CNS Specific Antisense Oligonucleotide Market

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2. CNS SPECIFIC ANTISENSE OLIGONUCLEOTIDE MARKET ANALYSIS • CNS Specific Antisense Oligonucleotide Market - by Drug (Approved and Pipeline), by Indication (Hereditary transthyretin amyloidosis (hATTR)/ Polyneuropathy, Spinal Muscular Atrophy, and  Huntington’s Disease), by Distribution Channel (Hospital Pharmacy, Retail Pharmacy, and Online Pharmacy), and by Region (North America, Latin America, Europe, Asia Pacific, Middle East, and Africa) - Size, Share, Outlook, and Opportunity Analysis, 2018 – 2026

3. Global CNS Specific Antisense Oligonucleotide Market – Breakthrough Treatment for CNS Disorders • Antisense oligonucleotide plays a major role in modern healthcare, as it acts on the messenger RNA level before the protein is formed to inhibit expression of certain diseases-causing genes. • The global CNS specific antisense oligonucleotide market size was valued at US$ 883.7 million in 2017, and is expected to exhibit a CAGR of 24.9% over the forecast period (2018 – 2026).

4. Increasing prevalence of cancer is expected to propel growth of the CNS specific antisense oligonucleotide market • CNS specific antisense oligonucleotide is used for treatment of neurodegenerative disorders and associated rare diseases. According to February 2017, report of National Center for Biotechnology Information (NCBI), an estimated the prevalence of Cerebral palsy ranges from 1.5 to more than 4 per 1,000 of children from age 10 to 14 years. The birth prevalence of Cerebral palsy is around 2 per 1,000 live births globally. • Furthermore, launch of new products for rare disease associated with CNS disorders is propelling growth of the CNS specific antisense oligonucleotide market over the forecast period. For instance, in October 2018, Ionis Pharmaceuticals, Inc. and Akcea Therapeutics, Inc. received the U.S.  Food and Drug Administration (FDA) approval for its novel drug TEGSEDITM (inotersen) indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults. In the same year, company received approval from Canadian Health authority for the novel drug TEGSEDITM (inotersen). • However, high cost of treatment and risk of toxicity is expected to hinder the market growth. According to the Journal Molecular Therapy, in May 2017, Biogen priced Spinraza (nusinersen injection) at US$ 750,000 for the first year’s treatment (US$125,000 per injection) and US$350,000 per year.

5. Government support for development and launch of antisense drugs in North America is expected to bolster the market growth • North America holds the dominant position in the global CNS specific antisense oligonucleotide market, owing to strong government support for antisense drugs. For instance, in March 2018, GeneTxBiotherapeutics LLC’s, GTX-101 received the U.S. Food and Drug Administration’s (FDA) orphan-drug designation for GTX-101 for the treatment of Angelman syndrome. It is a rare neurogenetic disorder that affects around one in 15,000 people, according to the FDA statement. • Increasing prevalence of Alzheimer disease in the region is also expected to boost the market growth. For instance, in August 2012, National Center for Biotechnology Information (NCBI) stated Alzheimer disease as the most frequent cause of dementia in Western societies and estimated around 5.5 million cases in the U.S. and the prevalence worldwide is estimated to be as high as 24 million. • Moreover, rich product pipeline for treatment of neurodegenerative disorders is expected to drive the market growth. For instance, in October 2017, Ionis Pharmaceuticals, Inc., initiated a Phase 1/2 clinical study of IONIS-MAPTRx in patients with mild Alzheimer's disease. Biogen funded US$ 10 million milestone payment to Ionis Pharmaceuticals, Inc. • Moreover, key players in the players are focused on receiving the U.S. Food and Drug Administration (FDA) approval for novel products to reduce the burden of CNS associated disorders. For instance, in September 2016, Sarepta Therapeutics received the U.S. Food and Drug Administration (FDA) approval for Exondys 51(eteplirsen) used for treatment of rare genetic disorder Duchenne muscular dystrophy.

6. Key Players • Major players operating in the global CNS specific antisense oligonucleotide market include Alnylam Pharmaceuticals Inc., Sarepta Therapeutics Inc., Biogen Inc., Ionis Pharmaceuticals Inc., Wave Life Sciences Ltd., Stroke Therapeutic Inc., Dynacure, ProQR Therapeutics N.V., and Q-STATE BIOSCIENCES, INC.

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