Gene Therapy for Rare Disease Market By Drug (Approved Drugs (Tisagenlecleucel (Kymriah), Axicabtagene ciloleucel (Yescarta), Voretigene neparvovec (Luxturna), Strimvelis), Pipeline Drugs (GT-AADC, Fidanacogene elaparvovec (SPK-9011), OTL-200, bb2121, AMT-061, and Others), By Therapeutic Application (Oncology, Neurological Disorders, Ophthalmic Disorders, Hematological Disorders, Immunodeficiency Disorders, Metabolic Disorders, and Others), and Region (North America, Latin America, Europe, Asia Pacific, the Middle East, and Africa) – Size, Share, Outlook, and Opportunity Analysis, 2019– 2027
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Gene therapy is an advanced technique which involves the insertion of the gene in order to treat various chronic diseases such as cancer or gene mutations. Gene therapy can treat genetic defects. According to the National Center for Advancing Translational Sciences, although around 7,000 rare diseases are identified, only a few hundred have approved treatments. Moreover, gene therapy for rare diseases is gaining significant traction, as more than 80% of rare diseases have a known monogenic (single-gene) cause. Drugs which have small molecule only treat the symptom rather than curing the cause of disease which requires continuous administration of the drugs for managing the condition. Additionally, a clinically proven and successful gene therapy require only a single dose to cure disease to treat the disease. Some of the commercially available gene therapies are Luxturna (Spark Therapeutics), Kymriah (Novartis AG), Yescarta (Gilead Sciences, Inc.), and Strimvelis (Orchard Therapeutics).
Rising approval and introduction of advanced gene therapy for the treatment of various chronic disease are projected to propel the market growth of gene therapy for rare disease market over the forecast timeframe
Recently many key players have acquired regulatory approvals and have introduced their therapies in the market. Continuous approvals of novel gene therapies along with the launch of a new therapy in the developed regions are projected to accelerate the gene therapy for rare disease market growth in the coming years. For instance, in December 2017, Spark Therapeutics, Inc. received the U.S. Food and Drug Administration (FDA) approval for its LUXTURNA (voretigene neparvovec-rzyl), a one-time gene therapy product indicated for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy. Furthermore, in August 2018, Novartis International AG received European Commission’s (EC) approval for Kymriah (tisagenlecleucel, formerly CTL019) indicated for treatment of patients up to 25 years of age with relapse or refractory B-cell acute lymphoblastic leukemia (ALL) and for the treatment of adult patients with relapsed or refractory (r/r) diffuse large B-cell lymphoma (DLBCL) after two or more lines of systemic therapy. Moreover, in August 2018, Kite Pharma, Inc., a Gilead Company, received European Commission (EC) Marketing approval for its novel Yescarta (axicabtagene ciloleucel), as a treatment for adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) and primary mediastinal large B-cell lymphoma (PMBCL), after two or more lines of systemic therapy.
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The global gene therapy for rare disease market size was valued at US$ 16.3 million in 2017 and is expected to witness a robust CAGR of 35.0% over the forecast period (2018–2026).
Rising research and development programs in the field of gene therapies for rare diseases are assumed to propel the growth of the global gene therapy for rare disease market
Presence of multiple novel therapies for various rare disease treatment is assumed to gain approval soon is projected to propel the market growth over the forecast timeline. For instance, Spark Therapeutics, Inc. is developing gene therapy namely Fidanacogene Elaparvovec, a novel, investigational gene therapy indicated for the treatment of patients suffering from hemophilia B. Currently, the therapy is under Phase 3 clinical trial and is expected to receive approval in near future. In August 2018, uniQure N.V. treated the first patient in its Phase IIb dose-confirmation study of AMT-061, an investigational AAV5-based gene therapy incorporating the FIX-Padua variant for the treatment of patients with severe and moderately severe hemophilia B. In May 2018, Orchard Therapeutics Plc. received Rare Pediatric Disease Designation from the U.S. Food and Drug Administration (FDA) for its gene therapy candidate OTL-200, indicated for the treatment of patients with metachromatic leukodystrophy (MLD). PTC Therapeutics In. has GT-AADC in phase 3 clinical trials and is indicated for Aromatic L-Amino Acid Decarboxylase (AADC) deficiency.
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On the other hand, lack of awareness regarding the rare disease and their treatment among the population is expected to hamper the market growth of the gene therapy for a rare disease market. For instance, in 2017, uniQure N.V. dropped their plant to pursue the renewal of its gene therapy Glybera, which was approved in 2012, for treatment for a small subset of patients with familial lipoprotein lipase deficiency (LPLD). The company dropped its plan due to very low demand for therapy in the market
Some of the major players operating in the global gene therapy for rare disease market include Kite Pharma, Inc. (Gilead Sciences, Inc.), Novartis International AG, Juno Therapeutics Inc. (Celgene Corporation), Bluebird Bio, Inc., Spark Therapeutics, Inc., uniQure N.V, Orchard Therapeutics Plc., PTC Therapeutics, Inc., and BioMarin Pharmaceutical Inc.
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