1 / 6

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

maryajones
Download Presentation

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

An Image/Link below is provided (as is) to download presentation Download Policy: Content on the Website is provided to you AS IS for your information and personal use and may not be sold / licensed / shared on other websites without getting consent from its author. Content is provided to you AS IS for your information and personal use only. Download presentation by click this link. While downloading, if for some reason you are not able to download a presentation, the publisher may have deleted the file from their server. During download, if you can't get a presentation, the file might be deleted by the publisher.

E N D

Presentation Transcript


  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 (?)

More Related