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Multiple sclerosis is a chronic disease that affects each person differently, with symptoms ranging from numbness in the limbs or forgetfulness to paralysis or loss of vision. MS is caused when the body’s immune system attacks the central nervous system, damaging the myelin sheath – the protective layer covering the nerves that carry signals between the brain and spinal cord and the rest of the body. Finding effective treatments for such a complex disease is no easy task, but our MS research is spearheaded by top experts employing some of the most advanced and creative scientific problem-solving skills in the industry.
For over a decade, Genzyme and our parent company Sanofi have each been working to develop novel treatments for MS. We have integrated these programs within Genzyme to establish ourselves as a leader in MS.
AUBAGIO ®(teriflunomide) is approved as a once-daily oral treatment for relapsing forms of MS. AUBAGIO is supported by a robust clinical program and has demonstrated significant efficacy in the three key measures for an MS treatment – reducing annualized relapse rate (ARR), delaying the progression of disability, and reducing MRI (magnetic resonance imaging) disease activity.
Our MS clinical pipeline includes the late-stage product candidate alemtuzumab. Alemtuzumab clinical studies researched a unique dosing regimen for relapsing MS. Alemtuzumab showed promise in head to head clinical studies against an approved MS therapy and could make a very important contribution to the treatment landscape. Alemtuzumab has completed phase 3 trials.
While many existing MS treatments modulate the disease’s symptoms, our focus is to research and develop future treatments that reset the damaged immune system. Our scientists are currently researching a small molecule that could actually repair the myelin sheath. This team is also studying whether alemtuzumab, which has completed phase 3 studies for relapsing/remitting MS, can effectively treat progressive MS, a much more advanced form of the disease.
Drawing on our vast experience in ultra-rare diseases, we recognize that truly personalized medicine involves developing relationships with patients and caregivers, health care providers, and disease organizations and foundations.
Though MS affects a much larger population than lysosomal storage disorders, we are building our MS business unit on the same core principle that guides our Rare Disease business unit: the importance of deeply understanding the experiences, challenges, and triumphs of those affected by MS. We aim to foster close connections with patients and their families to best understand their needs and to deliver services that have a positive impact.
Our MS leadership comprises experienced professionals with a strong foundation in MS. We have an exciting opportunity to draw on the diverse experiences of our MS team to forge a unique identity for Genzyme within the MS community.