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Amicus Therapeutics & Galafold ( migalastat ) Jiri Hermanek, GM CEE

Amicus Therapeutics & Galafold ( migalastat ) Jiri Hermanek, GM CEE. Product Pipeline Multiple technology platforms. Monotherapy - Personalized Medicine ( Galafold ). Co-Administration with ERT. Next-Generation ERT. Fabry Disease: Pathophysiology. Mutations in the GLA gene

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Amicus Therapeutics & Galafold ( migalastat ) Jiri Hermanek, GM CEE

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  1. Amicus Therapeutics & Galafold(migalastat)Jiri Hermanek, GM CEE

  2. Product PipelineMultiple technology platforms Monotherapy - Personalized Medicine (Galafold) Co-Administration with ERT Next-Generation ERT

  3. Fabry Disease: Pathophysiology Mutations in the GLA gene Reduced lysosomal alpha-galactosidase A (α-Gal A) activity Accumulation of globotriaosylceramide (GL-3) and Lyso-Gb3 Multisystemic disease: Cardiovascular disease, renal failure, stroke, gastrointestinal symptoms, pain, hearing loss Premature death

  4. CONFIDENTIAL GLP Amenability Assay 100% WT 3% WT • The assay data show that >300 of >800 tested α-Gal A mutations (to the date of marketing authorization approval) meet the criteria for amenability  www.galafoldamenabilitytable.com

  5. Galafold (migalastat) Personalized medicine for Fabry patients • Novel personalized treatment concept for Fabry disease: only patients with amenable mutations can be treated with Galafold (migalastat) • First orally administered small molecule (every other day dosing): • Comparable clinical efficacy to ERT • No risk of immunogenicity (small molecule) • No infusion-associated reactions

  6. Galafold (migalastat) Personalized medicine for Fabry patients • EU Marketing Authorization Approval granted May 30th 2016 • Reimbursed already in multiple EU countries (Austria, Belgium, Denmark, Finland, France, Germany, Ireland, Italy, Norway, Sweden, UK)  >250 patients on therapy • Hungary: • Pricing & reimbursement application submitted to NEAK on December 22nd2016  recommendation for reimbursement in “individual patient request” category  final stage of negotiating reimbursement agreement with NEAK  available from January 2018 (?)

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