Alexion Pharmaceuticals
Alexion is a biopharmaceutical organization focused on research, development and global delivery of therapies for rare diseases. Its activities include discovery and development of complement-targeted and other biologic therapies, conducting clinical trials and operating global manufacturing and supply-chain operations. The organization also maintains patient support programs, grantmaking for health equity, and regulatory and medical information infrastructures.
Industries
Nr. of Employees
Very Large (1000+)
Alexion Pharmaceuticals
Patents
Subcutaneous (SC) administration of anti-C5 antibodies for treatment of complement-associated conditions
US-12617846-B2
View DetailsMethod of treating neuromyelitis optica spectrum disorder (NMOSD) comprising administering an anti-C5 antibody
US-12612451-B2
View Details
Subcutaneous (SC) administration of anti-C5 antibodies for treatment of complement-associated conditions
US-12617846-B2
View DetailsMethod of treating neuromyelitis optica spectrum disorder (NMOSD) comprising administering an anti-C5 antibody
US-12612451-B2
View DetailsProducts
Complement-targeting biologic therapies
Biologic therapies that inhibit components of the complement system for treatment of complement-mediated rare diseases (intravenous or subcutaneous delivery).
Enzyme-replacement therapies for metabolic disorders
Therapies based on replacement of deficient enzymes to treat inherited metabolic and bone disorders.
Targeted small-molecule therapies in rare disease indications
Orally administered targeted therapies used for specific rare disease indications based on pathway modulation.
Voydeya
Danicopan tablets for oral use, indicated as an add-on therapy to ravulizumab or eculizumab for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH) who have residual hemolytic anemia due to extravascular hemolysis.
KANUMA
Kanuma (sebelipase alfa) is a recombinant enzyme replacement therapy indicated for the treatment of lysosomal acid lipase (LAL) deficiency in infants, children, and adults.
STRENSIQ
Strensiq (asfotase alfa) is a human recombinant tissue-nonspecific alkaline phosphatase-Fc-deca aspartate fusion protein solution for injection used as an enzyme replacement therapy for patients with confirmed diagnosis of pediatric-onset hypophosphatasia (HPP).
Complement-targeting biologic therapies
Biologic therapies that inhibit components of the complement system for treatment of complement-mediated rare diseases (intravenous or subcutaneous delivery).
Enzyme-replacement therapies for metabolic disorders
Therapies based on replacement of deficient enzymes to treat inherited metabolic and bone disorders.
Targeted small-molecule therapies in rare disease indications
Orally administered targeted therapies used for specific rare disease indications based on pathway modulation.
Voydeya
Danicopan tablets for oral use, indicated as an add-on therapy to ravulizumab or eculizumab for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH) who have residual hemolytic anemia due to extravascular hemolysis.
KANUMA
Kanuma (sebelipase alfa) is a recombinant enzyme replacement therapy indicated for the treatment of lysosomal acid lipase (LAL) deficiency in infants, children, and adults.
STRENSIQ
Strensiq (asfotase alfa) is a human recombinant tissue-nonspecific alkaline phosphatase-Fc-deca aspartate fusion protein solution for injection used as an enzyme replacement therapy for patients with confirmed diagnosis of pediatric-onset hypophosphatasia (HPP).
Services
End-to-end support for preclinical and clinical studies in rare diseases, including patient-centric design, site networks and data collection.
Support for including local sites and patients in multinational clinical trials to increase access to investigational therapies and foster international collaboration.
Personalized programs for patient education, reimbursement navigation, financial assistance and treatment support for rare disease patients and caregivers.
Competitive grant programs through a charitable foundation focused on diagnostics access, technology innovation, patient support and policy/research.
End-to-end support for preclinical and clinical studies in rare diseases, including patient-centric design, site networks and data collection.
Support for including local sites and patients in multinational clinical trials to increase access to investigational therapies and foster international collaboration.
Personalized programs for patient education, reimbursement navigation, financial assistance and treatment support for rare disease patients and caregivers.
Competitive grant programs through a charitable foundation focused on diagnostics access, technology innovation, patient support and policy/research.
Expertise Areas
- Rare disease drug discovery and development
- Complement system therapeutics
- Clinical trial management for rare diseases
- Genomic medicine and newborn screening
Key Technologies
- Complement cascade targeting
- Monoclonal antibodies and biologic modalities
- Enzyme-replacement therapy modalities
- Genomic sequencing and analysis
News & Updates
Article describing the use of data science and AI to accelerate diagnosis in rare diseases.
Discussion of genomic approaches to improve diagnostics and care pathways for rare diseases.
Article describing the use of data science and AI to accelerate diagnosis in rare diseases.
Discussion of genomic approaches to improve diagnostics and care pathways for rare diseases.