Pegaptanib is a selective vascular endothelial growth factor (VEGF) antagonist indicated for the treatment of neovascular (wet) age-related macular degeneration. Pegaptanib is an aptamer, a pegylated modified single-stranded oligonucleotide, which adopts a threedimensional conformation that enables it to bind to extracellular VEGF. Pegaptanib specifically binds to the 165 isoform of VEGF, a protein that plays a critical role in angiogenesis (the formation of new blood vessels) and increased permeability (leakage from blood vessels), two of the primary pathological processes responsible for the vision loss associated with neovascular age-related macular degeneration. Pegaptanib is administered in a 0.3 mg dose once every six weeks by intravitreal injection. An intravitreal injection is one that is administered directly into the eye, more specifically, into the vitreous humour, or the jelly-like fluid within the eye.

Immune globulin human (Human immunoglobulin) is a mixture of IgG1 and other antibodies derived from healthy human plasma and used to strengthen the body's natural defense system (immune system) to reduce the risk of infection in people with weakened immune systems. Human immunoglobulin is used as replacement therapy for inherited humoral immunodeficiency disorders, such as severe combined immunodeficiency syndrome, x-linked agammaglobulinemia, and Wiskott-Aldrich syndrome. Human immunoglobulin interacts with a number of different components of the immune system, including cytokines, complement, Fc receptors, and several immunocompetent cell surface molecules. Human immunoglobulin also acts on various effector cells of the immune system (B and T lymphocytes, dendritic cells, etc.) and regulates a wide range of genes. Human immune globulin competitively blocks gamma Fc receptors, preventing the binding and ingestion of phagocytes and inhibiting platelet depletion. Human immunoglobulin contains a number of different antibodies that prevent infection by attaching pathogenic microorganisms to the surface and facilitating their removal before they can infect cells. Antibodies remove pathogens by activating complement, agglutination or precipitation, blocking the pathogen receptor, “tagging” macrophages, or neutralizing the pathogen toxins. Serious adverse reactions are observed during intravenous treatment in clinical studies of aseptic meningitis. The most common adverse reactions were headache, fatigue, hyperthermia, nausea, chills, severity, pain in the limbs, diarrhea, migraine, dizziness, vomiting, cough, urticaria, asthma, sore throat and throat, rash, myalgia, itching, and cardiac murmur. During clinical trials of subcutaneous treatment, no serious adverse reactions were observed.

Amifampridine (Firdapse), currently approved in the European Union, is the first and only approved drug for the symptomatic treatment of Lambert-Eaton Myasthenic Syndrome (LEMS) in adults, a rare autoimmune disease with the primary symptoms of muscle weakness. In LEMS, the body’s own immune system attacks connections between nerves and muscles and disrupts the ability of nerve cells to send signals to muscle cells. Amifampridine blocks voltage-dependent potassium channels, thereby prolonging pre-synaptic cell membrane depolarization. Prolonging the action potential enhances the transport of calcium into the nerve ending. The resulting increase in intracellular calcium concentrations facilitates exocytosis of acetylcholine containing vesicles, which in turn enhances neuromuscular transmission. Amifampridine phosphate has been granted Orphan Drug Designation and Breakthrough Therapy designation by the FDA for the treatment of Lambert-Eaton Myasthenic Syndrome (LEMS).