Rising Number of Pipeline Products and Clinical Trials Drive the Demand for Leber Congenital Amaurosis Market

As clinical trial activity rises to new levels, the field appears poised to make rapid and important advances in IRD research and patient care. The continuous innovation for the treatment of inherited retinal diseases and Leber congenital amaurosis disorder can positively impact the market as the size of the market will increase, when these products are approved in the global market. Due to no pharmacological treatments for the LCA the major market players are continuously innovating the treatment or investing in the research and development for the treatment. The companies operating in this market continuously recruit clinical trials, which will aid the global leber congenital amaurosis market gain traction.

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Data Bridge Market Research analyses that the leber congenital amaurosis market is expected to grow at a CAGR of 4.8% in the forecast period of 2022 to 2029 and is expected to reach USD 89.84 million by 2029.

Leber Congenital Amaurosis Market

Increase In Incidence and Prevalence OF RPE65 Gene-Mediated in Retinal Diseases Is Expected To Drive the Market's Growth Rate

Inherited retinal dystrophies (IRDs) represent a genetically diverse group of progressive, visually debilitating diseases. Adult and pediatric patients with vision loss due to IRD caused by biallelic mutations in the 65-kDa retinal pigment epithelium (RPE65) gene are often clinically diagnosed as retinitis pigmentosa (RP), and Leber congenital amaurosis (LCA). The high prevalence of leber congenital amaurosis further neccistates proper treatment and therapy aid, which further boosts the market expansion. For instance, the prevalence of leber congenital amaurosis (LCA) has been estimated to be 1-2/100,000 births. This disorder affects males and females in equal numbers. Consequently, the high prevalence and incidence of leber congenital amaurosis further accelerates the market growth.

Report Scope and Market Segmentation

Report Metric

Details

Forecast Period

2022 to 2029

Base Year

2021

Historic Years

2020(Customizable to 2014 - 2019)

Quantitative Units

Revenue in USD Million, Volumes in Units, Pricing in USD

Segments Covered

By Disease Type  (Infantile Type, Juvenile Type And Other), Type(Therapy, Diagnosis), End User (Hospitals, Specialty Clinics, Ambulatory Surgical Centers, Home Healthcare And Others), Distribution Channel (Direct Tender And Retail Sales)

Countries Covered

U.S., Canada and Mexico in North America, Germany, France, U.K., Netherlands, Switzerland, Belgium, Russia, Italy, Spain, Turkey, Rest of Europe in Europe, China, Japan, India, South Korea, Singapore, Malaysia, Australia, Thailand, Indonesia, Philippines, Rest of Asia-Pacific (APAC) in the Asia-Pacific (APAC), Saudi Arabia, U.A.E, South Africa, Egypt, Israel, Rest of Middle East and Africa (MEA) as a part of Middle East and Africa (MEA), Brazil, Argentina and Rest of South America as part of South America.

Market Players Covered

Invitae Corporation (U.S.), Spark Therapeutics, Inc. (U.S.), LKC Technologies (U.S.), CENTOGENE N.V. (Austria), Optos (U.K.), Metrovision (U.S.), Blue Print Genetics Oy (Finland ), CD Genomics (U.S.), ProQR Therapeutics (Netherlands), OCUGEN, INC. (U.S.),  MeiraGTx Limited(U.S.),  Coave Therapeutics (U.K.) , IVERIC bio, Inc. (U.S.), Atsena Therapeutics(U.S.), Editas Medicine(U.S.), Eluminex Biosciences (China), Okuvision (German), Astellas Pharma Inc. (Japan), Kubota Pharmaceutical Holdings Co. Ltd. (U.S.), REGENXBIO Inc. (U.S.) among others

Data Points Covered in Report

In addition to the market insights such as market value, growth rate, market segments, geographical coverage, market players, and market scenario, the market report curated by the Data Bridge Market Research team also includes in-depth expert analysis, patient epidemiology, pipeline analysis, pricing analysis, and regulatory framework.

Segment Analysis:

The global leber congenital amaurosis market is categorized into two notable segments based on disease type, type, end user, and distribution channel.

  • Based on disease type, the global leber congenital amaurosis market is segmented into infantile type, juvenile type and others. In 2022, infantile type segment is expected to dominate the leber congenital amaurosis market, growing with the CAGR of 4.9% due to the most common cause of inherited blindness in childhood and is found in two to three out of every 100,000 babies. Moreover, children have a 25%chance of inheriting the two LCA genes (one from each parent) needed to cause the disorder.
  • Based on by type, the global leber congenital amaurosis market is segmented into diagnosis and therapy. In 2022, by therapy segment is expected to dominate the global leber congenital amaurosis market with the CAGR of 5.2%

The therapy segment is expected to dominate the global leber congenital amaurosis market

In 2022, by therapy segment is expected to dominate the global leber congenital amaurosis market with the CAGR of 5.2% in the forecast period of 2022 to 2029. The diagnosis segment is expected to dominate the global leber congenital amaurosis market due to the increasing prevalence of retinal diseases and strategic initiatives by market players.

  • Based on end user, the global leber congenital amaurosis market is segmented into hospitals, specialty clinics, ambulatory surgical centers, home healthcare and others. In 2022, the hospitals segment is expected to dominate the global leber congenital amaurosis market, growing with the highest CAGR of 7.2%

The hospitals segment is expected to dominate the global leber congenital amaurosis market

In 2022, the hospitals segment is expected to dominate the global leber congenital amaurosis market, growing with the highest CAGR of 7.2% in the forecast period of 2022 to 2029. The hospitals segment is expected to dominate the global leber congenital amaurosis market due to providing effective care facilities and treatment to a patient suffering from inherited retinal diseases.

Based on distribution channel, the global leber congenital amaurosis market is segmented into hospital pharmacies, retail pharmacies and others. In 2022, the hospital pharmacies segment is expected to dominate the global leber congenital amaurosis market, growing with the CAGR of 5.2% in the forecast period of 2022 to 2029 due to rising preference among customers due to guaranteed payment and high international sales of the product.

Major Players

Data Bridge Market Research recognizes the following companies as the major leber congenital amaurosis market players in leber congenital amaurosis market are Invitae Corporation (U.S.), Spark Therapeutics, Inc. (U.S.), LKC Technologies (U.S.), CENTOGENE N.V. (Austria), Optos (U.K.), Metrovision (U.S.), Blue Print Genetics Oy (Finland ), CD Genomics (U.S.), ProQR Therapeutics (Netherlands), OCUGEN, INC. (U.S.),  MeiraGTx Limited(U.S.),  Coave Therapeutics (U.K.) , IVERIC bio, Inc. (U.S.), Atsena Therapeutics(U.S.), Editas Medicine(U.S.), Eluminex Biosciences (China), Okuvision (German), Astellas Pharma Inc. (Japan), Kubota Pharmaceutical Holdings Co. Ltd. (U.S.), REGENXBIO Inc. (U.S.) among others.

Leber Congenital Amaurosis Market

Market Development

  • In September 2021, Gyroscope Therapeutics Limited clinical-stage gene Therapy Company focused on diseases of the eye, announced positive interim data from the ongoing open-label Phase I/II clinical trial of its investigational one-time gene therapy, GT005, in people with geographic atrophy (GA) secondary to age-related macular degeneration (AMD).
  • In January 2021, ViGeneron GmbH, a gene therapy company, announced a global collaboration and licensing agreement with Biogen Inc. to develop and commercialize gene therapy products based on adeno-associated virus (AAV) vectors to treat inherited eye diseases. The companies will use ViGeneron's proprietary vgAAV, novel engineered AAV capsids, to efficiently transduce retinal cells via intravitreal injections
  • In 2020, a landmark decision was made by the Therapeutics Goods Administration in which the first-ever therapy treatment of inherited retinal disease was approved a very short notice. This approval by the Australian government will change the life of the 15,000 affected population. This Gene therapy using Luxturna is also used for children with Leber's amaurosis associated with the RPE65 gene.
  • In January 2021, the Italian Medicines Agency (AIFA) has given the go-ahead to reimburse Novartis' ophthalmic gene therapy Luxturna (voretigene abeparvovec) for treating hereditary dystrophy in pediatric and adult patients with vision loss due to confirmed biallelic mutation of the RPE56 gene who have sufficient, viable retinal cells. As an innovative medicine, Luxturna will be paid for through Italy's special fund for non-oncology medicines whose reserve consists of USD 750 million.

Regional Analysis

Geographically, the countries covered in the leber congenital amaurosis market report are U.S., Canada and Mexico in North America, Germany, France, U.K., Netherlands, Switzerland, Belgium, Russia, Italy, Spain, Turkey, Rest of Europe in Europe, China, Japan, India, South Korea, Singapore, Malaysia, Australia, Thailand, Indonesia, Philippines, Rest of Asia-Pacific (APAC) in the Asia-Pacific (APAC), Saudi Arabia, U.A.E, South Africa, Egypt, Israel, Rest of Middle East and Africa (MEA) as a part of Middle East and Africa (MEA), Brazil, Argentina and Rest of South America as part of South America.

As per Data Bridge Market Research analysis:

North America is the dominant region in leber congenital amaurosis market during the forecast period

North America dominated the leber congenital amaurosis market owing to the due to the increase in technological advancement within the U.S.  North America will continue to dominate the leber congenital amaurosis market in terms of market share and market revenue and will continue to flourish its dominance during the forecast period. This is due to the growing adoption of advanced technology in this region. 

Asia-Pacific is estimated to be the fastest growing region in leber congenital amaurosis market

Asia-Pacific is expected to grow during the forecast period due to the rapid development of medical facilities in emerging economies in this region. In addition to this, rising healthcare expenditure and increasing per-capita income are expected to propel the market's growth rate in this region.

COVID-19 Impact Analysis

The outbreak of COVID-19 significantly impacted the healthcare industry. The leber congenital amaurosis market was, however adversely affected by it.  The imposition of the lockdown and social distancing restrictions by the government to curb the COVID-19 pandemic outbreak, led to emergence of various challenges such as the denied or postponed eye care services and others which limited the industry's expansion. The leber congenital amaurosis market was adversely affected by COVID-19 outbreak. As the admission of COVID-19 patients in hospitals increased, numerous elective procedures were canceled or postponed to reserve hospital beds and patient care staff to COVID-19 patient care.

On the brighter side, there has been a decline in COVID-19 patients worldwide, which will result in increased eye care service treatment. Moreover, the restrictions and measures are likely to relax, which will help market to witness a slight increment, as the manufacturers are focusing on various developments and innovations, market trends, and other expansion strategies.

The U.S. Food and Drug Administration approved a novel gene therapy to treat patients suffering from a rare form of inherited vision loss

The FDA approved Luxturna (voretigene neparvovec-rzyl), a new gene therapy, to treat children and adults with an inherited form of vision loss that can lead to blindness. Luxturna is the first directly administered gene therapy approved in the United States to treat a disease caused by a specific gene mutation. Luxturna is approved to treat patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy, which causes vision loss and, in some cases, complete blindness.

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