According to study, “Primary Immune Deficiency (PID) – Pipeline Review, H2 2018” some of the major players that are working in the primary immune deficiency are Biotest AG, Cellective Bio TherapyInc, ADMA Biologics Inc, Casebia Therapeutics, Genethon SA, GC Pharma, GigaGenIncm, Novartis AG, Leadiant Biosciences Inc, Octa pharma AG, Sangamo Therapeutics Inc, ProMetic Life Sciences Inc, Shire Plc, Therapure BiopharmaInc, Taiga Biotechnologies Inc, UCB SA, X4 Pharmaceuticals Inc.
Primary immunodeficiency (PTD) is a large group of disorders in which part of the body’s immune system is missing or does not function normally. It allows infections and other health problems to occur more easily. Some forms of PID are usually hereditary while some disorders are not recognized until adulthood. There are many signs and symptoms of deficiency such as blood disorders, digestive problems, delayed growth & development, autoimmune disorders, frequent & recurrent pneumonia, ear& skin infection and inflammation & infection of internal organs etc.
PIDs are classifies into many categories according to the component of the immune system such as T cell deficiencies, B cell deficiency, combination B & T cell deficiency, complement deficiencies and idiopathic growth hormone deficiency etc. T cell deficiency is caused by decreased function of individual T cell. T cell’s normal function is to help with the human body’s immunity. B cell deficiency is caused by a lack of infection-fighting producing B cells or immunoglobulin that is not functioning properly. Combined B-cell and T-cell immunodeficiency is a group of medical disorders that are the result of genetic defects in cellular and humoral immunity, also called severe combined immunodeficiency.
There are many complications are involved in PID such as recurrent infections, autoimmune disorders, damage to (heart, lungs, nervous system or digestive tract), slowed growth, increased risk of cancer and death from serious infection. There are many drug profiles are involved in primary immune deficiency, which are; BT-595, aldesleukin, cell therapyfor RAG deficient, cellular immunotherapy, gene therapy for x-linked SCID, elapegademase, gene therapy to activate IL2RG for x-linked SCID, hyaluronidase & immune globulin, OTL-101, RI-002, RPL-201, seletalisib, stem cell therapy to activate IL2RG for SCID-X1, TBX-1400, strimvelis and X-4P001.
BIVIGAM is an intravenous immune globulin indicated for the treatment of primary humeral immunodeficiency. It includes agammaglobulinemia, Wiskott-Aldrich syndrome and severe combined immunodeficiency. BIVIGAM is a purified, sterile, ready-to-use preparation of concentrated polyclonal immunoglobulin antibodies. Antibodies are proteins in the human immune system that work to defend against infections and disease. In June 2018, ADMA optimized the production process for BIVIGAM and submitted a prior approval supplement to the United States food & drug administration to amend the biologics license application with a target action under the prescription drug user fee act. If prior approval supplement is approved by the food & drug administration. The immunodeficiency can be subtle and vary in presentation which involves recurrent or unusual infections, malignancies or autoimmune phenomena.Primary care providers assess and treat PID’s patients a clinical immunologist can advise on further diagnostic tests and management of patients. Collaborative patient care among health care providers can save patient lives and improve long-term outcomes.
At present, there is no single assay that identifies all forms of PID. Therefore need a challenge of current newborn screening paradigms andit is estimated that PID will involve up-front next generation sequencing.
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