Gene Therapy for Rare Disease Market Growth Boost by 2026 with Eminent Players Kite Pharma, Inc., Novartis International AG, Juno Therapeutics Inc., Bluebird Bio, Inc., Spark Therapeutics, Inc., uniQure N.V

In recent past, most of the gene therapies received orphan drug designations. Orphan drugs are generally defined as those medicines with one or more indications approved under the Orphan Drug Act of 1983. The Orphan Drug Act supports the development of innovative treatments for rare disease patients. The creation of the orphan drug designation with the passage of the Orphan Drug Act in 1983 has facilitated the development and approval of drugs for rare diseases and 2017 and 2018, were marked by the highest number of orphan drug and indication approvals to date. Production of gene therapies is associated with use of high-end technologies, high research and development costs, and skilled scientists and researchers, which reflects in high prices of these therapies.

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Market Dynamics

Key players in the market are dedicated to the development of gene therapies for the novel indications such as Huntington’s disease for which there are no specific approved medication. For instance, uniQure N.V. is developing AMT-130, a novel Gene therapy for Huntington’s disease (HD), a rare, fatal, neurodegenerative genetic disorder. AMT-130 is currently in pre-clinical trial and is expected to receive approval in near future. Furthermore, companies are developing novel gene therapies for rare diseases such as Hemophilia B and Hemophilia A, which can offer long term treatment and can replace lifelong medication for this diseases. Launches of such novel therapies in near future is expected to significantly create lucrative opportunity for growth of the market. Moreover, increasing reimbursement support as well as innovative payment options are being adopted by manufacturers such as pay for performance. However, high prices of these therapies and potential challenges in commercialization of these therapies in emerging economies such as Asia Pacific and Latin America may restrain growth of the market.

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).

Major Key Players

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.

Increasing drug approvals and launches of novel gene therapies for treatment of various rare diseases are expected to boost the gene therapy for rare disease market growth over the forecast period

In recent past, key players in the market have gained regulatory approvals and have launched their therapies in the market. Frequent approvals of novel gene therapies and their launches in the developed regions are expected to significantly drive gene therapy for rare disease market growth over the forecast period. 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.

Ongoing research and development in gene therapies for rare diseases is expected to boost growth of the global gene therapy for rare disease market

Presence of multiple novel gene therapies for various rare disease treatment in pipeline, which are expected to gain approval is expected to boost the market growth over the forecast period. 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.

Detailed Segmentation:

Global 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
Others

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