Scioderm
Biopharmaceutical company focused on discovery, development and commercialization of therapies for rare genetic diseases. Activities described include preclinical research (pharmacological chaperones and gene therapy), clinical development (multiple Phase 1–3 studies and registries for Fabry and Pompe diseases), patient support and access programs, global medical affairs including independent medical education grants, and investigator-initiated study support.
Industries
Nr. of Employees
small (1-50)
Scioderm
Products
Galafold (migalastat)
Orally administered therapy indicated in the U.S. for adults with Fabry disease who have an amenable GLA variant based on in vitro assay data; approval under accelerated pathway based on reduction of a renal substrate biomarker.
Pombiliti (cipaglucosidase alfa) in combination with Opfolda (miglustat)
Combination regimen indicated in the U.S. for treatment of adult patients with late-onset Pompe disease (GAA deficiency) ≥40 kg who are not improving on current enzyme replacement therapy; includes boxed warnings and infusion/ hypersensitivity risks.
Galafold (migalastat)
Orally administered therapy indicated in the U.S. for adults with Fabry disease who have an amenable GLA variant based on in vitro assay data; approval under accelerated pathway based on reduction of a renal substrate biomarker.
Pombiliti (cipaglucosidase alfa) in combination with Opfolda (miglustat)
Combination regimen indicated in the U.S. for treatment of adult patients with late-onset Pompe disease (GAA deficiency) ≥40 kg who are not improving on current enzyme replacement therapy; includes boxed warnings and infusion/ hypersensitivity risks.
Services
Patient support program (patient assistance and access)
Personalized financial, insurance and case-management assistance for patients prescribed company therapies; multilingual phone and web support.
Expanded Access Program (EAP) management
Operational pathways, intake forms and policy to consider requests for investigational drug access for eligible patients.
Independent Medical Education (IME) grant funding
Funding and administration of accredited and non-accredited medical education grants with outcome assessment and reconciliation requirements.
Investigator-Initiated Study (IIS) support
Review, funding and, in some cases, provision of drug supply for investigator-led clinical, observational and non-clinical studies aligned with areas of interest.
Patient support program (patient assistance and access)
Personalized financial, insurance and case-management assistance for patients prescribed company therapies; multilingual phone and web support.
Expanded Access Program (EAP) management
Operational pathways, intake forms and policy to consider requests for investigational drug access for eligible patients.
Independent Medical Education (IME) grant funding
Funding and administration of accredited and non-accredited medical education grants with outcome assessment and reconciliation requirements.
Investigator-Initiated Study (IIS) support
Review, funding and, in some cases, provision of drug supply for investigator-led clinical, observational and non-clinical studies aligned with areas of interest.
Expertise Areas
- Clinical trial management for rare diseases
- Preclinical therapeutics development (pharmacological chaperones, gene therapy)
- Enzyme replacement therapy development
- Real-world evidence and patient registries
Key Technologies
- Gene therapy
- Pharmacological chaperones
- Enzyme replacement therapy
- Biomarker discovery and translational research