U.S. FDA grants Breakthrough Therapy Designation for Roche's investigational medicine ocrelizumab in primary progressive multiple sclerosis

Roche Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for the investigational medicine ocrelizumab (OCREVUSTM) for the treatment of people with primary progressive multiple sclerosis (PPMS). There are currently no approved treatments for PPMS, a debilitating form of MS characterised by steadily worsening symptoms and typically without distinct relapses or periods of remission.(1)

"Ocrelizumab is the first investigational medicine for MS to be granted Breakthrough Therapy Designation by the FDA," said Sandra Horning, M.D., Roche's Chief Medical Officer and Head of Global Product Development. "With no approved treatments for primary progressive MS, ocrelizumab has the potential to address an important unmet need. We are committed to working with the FDA to bring ocrelizumab to people with primary progressive MS as quickly as possible."

Breakthrough Therapy Designation is designed to expedite the development and review of medicines intended to treat serious or life-threatening diseases and to help ensure people have access to them through FDA approval as soon as possible. The designation is based on positive results from the pivotal Phase III study (called ORATORIO), which showed treatment with ocrelizumab significantly reduced disability progression and other markers of disease activity compared with placebo. Top-line results were presented at the 31st congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) in October 2015.

Roche plans to pursue marketing authorisation for both PPMS and relapsing multiple sclerosis (RMS), a more common form of the disease, and will submit data from three pivotal Phase III studies to global regulatory authorities in the first half of 2016.

OCREVUSTM is the proprietary name submitted to global regulatory authorities for the investigational medicine ocrelizumab.

About ocrelizumab
Ocrelizumab is an investigational, humanised monoclonal antibody designed to selectively target CD20-positive B cells. CD20-positive B cells are a specific type of immune cell thought to be a key contributor to myelin (nerve cell insulation and support) and axonal (nerve cell) damage, which can result in disability in people with MS. Based on preclinical studies, ocrelizumab binds to CD20 cell surface proteins expressed on certain B cells, but not on stem cells or plasma cells, and therefore important functions of the immune system may be preserved.

In addition to ORATORIO, the Phase III clinical development programme for ocrelizumab includes OPERA I and OPERA II, which are randomised, double-blind, double-dummy, global multi-centre studies in people with relapsing forms of MS.

About the ORATORIO study in PPMS
ORATORIO is a Phase III, randomised, double-blind, global multi-centre study evaluating the efficacy and safety of ocrelizumab compared with placebo in 732 people with PPMS.(2) The primary endpoint of the study was time to onset of confirmed disability progression (CDP) sustained for at least 12 weeks.

CDP measures a sustained protocol-defined increase in a patient's Expanded Disability Status Scale (EDSS) score. The EDSS is based on a physical and neurological exam of eight systems throughout the body. The functional systems include vision, coordination, limb movement, strength, thinking abilities, bowel and bladder control, sensation and walking ability.

About multiple sclerosis
Multiple sclerosis (MS) is a chronic disease that affects an estimated 2.3 million people around the world, for which there is currently no cure.(3,4) MS occurs when the immune system abnormally attacks the insulation and support around nerve cells (myelin sheath) in the brain, spinal cord and optic nerves, causing inflammation and consequent damage. Damage to these nerves can cause a wide range of symptoms, including muscle weakness, fatigue and difficulty seeing, and may eventually lead to progressive disability.(5,6,7)

Primary progressive MS (PPMS) is a debilitating form of MS characterised by steadily worsening symptoms but typically without distinct relapses or periods of remission.1 Approximately one in 10 people with MS are diagnosed with the primary progressive form of the disease. There are no approved treatments for PPMS.

About Roche in neuroscience
Neuroscience is a major focus of research and development at Roche. The company's goal is to develop treatment options based on the biology of the nervous system to help improve the lives of people with chronic and potentially devastating diseases. Roche has more than a dozen investigational medicines in clinical development for diseases that include multiple sclerosis, Alzheimer’s disease, spinal muscular atrophy, Parkinson’s disease, Down syndrome and autism.

About Roche
Roche is a global pioneer in pharmaceuticals and diagnostics focused on advancing science to improve people's lives.

Roche is the world's largest biotech company, with truly differentiated medicines in oncology, immunology, infectious diseases, ophthalmology and diseases of the central nervous system. Roche is also the world leader in in vitro diagnostics and tissue-based cancer diagnostics, and a frontrunner in diabetes management. The combined strengths of pharmaceuticals and diagnostics under one roof have made Roche the leader in personalised healthcare - a strategy that aims to fit the right treatment to each patient in the best way possible.

Founded in 1896, Roche continues to search for better ways to prevent, diagnose and treat diseases and make a sustainable contribution to society. Twenty-nine medicines developed by Roche are included in the World Health Organization Model Lists of Essential Medicines, among them life-saving antibiotics, antimalarials and cancer medicines. Roche has been recognised as the Group Leader in sustainability within the Pharmaceuticals, Biotechnology & Life Sciences Industry seven years in a row by the Dow Jones Sustainability Indices.

The Roche Group, headquartered in Basel, Switzerland, is active in over 100 countries and in 2015 employed more than 91,700 people worldwide. In 2015, Roche invested CHF 9.3 billion in R&D and posted sales of CHF 48.1 billion. Genentech, in the United States, is a wholly owned member of the Roche Group. Roche is the majority shareholder in Chugai Pharmaceutical, Japan.

1. MS International Federation. Types of MS. Available at: http://www.msif.org/about-ms/types-of-ms/.
2. F. Hoffmann-La Roche. ClinicalTrials.gov NCT01194570. National Library of Medicine. Available at: https://clinicaltrials.gov/ct2/show/NCT01194570.
3. Multiple Sclerosis International Federation. (2013). Atlas of MS 2013. Available at: http://www.msif.org/about-us/advocacy/atlas/.
4. National Institutes of Health-National Institute of Neurological Disorders and Stroke. (2015). Multiple Sclerosis: Hope Through Research. Available at: http://www.ninds.nih.gov/disorders/multiple_sclerosis/detail_multiple_sclerosis.htm#280373215.
5. Ziemssen T. (2005). Modulating processes within the central nervous system is central to therapeutic control of multiple sclerosis. J Neurol, 252(Suppl 5), v38-v45.
6. Hauser S.L. et al. (2012). Multiple sclerosis and other demyelinating diseases. In Harrison’s Principles of Internal Medicine (pp.3395-3409). New York, NY: McGraw Hill Medical.
7. Hadjimichael O. et al. (2007). Persistent pain and uncomfortable sensations in persons with multiple sclerosis. Pain, 127(1-2), 35-41.

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