Summary

The rare genetic disorders treatment market is expected to grow at a strong CAGR of around 11.9% during the forecast period. The increase in research and development spending by major key pharmaceutical players in the market is another factor influencing the growth of the market. Because public awareness and comprehension of rare diseases have grown, a number of significant clinical-stage biopharmaceutical startups and established market players now have orphan drug pipeline candidates in various phases of clinical trials. For instance, in 2021, Biogen acquired TMS-007, a preclinical gene therapy for Friedreich's ataxia, a rare genetic disease that affects the nervous system. Biogen also announced plans to acquire GSK's rare disease unit for USD 5.1 billion, which includes several drugs for rare genetic diseases such as Hunter syndrome and Morquio syndrome.

Some of the major players operating in the market include F. Hoffmann-La Roche Ltd.; Pfizer, Inc.; AstraZeneca; Bristol Myers Squibb; Sanofi; Novartis AG; AbbVie Inc.; GlaxoSmithKline Plc; Amgen Inc.; and Johnson & Johnson Inc. Several M&As along with partnerships have been undertaken by these players to facilitate customers with hi-tech and innovative products/technologies.

Insights Presented in the Report
“Amongst application, the neurological disorders segment to grow with high CAGR during the forecast period”
Based on application, the market is segmented into cancer, neurological disorders, cardiovascular diseases, metabolic disorders, hematology diseases, and others. The neurological disorders segment is expected to grow with high CAGR during the forecast period. Neurological diseases are a common type of rare genetic disorder, with an estimated prevalence of 350 million people worldwide. This high prevalence has driven research and development efforts in this area. Rare neurological diseases are caused by genetic mutations that affect the brain and nervous system. Some examples include Huntington's disease, spinal muscular atrophy, and Niemann-Pick disease. For instance, according to the National Institutes of Health (NIH), Spinal Muscular Atrophy (SMA) is a rare genetic disease that affects the nervous system, causing muscle weakness and atrophy. The rising number of product approvals for rare genetic disorders is driving the growth of the segment. For instance, Evrysdi is a small molecule drug that was approved by the FDA in 2020 for the treatment of SMA.

“Amongst drug type, the non-biologics category to grow with high CAGR during the forecast period”
By drug type, the market is bifurcated into biologics & biosimilars, and non-biologics. The non-biologics category is anticipated to grow with high CAGR during the forecast period. Non-biologics therapy for rare genetic disorders is a growing area of interest in the field of medicine. Unlike biologics, which are made from living cells or organisms, non-biologics are chemical compounds that are synthesized in a laboratory. They can be small molecules, peptides, or nucleic acid-based therapies, and they are often more stable and easier to produce than biologics. The market for non-biologics therapy for rare genetic disorders is expected to grow significantly in the coming years, as more therapies are developed and approved for use. For instance, in December 2020, the US FDA approved Oxlumo (lumasiran) for the treatment of primary hyperoxaluria type 1 (PH1), a rare genetic disorder that can cause kidney damage and failure. Another, in November 2020, the US FDA approved lumasiran for the treatment of primary hyperoxaluria type 1 (PH1), a rare genetic disorder that can cause kidney damage and failure.

“Amongst distribution channel, the hospital pharmacy category held a significant share of the market in 2021”
By distribution channel, the market is segmented into hospital pharmacy, online pharmacy, and retail pharmacy. The hospital pharmacy held a significant share of the market in 2021. Hospital pharmacy play a critical role in ensuring that patients have access to these treatments and that they are used safely and effectively. Hospital pharmacists are responsible for ensuring that medications are prescribed and dispensed safely and effectively, and that patients are educated about how to take their medications correctly. Moreover, these are also involved in clinical research and development. Many hospitals participate in clinical trials to test new orphan drugs and to gather data on the safety and efficacy of these treatments.

“North America held dominant share of the market in 2021”
In 2021, North America held a dominant share of the market. The growing demand for rare genetic disorders treatments can be attributed due to the surge in the prevalence of rare cancers and other diseases. According to an article shared by the American Cancer Society, in the US in January 2022, there are approximately 54,000 new cases of oropharyngeal or oral cancer each year. Oropharyngeal or oral cancer claims the lives of 11,230 people. Moreover, rising healthcare expenditure is also acting as a major driver for the growth of the market in the region.

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• The study includes market sizing and forecasting analysis validated by authenticated key industry experts.
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• Detailed examination of drivers, restraints, key trends, and opportunities prevailing in the industry.
• The study comprehensively covers the market across different segments.
• Deep dive regional level analysis of the industry.

Customization Options:
The global rare genetic disorders treatment market can further be customized as per the requirement or any other market segment. Besides this, UMI understands that you may have your own business needs, hence feel free to connect with us to get a report that completely suits your requirements.

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