Amryt Pharma
Amryt Pharma is a global biopharmaceutical company focused on acquiring, developing, and commercializing treatments for rare and orphan diseases. Their mission is to transform the lives of patients affected by rare, debilitating conditions by providing innovative medicines. They operate with responsibility, integrity, and ethical practices, engaging with patient groups, healthcare professionals, and communities to improve access to treatments and support charitable organizations. The company has a strong infrastructure for global growth and a leadership team with expertise in rare diseases. Amryt Pharma is committed to scientific research, regulatory compliance, and transparency in its interactions and support for patient organizations. It offers a portfolio of medicines including Myalept®, Mycapssa®, Juxtapid®/Lojuxta®, and Filsuvez®, and is involved in clinical trials, expanded access programs, and advocacy efforts.
Industries
Nr. of Employees
large (251-1000)
Amryt Pharma
Dept 920A, 196 High Road, Wood Green, London N22 8HH, United Kingdom
Products
Metreleptin (recombinant leptin replacement therapy)
Adjunctive replacement therapy for complications of leptin deficiency in patients with congenital or acquired generalised lipodystrophy and, in some jurisdictions, certain partial lipodystrophy patients.
Oral octreotide capsule formulation (oral somatostatin analogue)
Oral somatostatin analogue formulation indicated for long-term maintenance therapy in patients who have responded to and tolerated injectable somatostatin analogues.
Lomitapide (MTP inhibitor for HoFH)
Oral lipid-lowering therapy used as an adjunct to diet and other lipid-lowering medicinal products for adults with Homozygous Familial Hypercholesterolaemia.
Topical oleogel formulation for epidermolysis bullosa wounds
Topical oleogel indicated for treatment of partial thickness wounds associated with junctional and dystrophic forms of Epidermolysis Bullosa in pediatric and adult patients.
AP103 (preclinical gene therapy candidate)
Preclinical gene therapy candidate targeting dystrophic epidermolysis bullosa, leveraging a polymer-based delivery platform with potential application to other genetic disorders.
Metreleptin (recombinant leptin replacement therapy)
Adjunctive replacement therapy for complications of leptin deficiency in patients with congenital or acquired generalised lipodystrophy and, in some jurisdictions, certain partial lipodystrophy patients.
Oral octreotide capsule formulation (oral somatostatin analogue)
Oral somatostatin analogue formulation indicated for long-term maintenance therapy in patients who have responded to and tolerated injectable somatostatin analogues.
Lomitapide (MTP inhibitor for HoFH)
Oral lipid-lowering therapy used as an adjunct to diet and other lipid-lowering medicinal products for adults with Homozygous Familial Hypercholesterolaemia.
Topical oleogel formulation for epidermolysis bullosa wounds
Topical oleogel indicated for treatment of partial thickness wounds associated with junctional and dystrophic forms of Epidermolysis Bullosa in pediatric and adult patients.
AP103 (preclinical gene therapy candidate)
Preclinical gene therapy candidate targeting dystrophic epidermolysis bullosa, leveraging a polymer-based delivery platform with potential application to other genetic disorders.
Services
Review and provision of investigational medicines outside clinical trials for individual patients, subject to regulatory and ethics approvals and governance review.
Global medical information contact service for enquiries, adverse event reporting, product complaints and RMP material requests via email and country-specific phone numbers.
Identification and transaction execution for in‑licensing or acquiring rare disease assets to expand the company portfolio.
Funding programme to support independent medical education and patient organisation activities, with application, review and post-event reconciliation requirements.
Review and provision of investigational medicines outside clinical trials for individual patients, subject to regulatory and ethics approvals and governance review.
Global medical information contact service for enquiries, adverse event reporting, product complaints and RMP material requests via email and country-specific phone numbers.
Identification and transaction execution for in‑licensing or acquiring rare disease assets to expand the company portfolio.
Funding programme to support independent medical education and patient organisation activities, with application, review and post-event reconciliation requirements.
Expertise Areas
- Clinical trial management
- Expanded access and compassionate use
- Pharmacovigilance and risk management
- Regulatory submissions and global approvals
Key Technologies
- Polymer-based gene delivery platform
- Gene therapy (preclinical vector development)
- Oral peptide formulation technology
- Topical oleogel wound-healing formulation