뮤코다당증 세계 시장은 2024년에 23억 1,308만 달러에 달했고, 2033년에는 43억 8,985만 달러에 달할 것으로 예측되며, 예측 기간인 2025-2033년 7.5%의 연평균 복합 성장률(CAGR)을 보일 것으로 예상됩니다. 뮤코다당증는 글리코사미노글리칸의 분해를 촉매하는 효소의 유전자 돌연변이로 인해 발생하는 유전성 리소좀 저장 질환군입니다. 글리코사미노글리칸은 점액 다당류로도 알려져 있으며, 세포 신호 전달, 뼈, 결합 조직, 각막의 성장과 발달에 중요한 역할을 하는 탄수화물 분자입니다. 이러한 글리코사미노글리칸은 11가지 효소에 의해 분해되는데, 이 효소가 돌연변이를 일으키면 그 기능이 부적절해지거나 상실될 수 있습니다. 그 결과, 글리코사미노글리칸은 혈액, 뇌, 척수, 결합조직에 축적되어 신체적 형태 이상에서 인지 장애에 이르기까지 다양한 증상을 유발합니다.
성장 촉진요인과 억제요인
신제품 승인 및 개발 활동 증가
뮤코다당증는 미충족 수요가 높은 질환군입니다. 현재 연구 개발은 질병 변형 치료제를 개발하여 시장에 출시하는 데 초점을 맞추었습니다. 최근 전 세계 규제 당국으로부터 여러 치료제가 시판 허가를 받으면서 뮤코다당증 치료 환경이 개선되고 있습니다. 또한, 일부 파이프라인 의약품은 임상 마일스톤 및 특별 지정을 받아 승인 절차를 가속화하고 시장 진입을 앞당길 수 있는 기회를 얻었습니다. 예측 기간 동안 산필리포 증후군과 같은 미충족 수요가 높은 뮤코다당증 치료제들이 시장에 진입할 것으로 예상됩니다.
예를 들어, 2025년 2월 5일, Ultragenyx Pharmaceutical Inc.는 산필리포 증후군 A형(MPS IIIA) 환자를 치료하기 위한 새로운 AAV 기반 유전자 치료제 UX111의 양호한 임상시험 데이터를 보고했습니다. 임상시험 결과, UX111은 치료받은 환자의 인지 능력과 수용적, 표현적 의사소통을 유의미하게 개선하는 것으로 나타났습니다. 2025년 2월 18일, 울트라제닉스는 미국 FDA가 UX111의 생물학적 제제 승인 신청서(BLA)를 접수하고 우선 심사 대상으로 검토 중이라고 발표했습니다. 울트라제닉스는 조기 승인을 요청하고 있으며, 2025년 8월 18일까지 결정될 예정입니다. 울트라제닉스가 승인을 받으면 이 획기적인 치료제를 상업적으로 출시할 수 있는 준비를 갖추게 됩니다.
이러한 파이프라인의 의약품은 임상시험에서 좋은 결과를 얻었으며, 규제 당국의 승인을 앞두고 있는 의약품이 몇 가지 있습니다. 이들 의약품이 승인되면 뮤코다당증의 치료 상황에 혁명을 가져올 것으로 기대되고 있습니다.
다음은 뮤코다당증 유형별로 다양한 임상 단계에 있는 주요 파이프라인 의약품의 목록입니다.
승인된 약품의 높은 비용이 시장 성장을 억제할 가능성.
효소대체요법(ERT)은 현재 MPS의 표준 치료법이지만, 환자는 장기간의 치료가 필요합니다. 이는 적절한 보험에 가입되어 있지 않고 보험사의 엄격한 규정을 충족하지 못하는 환자에게는 경제적으로 큰 부담이 될 수 있습니다. 또한, 예측 기간 동안 몇 가지 질병 변형 치료법이 시장에 진입할 것으로 예상됩니다. 이러한 치료법은 현재 표준 치료인 ERT에 비해 가격이 높을 것으로 예상됩니다. 따라서 미충족 수요는 높은 편이며, 이는 채택률을 저해할 수 있습니다.
The global Mucopolysaccharidoses market reached US$ 2,313.08 million in 2024 and is expected to reach US$ 4,389.85 million by 2033, growing at a CAGR of 7.5% during the forecast period 2025-2033. Mucopolysaccharidoses are a group of inherited lysosomal storage diseases caused by genetic mutations in the enzymes that catalyze glycosaminoglycans breakdown. Glycosaminoglycans, also known as mucopolysaccharides, are the carbohydrate molecules that play a crucial role in cell signaling, growth & development of bones, connective tissues, and corneas. These glycosaminoglycans are broken down by 11 different types of enzymes, whose mutation leads to improper or loss of their function. As a result, the glycosaminoglycans accumulate in the blood, brain, spinal cord, and connective tissue, leading to a variety of symptoms ranging from physical dysmorphia to cognitive impairment.
Market Dynamics: Drivers & Restraints
Rising Novel Product Approvals and Development Activities
Mucopolysaccharidoses are a group of conditions with high unmet needs. The current research efforts were focused on developing disease-modifying therapies and subsequently launching them into the market. In recent times, the mucopolysaccharidosis treatment landscape has seen traction due to the marketing authorization of several therapies provided by regulatory bodies across the world. Moreover, several pipeline drugs have received clinical milestones and special designations that can expedite their approval process and gain early market access. In the forecast period, several pipeline products are expected to make market entry for some mucopolysaccharidose conditions with high unmet needs, such as Sanfilippo syndrome.
For instance, on February 5, 2025, Ultragenyx Pharmaceutical Inc. reported positive clinical trial data for its novel AAV-based gene therapy UX111, which is aimed at the treatment of patients with Sanfilippo syndrome type A (MPS IIIA). The clinical results showed that UX111 has significantly improved cognitive ability and receptive & expressive communication in treated patients. This is a significant milestone in the Sanfilippo treatment landscape. On February 18, 2025, Ultragenyx announced that the U.S. FDA accepted the Biologics License Application (BLA) of UX111, and it is being considered for priority review. Ultragenyx is seeking accelerated approval, and the decision is expected by August 18, 2025. Once Ultragenyx receives a positive nod, the company will be ready to launch this revolutionary therapy commercially into the market.
Several such pipeline drugs are performing well in clinical trials and are on the verge of regulatory approval. Once these drugs are approved, they are expected to revolutionize the mucopolysaccharidoses treatment landscape.
Below is the representation of key pipeline drugs that are in various clinical phases for different types of mucopolysaccharidoses.
High- Cost of Approved Drugs May Restrain the Market Growth.
Enzyme replacement therapies (ERTs) are the current standard of treatment for MPS, however, patients need to be on long-term therapy. This can be a significant economic burden to the patient who does not have proper insurance coverage and does not meet the stringent rules put forth by the insurance companies. Moreover, several disease-modifying therapies are expected to make market entry in the forecast period. These therapies are expected to have higher prices as compared to the current standard of care ERTs. This can hinder the adoption rate, although the unmet needs are high.
The global Mucopolysaccharidoses market is segmented based on type, drug, and region.
Elosulfase Alfa in the drug segment is dominating the market.
Elosulfase Alfa, sold under the brand name VIMIZIM, manufactured by BioMarin Pharmaceutical Inc. is an enzyme replacement therapy indicated for the treatment of Mucopolysaccharidosis type IVA, also known as Morquio A syndrome. The drug first received marketing authorization in the U.S. in February 2014, followed by EU marketing authorization in April 2014 and Japanese approval in December 2014. Elosulfulase alfa is the first-in-therapy drug for MPS IVA, and no drug has ever been approved since then for the indication. Currently, this drug is the top-selling one in the mucopolysaccharidoses treatment landscape. In 2023, BioMarin reported US$ 701 million in revenue for VIMIZIM, and dataM estimates a revenue of approximately US$ 732 million in 2024. The drug had approximately 32% market share in 2024.
North America dominated the Mucopolysaccharidoses market.
The North America region has the highest market share in the global mucopolysaccharidoses market. The factors contributing to the region's dominance include high diagnosis and treatment rates, first availability of approved drugs,
The patient population in the region, especially in the U.S., is highly aware of their disease and their therapeutic choices. Several non-profit organizations in the U.S. create awareness about the disease, which is the major contributing factor to the high diagnosis and treatment rate. For instance, the National MPS Society is an NGO in the U.S. that supports scientific research, family support, and public awareness of MPS disorders. They raise funds, sponsor events, and advocate for support programs, offering resource guides, emotional forums, and family conferences. Several such organizations that are advocating for MPS patients include The National Organization for Rare Disorders (NORD), Project Alive, Angel's Hands Foundation, etc. All these organizations are contributing to the improved diagnosis rate of rare diseases like mucopolysaccharidoses.
In addition to the higher diagnosis rate, the percentage of the population receiving treatment is also high. This is due to the availability of disease-modifying therapies at the patient's convenience. 5 out of seven approved therapies, such as Aldurazyme, Elaprase, VIMIZIM, Naglazyme, and Mepsevii, are available in the region. This represents the region's higher demand for disease-modifying therapies and how manufacturers favor the region first for their product launch.
In addition to these factors, the high sales generated by market players and the robust clinical pipeline are major factors contributing to the region's dominance.
The major players in the Mucopolysaccharidoses market are Sanofi, Takeda Pharmaceuticals, GC Biopharma Corporate., JCR Pharmaceuticals Co., Ltd., BioMarin Pharmaceutical Inc., and Ultragenyx Pharmaceutical Inc., among others.
The global melanoma therapeutics market report would provide approximately 45 tables, 46 figures, and 180 pages.
Target Audience 2024
Key Market Players
Emerging Market Players
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