Global Lysosomal Storage Disorder Drugs Market – Industry Trends and Forecast to 2030

Análisis y tamaño del mercado de medicamentos para trastornos de almacenamiento lisosomal

Los trastornos de depósito lisosomal son trastornos metabólicos hereditarios que se caracterizan por una acumulación anormal de numerosos productos químicos nocivos en las células del cuerpo debido a la falta de enzimas. Existen aproximadamente 50 de estas enfermedades y pueden afectar el esqueleto, el cerebro, la piel, el corazón y el sistema nervioso central, entre otros componentes corporales. Aún se están descubriendo más trastornos de depósito lisosomal. Si bien se están realizando ensayos clínicos para posibles tratamientos para algunos de estos trastornos, muchas enfermedades de depósito lisosomal carecen de un medicamento aprobado.                           

Data Bridge Market Research analiza que los medicamentos para el trastorno de almacenamiento lisosomal, que fueron de USD 9738,63 millones en 2022, se dispararían hasta USD 21646,92 millones para 2030 y se espera que experimenten una CAGR del 10,50% durante el período de pronóstico. La "terapia de reemplazo enzimático" domina el segmento de tratamiento de los medicamentos para el trastorno de almacenamiento lisosomal debido a la creciente prevalencia de la enfermedad lisosomal. Además de los conocimientos sobre escenarios de mercado como el valor de mercado, la tasa de crecimiento, la segmentación, la cobertura geográfica y los principales actores, los informes de mercado seleccionados por Data Bridge Market Research también incluyen análisis profundo de expertos, epidemiología del paciente, análisis de la cartera, análisis de precios y marco regulatorio.

Alcance y segmentación del mercado de medicamentos para trastornos de almacenamiento lisosómico

Definición de mercado

La enfermedad de depósito lisosomal o enfermedad por almacenamiento lisosomal es una enfermedad metabólica hereditaria poco frecuente. Es causada por un metabolismo defectuoso o mutante, que resulta en la falta o deficiencia de enzimas, lo que resulta en una acumulación anormal de material en varias células del cuerpo. Si se realiza antes de los dos años, el trasplante de médula ósea (BMT) previene con éxito el retraso mental progresivo en niños con MPS IH (enfermedad de Hurler). La terapia de reemplazo enzimático (ERT) es exitosa para aquellos con enfermedad de Gaucher tipo I. La anemia y los recuentos bajos de plaquetas han mejorado, el agrandamiento del hígado y el bazo ha disminuido significativamente y las anomalías esqueléticas han mejorado. Las personas con enfermedad de Gaucher tipos II y III que reciben ERT también ven mejoras en estos signos sistémicos.

Dinámica del mercado de medicamentos para trastornos de almacenamiento lisosomal

Conductores

• Prevalencia y concientización:

El creciente reconocimiento y diagnóstico de los trastornos de depósito lisosomal en todo el mundo ha impulsado la demanda de opciones de tratamiento eficaces. A medida que aumenta la conciencia sobre estos trastornos genéticos poco frecuentes, se identifican más pacientes, lo que genera una mayor necesidad de desarrollar medicamentos .

• Necesidad médica insatisfecha

Los trastornos de almacenamiento lisosomal son un grupo de enfermedades genéticas raras que se caracterizan por la deficiencia de enzimas específicas dentro de los lisosomas, lo que da como resultado la acumulación de materiales no digeridos en las células. Estos trastornos a menudo provocan síntomas graves y progresivos y pueden poner en peligro la vida. Debido a las limitadas opciones de tratamiento disponibles, existe una importante necesidad médica no satisfecha de terapias efectivas, lo que impulsa el desarrollo de medicamentos en este campo .

• Incentivos para medicamentos huérfanos

Los gobiernos y las agencias regulatorias de todo el mundo han implementado diversos incentivos para promover el desarrollo de medicamentos para enfermedades raras, incluidos los trastornos de almacenamiento lisosomal. Estos incentivos pueden incluir exclusividad de mercado extendida, créditos fiscales, subvenciones de investigación y vías regulatorias aceleradas. Dichos incentivos alientan a las compañías farmacéuticas a invertir en esfuerzos de investigación y desarrollo para estas enfermedades raras .

• Avances en biotecnología

The advancements in biotechnology and drug discovery techniques have significantly contributed to developing novel therapies for lysosomal storage disorders. Innovative approaches such as enzyme replacement therapies, substrate reduction therapies, gene therapies, and small molecule inhibitors have shown promising results in preclinical and clinical studies, fueling further investment in this field.

Opportunities

• Large Addressable Market

While individual LSDs are rare, there are over 50 known lysosomal storage disorders, each with unique genetic defects and clinical manifestations. Collectively, these disorders affect a substantial number of patients worldwide. The combined prevalence of LSDs makes for a sizable addressable market, presenting an opportunity for drug developers and manufacturers.

• Limited Treatment Options

Historically, treatment options for LSDs were limited, primarily focusing on symptomatic management. However, recent advancements in understanding the underlying biology of these disorders have led to the development of innovative therapies. The availability of disease-modifying treatments, such as enzyme replacement therapies, substrate reduction therapies, gene therapies, and small molecule inhibitors, has expanded the treatment landscape, providing better options for patients and driving market growth.

Restraints/Challenges

• Limited Patient Population

Lysosomal storage disorders are rare genetic diseases, and each specific disorder affects a relatively small number of patients. The limited patient population poses challenges for drug developers and manufacturers in achieving economies of scale and generating substantial revenue. The small market size may also deter some companies from investing in the research and development of LSD drugs.

• High Development Cost

Developing drugs for rare diseases, including LSDs, can be expensive and time-consuming. The cost of conducting clinical trials, obtaining regulatory approvals, and ensuring safety and efficacy can be substantial. The relatively small patient population and the complex nature of LSDs may result in higher per-patient costs, making the development of LSD drugs financially challenging for some companies.

This   Hospice  Market report provides details of new recent developments, trade regulations, import-export analysis, production analysis, value chain optimization, market share, the impact of domestic and localized market players, analyses opportunities in terms of emerging revenue pockets, changes in market regulations, strategic market growth analysis, market size, category market growths, application niches and dominance, product approvals, product launches, geographic expansions, technological innovations in the market. To gain more info on the Hospice  Market Contact Data Bridge Market Research for an Analyst Brief, our team will help you take an informed market decision to achieve market growth.

Recent Development

• In January 2021, M6P Therapeutics, a privately held life sciences company developing next-generation recombinant enzyme and gene therapies for lysosomal storage disorders (LSDs), announced that the U.S. Food and Drug Administration (FDA) granted six rare pediatric disease designations (RPDDs) for various programs within its development pipeline for LSDs, including four recombinant enzyme and two gene therapy programs
• In July 2019, Takeda Pharmaceutical Company launched an enzyme replacement therapy portfolio for lysosomal storage disorders (LSD) in India, expanding its range of rare disease therapies nationwide

Global Lysosomal Storage Disorder Drugs Scope

The lysosomal storage disorder drugs are segmented on the basis of treatment, indication, and end User. The growth amongst these segments will help you analyze meagre growth segments in the industries and provide the users with a valuable market overview and market insights to help them make strategic decisions for identifying core market applications.

Treatment

• Enzyme Replacement Therapy
• Stem Cell Therapy
•  Substrate Reduction Therapy
•  Others

Indication  

• Gaucher's Disease
• Fabry Disease
•  Pompe’s Syndrome
•  Mucopolysaccharidosis,
• Others

End user

• Hospitals,
• Clinics
• Others

Lysosomal Storage Disorder Drugs Regional Analysis/Insights

The lysosomal storage disorder drugs is analysed, and market size insights and trends are provided by country, treatment, indication, and end user as referenced above.

The countries covered in the lysosomal storage disorder drugs report are U.S., Canada and Mexico, Germany, France, U.K., Netherlands, Switzerland, Belgium, Russia, Italy, Spain, Turkey, Rest of Europe, China, Japan, India, South Korea, Singapore, Malaysia, Australia, Thailand, Indonesia, Philippines, Rest of Asia-Pacific , Saudi Arabia, U.A.E, South Africa, Egypt, Israel, Rest of Middle East and Africa, Brazil, Argentina and Rest of South America

North America dominates the lysosomal storage disorder drugs because of the strong base of healthcare facilities, the strong presence of major players in the market, the rising prevalence of lysosomal disease, and the rising number of research activities in this region.   

Asia-Pacific is expected to witness significant growth during the forecast period of 2023 to 2030 due to the increase in government initiatives to promote awareness, rise in medical tourism, growing research activities in the region, availability of massive untapped markets, large population pool, and the growing demand for quality healthcare in the region.

The country section of the report also provides individual market-impacting factors and changes in regulation in the market domestically that impact the current and future trends of the market. Data points like down-stream and upstream value chain analysis, technical trends and porter's five forces analysis, case studies are some of the pointers used to forecast the market scenario for individual countries. Also, the presence and availability of global brands and their challenges faced due to large or scarce competition from local and domestic brands, impact of domestic tariffs and trade routes are considered while providing forecast analysis of the country data.   

Healthcare Infrastructure Growth Installed base and New Technology Penetration

The   lysosomal storage disorder drugs  also provides you with detailed market analysis for every country growth in healthcare expenditure for capital equipment, installed base of different kind of products for   lysosomal storage disorder drugs , impact of technology using life line curves and changes in healthcare regulatory scenarios and their impact on the   lysosomal storage disorder drugs . The data is available for historic period 2010-2020.

Competitive Landscape and lysosomal storage disorder drugs Share Analysis

The   lysosomal storage disorder drugs  competitive landscape provides details by competitor. Details included are company overview, company financials, revenue generated, market potential, investment in research and development, new market initiatives, global presence, production sites and facilities, production capacities, company strengths and weaknesses, product launch, product width and breadth, application dominance. The above data points provided are only related to the companies' focus related to   lysosomal storage disorder drugs .

Some of the major players operating in the   lysosomal storage disorder drugs  are:

• Novartis AG (Switzerland)
• Johnson & Johnson Private Limited (U.S.)
• Teva Pharmaceutical Industries Ltd. (Ireland)
• Merck & Co., Inc. (U.S.)
• Allergan (Ireland)
• Pfizer Inc. (U.S.)
• GlaxoSmithKline plc (U.K.)
• Sanofi (France)
• Merck KGaA (Germany)
• Abbott (U.S.)
• Boehringer Ingelheim International GmbH. (Germany)
• Takeda Pharmaceutical Company Limited (Japan)
• Amicus Therapeutics, Inc. (U.S.)
• Moderna, Inc. (U.S.)
• Greenovation Biotech GmbH (Germany)
• Biomarin (U.S.)
• JCR Pharmaceuticals Co., Ltd (Japan)
• ISU ABXIS (South Korea)
• Idorsia Pharmaceuticals Ltd (Switzerland)
• AVROBIO, Inc. (U.S.)
• Resverlogix Corp. (Canada)
• Enzyvant (U.S.)
• CHIESI Farmaceutici SpA (Italy) 

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