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

Galafold from Amicus Therapeutics offers personalized treatment for Fabry patients with amenable mutations. Approved in EU, reimbursed in multiple countries, and shows comparable efficacy to ERT. No immunogenicity risk or infusion reactions.

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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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