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

2. 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

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 

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