CHMP recommends EU label expansion of Novartis' Gilenya and data at AAN confirm efficacy on pre-treated MS patients
CHMP has recognized Gilenya's favorable benefit/risk profile and recommends EU label expansion to patients not responding to DMTs beyond interferon
AAN data: Gilenya reduces relapse rates, new MRI lesion counts, brain volume loss & disability progression in pre-treated MS patients with high disease activity
Gilenya is the only oral DMT that is effective across four key measures of MS (relapse rates, MRI lesions, brain volume loss and disability progression)
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Basel, April 29, 2014- Novartis announced today that the Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion to expand the EU label for Gilenya® (fingolimod) in relapsing remitting multiple sclerosis (RRMS). The recommendation is to expand the label to include adult patients who have not responded to at least one disease-modifying therapy (DMT), including newly-approved oral DMTs. Gilenya is currently licensed in the EU for adult patients with RRMS who have not responded to treatment with interferons, or have rapidly evolving severe MS.
Novartis also announced new pooled analyses presented at the 66th American Academy of Neurology (AAN) Annual Meeting in Philadelphia, Pennsylvania from the pivotal FREEDOMS and FREEDOMS II trials in multiple sclerosis (MS), confirming the consistent efficacy of Gilenya across four key measures of MS (relapse rates, MRI lesions, brain volume loss and disability progression). Addressing these four measures through effective treatment and disease management is important for improving the course of MS for patients
"We are very pleased that the CHMP has recognized the favorable benefit-risk profile of Gilenya and made a recommendation to broaden its label to allow patients who have failed on other disease-modifying therapies to be switched to Gilenya treatment," said Tim Wright, Global Head of Development, Novartis Pharmaceuticals. "Additionally, the new analyses at AAN confirm Gilenya's robust efficacy across four key measures of MS disease activity, which is important to give patients as much time free of functional impairment."
The pooled analyses from the FREEDOMS and FREEDOMS II trials show that in patients with high disease activity previously treated in the past year, Gilenya demonstrated significant efficacy across the following measures:
Relapses - Gilenya reduced relapses (as measured by the annualized relapse rate) by almost half (48%) compared to placebo
MRI lesions - new T2 lesion formation was reduced by 69% compared to placebo
Brain volume loss - Gilenya reduced the rate of brain volume loss by 46% compared to placebo
Disability progression - using a stringent six-month disability measure, Gilenya reduced disability progression by 45% compared to placebo.
About the FREEDOMS and FREEDOMS II trials FREEDOMS and FREEDOMS II are pivotal phase 3, 2-year, placebo-controlled trials that assessed the efficacy and safety of Gilenya,. Although the study designs were similar, patient baseline characteristics were different between FREEDOMS and FREEDOMS II, including patient age, disease duration and previous treatment history-.
The pooled post-hoc analyses presented at AAN look at clinical and MRI outcomes in patients previously treated in the past year who had at least one relapse in the previous year and either at least one gadolinium-enhancing T1 lesion or at least 9 T2 lesions at baseline; or a patient who had equal or more relapses in the year before baseline than in the previous year.
The clinical and MRI outcomes were annualized relapse rate (ARR), time to 3 and 6 month confirmed disability progression, the percentage of change from baseline in brain volume (brain volume loss), the number of gadolinium-enhancing T1 lesions and the number of new/newly enlarged T2 lesions (MRI lesions).
About Multiple Sclerosis Multiple sclerosis (MS) is a chronic disorder of the central nervous system (CNS) that disrupts the normal functioning of the brain, optic nerve and spinal cord. The evolution of MS results in an increasing loss of both physical (e.g. difficulty with walking) and cognitive (e.g. problems with mental tasks or memory) function. This has a substantial negative impact on the approximately 2.3 million people worldwide affected by MS, a disease that begins in early adulthood, most often between the ages of 20 and 40.
The loss of physical and cognitive function is driven by two main types of damage that both contribute to widespread loss of neurons (nerve cells in the brain and spinal cord that transmit impulses): discrete inflammatory lesions, focal damage, in the brain that can clinically manifest as relapses; and ongoing, more diffuse damage that starts early in the disease and causes the progressive loss of brain tissue, including neurons, and over time is associated with both physical and cognitive problems-.
About Gilenya Gilenya is the only oral disease modifying therapy (DMT) that works on four key measures of multiple sclerosis (MS) disease activity - relapses, MRI lesions, brain volume loss and disability progression,,-.
Gilenya reduces both the distinct inflammatory lesions in the brain (focal damage) that can clinically manifest as relapses, and the ongoing, underlying damage in the brain (diffuse damage) that starts early in the disease-,-. Diffuse damage often goes unnoticed, causes the loss of neurons and over time is associated with both physical and cognitive problems-. Gilenya's reduction of both focal damage and diffuse damage is due to its impact on the inflammatory process (peripheral action) and its ability to enter the CNS and impact from within the CNS (central action)-. It is by addressing both focal and diffuse damage that the course of MS can be effectively impacted, helping to preserve a patient's physical (e.g. difficulty with walking) and cognitive (e.g. problems with mental tasks or memory) function.
To date, more than 91,500 patients worldwide have been treated with Gilenya in both clinical trial and post-marketing setting.
About Novartis in Multiple Sclerosis Novartis is committed to the research and development of new treatment options to offer the right treatment to the right patient at the right time, to meet patients' needs at every stage of disease with innovative and targeted drugs.
In addition to its ongoing development program for Gilenya in primary progressive MS (PPMS), pediatric MS and chronic inflammatory demyelinating polyneuropathy (CIPD), the Novartis MS portfolio includes Extavia® (interferon beta-1b for subcutaneous injection). Investigational compounds include BAF312 (siponimod), currently in Phase III clinical development and being developed as the first oral therapy for secondary progressive MS (SPMS), and VAY736, an anti-B-cell compound for MS that is currently being investigated in proof of concept studies. Novartis is also exploring the IL-17 pathway in MS.
Disclaimer The foregoing release contains forward-looking statements that can be identified by words such as "recommends," "positive opinion," "can," "committed," "ongoing," "investigational," "being developed," "being investigated," "exploring," or similar terms, or by express or implied discussions regarding potential future indications or labeling for Gilenya, potential future marketing submissions or approvals for the other investigational compounds in the Novartis MS portfolio, or regarding potential future revenues from any or all of the products and investigational compounds in the Novartis MS portfolio, including Gilenya. You should not place undue reliance on these statements. Such forward-looking statements are based on the current beliefs and expectations of management regarding future events, and are subject to significant known and unknown risks and uncertainties. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those set forth in the forward-looking statements. There can be no guarantee that Gilenya will be submitted or approved for any additional indications or labeling in any market, or at any particular time. Nor can there be any guarantee that any of the investigational compounds in the Novartis MS portfolio will be submitted or approved for sale in any market, or at any particular time. Neither can there be any guarantee that any of the products and investigational compounds in the Novartis MS portfolio will be commercially successful in the future. In particular, management's expectations regarding these products could be affected by, among other things, the uncertainties inherent in research and development, including unexpected clinical trial results and additional analysis of existing clinical data; unexpected regulatory actions or delays or government regulation generally; the company's ability to obtain or maintain proprietary intellectual property protection; general economic and industry conditions; global trends toward health care cost containment, including ongoing pricing pressures; unexpected manufacturing issues, and other risks and factors referred to in Novartis AG's current Form 20-F on file with the US Securities and Exchange Commission. Novartis is providing the information in this press release as of this date and does not undertake any obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise.
About Novartis Novartis provides innovative healthcare solutions that address the evolving needs of patients and societies. Headquartered in Basel, Switzerland, Novartis offers a diversified portfolio to best meet these needs: innovative medicines, eye care, cost-saving generic pharmaceuticals, preventive vaccines, over-the-counter and animal health products. Novartis is the only global company with leading positions in these areas. In 2013, the Group achieved net sales of USD 57.9 billion, while R&D throughout the Group amounted to approximately USD 9.9 billion (USD 9.6 billion excluding impairment and amortization charges). Novartis Group companies employ approximately 135,000 full-time-equivalent associates and sell products in more than 150 countries around the world. For more information, please visit http://www.novartis.com.
References  http://www.ema.europa.eu/ema/index.jsp?curl=pages/medicines/human/medicines/002202/human_med_001433.jsp&mid=WC0b01ac058001d125. Accessed April 2014.  Bergvall et al. Efficacy of fingolimod in pre-treated patients with disease activity: pooled analyses of FREEDOMS and FREEDOMS II. Poster presented at: 66th AAN Annual Meeting; April 26 - May 3, 2014; Philadelphia, Pennsylvania. Poster P03.174.  Kappos L, Radue E-W, O'Connor P, et al; for FREEDOMS Study Group. A placebo-controlled trial of oral fingolimod in relapsing multiple sclerosis.  Vollmer et al. Long-Term Safety of Fingolimod in Patients with Relapsing-Remitting Multiple Sclerosis: Results from Phase 3 FREEDOMS II Extension Study. Abstract presented at AAN, San Diego, March 2013.  http://www.ncbi.nlm.nih.gov/pubmedhealth/PMH0001747/. Accessed April 2014.  http://www.nationalmssociety.org/about-multiple-sclerosis/what-we-know-about-ms/symptoms/index.aspx. Accessed April 2014.  http://www.msif.org/includes/documents/cm_docs/2013/m/msif-atlas-of-ms-2013-report.pdf?f=1. Accessed April 2014.  http://emsp.org/multiple-sclerosis/ms-fact-sheet. Accessed April 2014.  Filippi M et al. Association between pathological and MRI findings in multiple sclerosis. Lancet Neurol. 2012 Apr;11(4):349-60.  Kutzelnigg A et al. Cortical demyelination and diffuse white matter injury in multiple sclerosis. Brain. 2005 Nov;128(Pt 11):2705-12.  Sormani MP, Arnold DL & De Stefano N. Treatment effect on brain atrophy correlates with treatment effect on disability in multiple sclerosis. Ann Neurol. 2014 Jan;75(1):43-9.  Cohen JA et al.; for TRANSFORMS Study Group. Oral fingolimod or intramuscular interferon for relapsing multiple sclerosis. N Engl J Med. 2010;362(5):402-415.  Kappos L, Radue E-W, O'Connor P, et al. Phase 3 FREEDOMS study extension: fingolimod (FTY720) efficacy in patients with relapsing-remitting multiple sclerosis receiving continuous or placebo-fingolimod switched therapy for up to 4 years. Poster presented at: 28th Congress of the European Committee for Treatment and Research in Multiple Sclerosis; October 10-13, 2012; Lyon, France. Poster P979.  Montalban et al. Long-term efficacy of fingolimod in patients with relapsing-remitting multiple sclerosis previously treated with interferon beta-1a or disease-modifying therapies: A Post-hoc analysis of the TRANSFORMS 4.5 year extension study. European Neurological Society, June 10, 2013 P539.  Chin PS et al. Early effect of fingolimod on clinical and MRI related outcomes in relapsing multiple sclerosis. Poster presented at: 28th Congress of the European Committee for Treatment and Research in Multiple Sclerosis; October 10-13, 2012; Lyon, France. Abstract P459.  Brinkmann V. FTY720 (fingolimod) in multiple sclerosis: therapeutic effects in the immune and the central nervous system. Br J Pharmacol 2009;158(5):1173-1182.  Chun J & Hartung HP. Mechanism of Action of Oral Fingolimod (FTY720) in Multiple Sclerosis. Clin Neuropharmacol. 2010 March-April;33(2):91-101.  Data on file. Novartis Pharmaceuticals.
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