein in infants and children with SMA.
Both the U.S. and EU regulatory agencies have granted special status to nusinersen in an effort to expedite the review process, including Fast Track Designation in the U.S. Additionally, nusinersen has received an Orphan Drug designation in both U.S. and EU.
We acknowledge support from the following organizations for nusinersen: Muscular Dystrophy Association, SMA Foundation, Cure SMA and intellectual property licensed from Cold Spring Harbor Laboratory and the University of Massachusetts Medical School.
Through cutting-edge science and medicine, Biogen discovers, develops and delivers worldwide innovative therapies for people living with serious neurological, autoimmune and rare diseases. Founded in 1978, Biogen is one of the world’s oldest independent biotechnology companies and patients worldwide benefit from its leading multiple sclerosis and innovative hemophilia therapies. For more information, please visit www.biogen.com. Follow us on Twitter.
About Ionis Pharmaceuticals Inc.
Ionis is the leading company in RNA-targeted drug discovery and development focused on developing drugs for patients who have the highest unmet medical needs, such as those patients with severe and rare diseases. Using its proprietary antisense technology, Ionis has created a large pipeline of first-in-class or best-in-class drugs, with over a dozen drugs in mid- to late-stage development. Drugs currently in Phase 3 development include volanesorsen, a drug Ionis is developing and plans to commercialize through its wholly owned subsidiary, Akcea Therapeutics, to treat patients with either familial chylomicronemia syndrome or familial partial lipodystrophy; IONIS-TTRRx, a drug Ionis is developing with GSK to treat patients with all forms of TTR amyloidosis; and nusinersen, a drug Ionis is developing with Biogen to treat infants and children with spinal muscular atrophy. Ionis' patents provide strong and extensive protection for its drugs and technology. Additional information about Ionis is available at www.ionispharma.com.
Biogen Safe Harbor
This press release contains forward-looking statements, including statements relating to the submission of marketing authorization applications for nusinersen to regulatory authorities and the timing thereof and the anticipated regulatory filing review process. These statements may be identified by words such as “believe,” “except,” “may,” “plan,” “potential,” “will” and similar expressions, and are based on our current beliefs and expectations. Drug development and commercialization involve a high degree of risk, and only a small number of research and development programs result in commercialization of a product. Factors which could cause actual results to differ materially from our current expectations include the risk that unexpected concerns 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 our drug candidates or grant marketing approval that is more restricted than anticipated, or we may encounter other unexpected hurdles. For more detailed information on the risks and uncertainties associated with our drug development and commercialization activities, please review the Risk Factors section of our most recent annual report or quarterly report filed with the Securities and Exchange Commission. Any forward-looking statements speak only as of the date of this press release and we assume no obligation to update any forward-looking statement.
Ionis Forward-Looking Statement
This press release includes forward-looking statements regarding Ionis' strategic relationship with Biogen and the development, activity, therapeutic potential, safety and commercialization of nusinersen. Any statement describing Ionis' goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties, particularly those inherent in the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such drugs. Ionis' forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause its results to differ materially from those expressed or implied by such forward-looking statements. Although Ionis' forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Ionis. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Ionis' programs are described in additional detail in Ionis' annual report on Form 10-K for the year ended December 31, 2015, and its most recent quarterly report on Form 10-Q, which are on file with the SEC. Copies of these and other documents are available from the Company.
BIOGEN and the BIOGEN logo are registered trademarks of BIOGEN.
Ionis Pharmaceuticals™ is a trademark of Ionis Pharmaceuticals, Inc. Akcea Therapeutics™ is a trademark of Ionis Pharmaceuticals, Inc.
|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.||Rigo F, Hua Y, Krainer AR, Bennett CF. Antisense-based therapy for the treatment of spinal muscular atrophy. J Cell Biol. 2012;199(1):21-25|
|7.||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|
Ligia Del Bianco, +1 781-464-3260
Wade Walke, +1 760-603-2741
Ben Strain, +781-464-2442
Wade Walke, +1 760-603-2741
Om Business Wire
Business Wire, a Berkshire Hathaway company, is the global leader in multiplatform press release distribution.
Følg saker fra Business Wire
Registrer deg med din epostadresse under for å få de nyeste sakene fra Business Wire på epost fortløpende. Du kan melde deg av når som helst.
Siste saker fra Business Wire
AstraZeneca and Chi-Med’s Savolitinib Shows Encouraging Clinical Activity in 2nd-Line EGFR Mutation-positive Lung Cancer with MET-amplification16.10.2017 22:00 | Pressemelding
AstraZeneca and its partner Chi-Med today presented preliminary safety and clinical activity of savolitinib when given in combination with either Tagrisso (osimertinib) or Iressa (gefitinib) in two Phase Ib trials conducted in patients with epidermal growth factor receptor (EGFR) mutation-positive non-small cell lung cancer (NSCLC) with MET-amplification who had progressed following 1st-line treatment with an EGFR inhibitor.1,2 In both trials, the addition of savolitinib (600mg, once daily), an investigational selective inhibitor of c-MET (mesenchymal epithelial transition factor) receptor tyrosine kinase, to osimertinib (80mg, once daily) or gefitinib (250mg, once daily) demonstrated preliminary anti-tumour activity. The data were shared in two oral presentations at the International Association for the Study of Lung Cancer 18th World Conference on Lung Cancer (WCLC) in Yokohama, Japan, 15-18 Oc
Orlando Melbourne Int’l (MLB) Lands Largest FAA Grant in Its History16.10.2017 21:34 | Pressemelding
When Air Force One landed at Orlando Melbourne International Airport (MLB) in February, Airport Executive Director Greg Donovan took advantage of the opportunity and advised the president of the United States that his 747-200 just landed on a commercial runway officially rated ‘poor’ in aviation terms. That’s going to change, thanks to an $18,132,588 FAA grant announced by U.S. Secretary of Transportation Elaine Chao today. This press release features multimedia. View the full release here: http://www.businesswire.com/news/home/20171016006292/en/ Greg Donovan, A.A.E. (Photo: Business Wire) “We asked for, and received, tremendous support from the Florida congressional delegation,” said Jack L. Ryals, chairman of the Melbourne Airport Authority. “We are deeply grateful to Congressman Posey, Senator Nelson and Senator Rubio for their advocacy on this critic
Colombia Selects IDEMIA to Upgrade Its Identification and Civil Registry Platform16.10.2017 14:34 | Pressemelding
IDEMIA, the global leader in trusted identities for an increasingly digital world, has been selected by the National Civil Registry of Colombia (Registraduría Nacional del Estado Civil de Colombia – RNEC 1 ) to update the identification and civil registry platform. This platform is the trusted repository of citizens’ biometric and alphanumeric data and ensures the uniqueness of identities. IDEMIA (formerly known as OT-Morpho) has worked with the RNEC since the beginning of this project and has constantly supported the successive improvements of the solution. In 2017, when in need to strengthen the platform in order to offer new services to the government and to the population while maintaining a high level of security and satisfaction of the users, RNEC decided to pursue its partnership with IDEMIA. Through this new contract, IDEMIA will e
ATB Financial Goes-Live on Murex’s SaaS Solution16.10.2017 14:00 | Pressemelding
Sibos - Murex, a global leader in trading, risk management and processing solutions, is pleased to announce the successful launch of the MX.3 SaaS solution at ATB Financial. MUREX SaaS is a managed services offering for the MX.3 platform, encompassing infrastructure and application management. This press release features multimedia. View the full release here: http://www.businesswire.com/news/home/20171016005092/en/ ATB pursued this initiative in order to create a simple yet comprehensive foreign exchange experience for their customers. In addition to the customer benefit, ATB was also able to rationalize their IT landscape, broaden the scope of financial instruments they provide on their digital platform, and prepare for a large volume increase in their trading activities. Due to the strength of the solution’s risk management capabilities, extensive asset class coverage, and the ra
Leave a review on print24.com and win a VW up!16.10.2017 13:59 | Pressemelding
The online print shop UNITEDPRINT SE, owner of renowned brands including print24.com, is asking its customers for their opinions on the company’s products and services. In return for their views, customers will receive vouchers and be entered into a prize draw. The online print shop is offering one voucher for every review submitted across various portals. The vouchers – worth up to €25.00 and valid until 31 March 2018 – will be subject to a minimum order amount (product price inc. VAT, not including delivery) of €250.00 per voucher. All reviews received up to 31 December 2017 will be eligible, regardless of length and content. You can learn more about the review campaign at https://print24.com/rate/ But not only that, print24.com is also giving every reviewer the chance to win a brand-new VW up! Ali Jason Bazooband, Managing Director for Innovation/Marketing at print24.com,
Bentley Systems Announces Winners of 2017 Be Inspired Awards16.10.2017 13:52 | Pressemelding
The Year in Infrastructure 2017 Conference – Bentley Systems, Incorporated, a leading global provider of comprehensive software solutions for advancing infrastructure, has announced the winners of the 2017 Be Inspired Awards. The annual awards program honors the extraordinary work of Bentley users advancing the world’s infrastructure. At a ceremony and gala at their Year in Infrastructure 2017 Conference, Bentley acknowledged 17 Be Inspired Awards winners and six Special Recognition Awards winners. The Year in Infrastructure Conference is Bentley’s annual global gathering of leading professionals in the world of infrastructure design, construction, and operations. In addition to thought-provoking keynotes, technology demonstrations, industry forums, and panel discussions, the agenda included presentations by finalists in the Be Inspired Awards program, culminating in the selection of t
I vårt presserom finner du alle våre siste saker, kontaktpersoner, bilder, dokumenter og annen relevant informasjon om oss.Besøk vårt presserom