Chiasma, Inc.
Amryt Pharma is a global biopharmaceutical company dedicated to acquiring, developing, and commercializing innovative treatments for rare and orphan diseases. Their mission is to transform the lives of patients affected by rare, debilitating conditions by providing medicines that bring hope to those in greatest need. They operate with responsibility, integrity, and ethical practices, forging partnerships and collaborations to ensure their medicines reach those who need them. The company has a strong focus on rare diseases, with a portfolio of commercial and development assets, and a leadership team with deep heritage in rare diseases. They aim to be a leading global rare disease company by acquiring, developing, and commercializing medicines that improve patient lives worldwide.
Industries
Nr. of Employees
medium (51-250)
Chiasma, Inc.
Needham, Massachusetts, United States, North America
Products
Metreleptin (recombinant leptin therapy)
Recombinant leptin replacement therapy indicated as adjunct to diet for complications of leptin deficiency in certain forms of lipodystrophy.
Oral octreotide capsules (oral somatostatin analog formulation)
Oral peptide formulation for long-term maintenance therapy in acromegaly patients who have responded to and tolerated somatostatin analogs.
Lomitapide (small-molecule lipid-lowering agent)
Small-molecule therapy indicated as adjunct to diet and other lipid-lowering medicinal products for adults with Homozygous Familial Hypercholesterolaemia (HoFH).
Oleogel-S10 (topical wound therapy formulation)
Topical formulation approved in the EU and Great Britain for treatment of partial thickness wounds associated with certain forms of Epidermolysis Bullosa.
Metreleptin (recombinant leptin therapy)
Recombinant leptin replacement therapy indicated as adjunct to diet for complications of leptin deficiency in certain forms of lipodystrophy.
Oral octreotide capsules (oral somatostatin analog formulation)
Oral peptide formulation for long-term maintenance therapy in acromegaly patients who have responded to and tolerated somatostatin analogs.
Lomitapide (small-molecule lipid-lowering agent)
Small-molecule therapy indicated as adjunct to diet and other lipid-lowering medicinal products for adults with Homozygous Familial Hypercholesterolaemia (HoFH).
Oleogel-S10 (topical wound therapy formulation)
Topical formulation approved in the EU and Great Britain for treatment of partial thickness wounds associated with certain forms of Epidermolysis Bullosa.
Services
Expanded access programme
Review and potential supply of investigational medicines to individual patients outside clinical trials, contingent on regulatory/ethics approvals and program criteria.
Global medical information service
Centralised medical information contact for medical enquiries, adverse event reporting and provision of RMP materials with country-specific contact numbers.
Grants and donations administration
Management and disbursement of educational grants and charitable donations to support independent medical education and patient organisations, including application and reconciliation processes.
Business development and licensing support
Evaluation and execution support for in-licensing, asset acquisition and partnership opportunities in rare disease therapeutics.
Clinical trials and registries support
Operational support for running clinical studies and patient registries to generate clinical evidence for regulatory submissions and reimbursement.
Compliance and transparency reporting
Administration of corporate compliance resources, Open Payments reporting and country-specific disclosure publishing for HCP/HCO interactions and patient organisation support.
Expanded access programme
Review and potential supply of investigational medicines to individual patients outside clinical trials, contingent on regulatory/ethics approvals and program criteria.
Global medical information service
Centralised medical information contact for medical enquiries, adverse event reporting and provision of RMP materials with country-specific contact numbers.
Grants and donations administration
Management and disbursement of educational grants and charitable donations to support independent medical education and patient organisations, including application and reconciliation processes.
Business development and licensing support
Evaluation and execution support for in-licensing, asset acquisition and partnership opportunities in rare disease therapeutics.
Clinical trials and registries support
Operational support for running clinical studies and patient registries to generate clinical evidence for regulatory submissions and reimbursement.
Compliance and transparency reporting
Administration of corporate compliance resources, Open Payments reporting and country-specific disclosure publishing for HCP/HCO interactions and patient organisation support.
Expertise Areas
- Clinical trial management
- Expanded access / compassionate use management
- Pharmacovigilance and safety monitoring
- Regulatory strategy and submissions (FDA/EMA)
Key Technologies
- Gene therapy
- Polymer-based delivery platforms
- Recombinant protein biologics
- Oral peptide formulation