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

Product Pipeline Multiple technology platforms PRODUCT/PLATFORM DISCOVERY PRECLINICAL PHASE 1 PHASE 2 PHASE 3 Regulatory Fabry   Migalastat Pharmacological Chaperone Monotherapy & Combination w/ ERT Pompe Next-Generation ERT + Chaperone Monotherapy - Personalized Medicine (Galafold) Co-Administration with ERT Next-Generation ERT

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

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

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

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 22nd 2016  recommendation for reimbursement in “individual patient request” category  final stage of negotiating reimbursement agreement with NEAK  available from January 2018 (?)