Metabolism Drugs Market Analysis by Automation Type and Applications with Top Players Merck, Novartis AG, Amicus Therapeutics, AstraZeneca, Boehringer Ingelheim, Shire

Manufacturers are actively working on developing new therapies for various metabolic diseases. For instance, various pipeline products include NeoGAA GZ402666 (Sanofi) in phase 3, AT-982 in phase 3 (Audentes Therapeutics)), and BMN 701 in phase 2 (BioMarin Pharmaceutical, Inc.) clinical trial are also expected to create a meteoric growth in this market during the forecast period.

Amicus Therapeutics was granted with orphan drug designation for its investigational combination drug therapy ATB200/AT2221, in November 2017. This drug is intended to cure Pompe disease (acid maltase deficiency or AMD). in March 2018, Shire plc entered into a preclinical research collaboration with NanoMedSyn— French biotech company—to develop enzyme replacement treatment for a lysosomal storage disorder based on NanoMedSyn’s synthetic derivative technology named AMFA.

Lixte Biotechnology Holdings, Inc., has LB-201/LB-205 in the preclinical stage, JCR Pharmaceuticals Co. Ltd. filed for JR-051 in Japan in 2017, and has many other products in the pipeline for treatment of metabolic diseases. Moreover, manufacturers such as Pharming Group NV and Orphazyme ApS also have products in the pipeline for metabolic diseases. Partnerships and collaboration from individual organizations for research and development of effective therapies is expected to boost growth of the metabolism drugs market. For instance, in 2014, Shire Plc announced collaboration with ArmaGen to develop AGT-182 drug for treatment of hunter syndrome.

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Moreover, in 2017, Research funded by the National Institute of Neurological Disorders and Stroke (NINDS) demonstrated that viral-delivered gene therapy can stop the buildup materials stored in the brain cells and improve memory and learning. High return on investments, health insurance coverage, and various reimbursement policies for such costly drugs are influencing pharmaceutical companies to invest in drug development.

For instance, in 2015, Orphan Product Grants Program funded 18 new grant awards from 92 grant applications and provided funding and support to around 67 ongoing clinical study projects related to rare disease. Furthermore, initiatives from the governments of economies such as Sweden, the Netherlands, and South Korea that focus on approving guidelines for the reimbursement of individuals undergoing enzyme replacement therapy (ERT) are also expected to boost growth of the metabolic drugs market (as enzyme replacement being major therapies in the market) in the near future.

Metabolism diseases results from malfunctioning or inadequate function of the body’s original metabolism. Metabolism diseases can also have a genetic origin, as they can be caused by genetic birth defects as well. Metabolic diseases from genetic defects have low incidence and prevalence rate. Metabolism involves breaking down of carbohydrates, proteins, and fats present in the consumed food, to generate energy.

Furthermore, metabolism also involves excretion of nitrogen through urine, and breaking down or converting other substances and chemicals. This metabolism is carried out by certain enzymes and proteins designated for the respective role. However, metabolism functions improperly when enzymes and proteins are either not produced by the body (inherited disorders), or the produced enzymes do not function the way they are mean to.

Metabolic disorders occur due to buildup of proteins, lipids, glycogen, urea, calcium stones, and toxic substances among others. Each of these molecules have certain designated metabolic pathways, and metabolic agents (enzymes, proteins) at work. Inability of these agents to carry out their tasks cause buildup of the molecules in the blood and other places. Few examples of metabolic diseases include gaucher disease, hereditary tyrosinemia, hereditary orotic aciduria, and fabry disease.

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