Lipodystrophy is abnormal central fat accumulation or localized loss of fat tissue or mixed clinical presentations representing both. It is also known as the Lawrence-Seip syndrome, a rare group of syndromes, either acquired or congenital. People with lipodystrophy experience an uncontrolled loss of fat tissue, especially fat under the skin. This causes a drop in an important hormone called leptin. Without enough fat tissue or Leptin, the body's system for regulating energy use falls out of balance.
As per the Lipodystrophy United, the global prevalence of lipodystrophy has been estimated at less than 1 case in 100,000 people. According to an article published in the Journal of Diabetes, Metabolic Syndrome and Obesity: Targets and Therapy, in 2017 in Europe, the prevalence of lipodystrophy was 2.63 cases/million. The two most common acquired varieties of lipodystrophy include acquired generalized lipodystrophy and acquired partial lipodystrophy. Highly active antiretroviral therapy-induced lipodystrophy in HIV-infected patients and drug-induced localized lipodystrophy are other common sub-types. Lipodystrophy is a rare disease that affects fewer than 200,000 people in the U.S.. The global lipodystrophy treatment market is expected to expand at a robust pace during the forecast period. High return on investments for the development of orphan drugs, strong research and development initiatives for orphan drugs in developed countries, and increase in the number of rare diseases related to genetic mutations that are treated using orphan drugs are expected to drive the market in the coming years.
The global lipodystrophy treatment market can be segmented based on disease, drug class, end-user, and region. Based on disease, the lipodystrophy treatment market can be divided into acquired generalized lipodystrophy and acquired partial lipodystrophy. Till now, there is no cure for lipodystrophy, and the effects of treatment vary from person to person. There is only one FDA-approved treatment for generalized lipodystrophy (myalept), and there are no approved treatments for partial lipodystrophy. In terms of end-user, the global lipodystrophy treatment market ca be segmented into academic & research institutes, hospital, and others. Increase in R&D in the health care sector, which utilizes gene sequencing for the testing of orphan diseases, is expected to drive the lipodystrophy treatment market during the forecast period.
High rate of growth of the lipodystrophy treatment market can be attributed to the entry of novel, expensive therapies and high unmet needs. More high-priced therapies are expected during the forecast period. Kalydeco for cystic fibrosis and exon-skipping therapies for Duchenne muscular dystrophy are new therapies introduced in the market. Competitive activity is increasing as larger companies realize the rewards of investing in orphan therapeutics. High unmet needs and low competition in several regions are expected to attract more companies to the market in the near future.
Based on region, the lipodystrophy treatment market can be categorized into North America, Europe, Asia Pacific, Latin America, and Middle East & Africa. North America accounted for a major share of the lipodystrophy treatment market. According to a report of Research Office, Legislative Council Secretariat, there are between 25-30 million people suffering from rare diseases in the U.S.. Strong focus on research and development and government aids, such as research grants, tax credits, and fee waivers, to promote R&D contribute to the growth of the market. As a result, over 4,171 drugs have been designated the orphan drug status in the U.S. as of June 2017. In addition, favorable reimbursement offered by the government and private payers for the treatment of rare diseases drives the market in the region.
Europe is a prominent region of the orphan drugs market. According to a Research Office Legislative Council Secretariat report, in 2015, over 30 million people were suffering from rare diseases in the European Union. Incentives offered to promote the manufacture of orphan drugs in the region, such as a10-year period of marketing exclusivity, research grants, and reduced fees for marketing authorization applications, aid in the expansion of the market. Over 1,805 drugs have been granted orphan drug status in the European Union, and over 128 drugs were granted marketing authority from 2000 to 2016.
Players operating in the global market are Aegerion Pharmaceuticals, Inc. Akcea Therapeutics Inc, Ambrx, Inc., Amunix Operating Inc., Bolder Biotechnology, Inc., Theratechnologies Inc., GlaxoSmithKline plc, Novelion Therapeutics Inc., and Sun Pharmaceutical Industries Limited.
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