Rare Disease Treatment Market Future Scope, Revenue, Top Trends, Growth Opportunity & Regional Outlook 2021-2026

Rare Disease Treatment Market Future Scope, Revenue, Top Trends, Growth Opportunity & Regional Outlook 2021-2026

The rare disease treatment market is poised to accumulate noteworthy gains in coming years owing to the growing prevalence of rare diseases across the world. As per National Institutes of Health, nearly 30 million people in America suffer from one of the 7,000 rare diseases with only 5% of the rare disorders having substantial treatment options. The surging focus on research activities and development of novel therapeutic drugs has further elevated the rare disease treatment market value.

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Favorable government policies and measures for the treatment of rare disorders will also contribute towards market growth in the years to come. These initiatives are driving new drug launches across developed regions, which is expected to positively impact the rare disease treatment market growth. In fact, major diagnostic companies have been actively engaged in expanding their product portfolio recently. Citing an instance, in November 2020, Amylyx Pharmaceuticals, a leading pharmaceuticals company focused towards development of novel treatments for Alzheimer’s disease, ALS, as well as other neurodegenerative diseases, reportedly announced that its drug candidate for treating Wolfram syndrome, AMX0035, was granted orphan drug designation by the U.S. FDA.

AMX0035 is specifically designed to target ER stress, with preclinical data suggesting that it might be a highly promising approach to stop the irreversible optic nerve atrophy progression in Wolfram syndrome patients.

GMI, estimates that the rare disease treatment market would surpass $317 billion by 2026.

With respect to route of administration, in 2019, the oral route of administration segment held a considerable market share and exceeded a valuation of more than $80.2 billion. Oral route of administration is highly preferred for consumption of drug since it is convenient, cost-effective, and non-invasive in comparison to other injectable administration. The segment is also driven by the additional benefits associated with the administration method, such as ease of ingestion, and safety of patients. Consequently, preference for the oral route of administration is rapidly increasing among patients, which would effectively lead to substantial development of novel oral drugs.

On the geographical front, Brazil dominated the overall rare disease treatment market in Latin America and is estimated to expand at a CAGR of more than 12.8% over the projected time frame. This anticipated growth is ascribed to the improving public healthcare infrastructure across the country. Regional government authorities are introducing various measures to focus on treatment of rare diseases. The Brazilian Health Authorities (ANVISA) has set up a standard procedure for the approval of clinical trials for the treatment, diagnosis, as well as prevention of rare diseases. In addition, major key players in the country are actively focusing towards the development of new therapeutic drugs. The afore-mentioned factors are likely to fuel the regional rare diseases treatment market growth in the years to come.

At present, prominent industry players are implementing numerous strategic initiatives such as mergers, partnerships, and collaborations in order to expand their offerings and subsequently strengthen their presence in the market. Citing an instance, in November 2020, Evox Therapeutics Ltd, a renowned exosome therapeutics company, reportedly announced the launch of a new collaboration largely focused on rare diseases with the Oxford-Harrington Rare Disease Centre, University of Oxford rooted within the Pediatrics department.

This collaboration would run over a period of three years and would capitalize on the top-class research environment in Oxford, using exosome therapeutics expertise as well as resources from both the University and Evox to fuel innovation within rare disease drug development. Such unique collaborations would bring in new treatment options and further shape the market in the coming years.

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Eli Lilly and Company, Bayer AG, Sanofi, Actelion Pharmaceuticals Ltd, Novartis AG, Celgene Corporation, AbbVie Inc., Johnson & Johnson Services, Inc., Alexion Pharmaceuticals, Inc., and Pfizer Inc among many others are some of the key players operating in the rare disease treatment market.