The demand for solutions in the US Alagille Syndrome market has witnessed a notable increase, reflecting a growing awareness and understanding of this rare genetic disorder. Alagille Syndrome is a rare genetic condition characterized by liver, heart, and other organ abnormalities, caused by mutations in the JAG1 or NOTCH2 genes. The rising demand can be attributed to various factors, including improved diagnostic capabilities, advancements in therapeutic options, and a concerted effort to address the specific needs of individuals affected by Alagille Syndrome.
Advancements in diagnostic capabilities have played a crucial role in meeting the demand for early and accurate identification of Alagille Syndrome. Genetic testing, including molecular testing for JAG1 and NOTCH2 gene mutations, has become a key tool in diagnosing this rare disorder. The ability to precisely identify the underlying genetic cause of Alagille Syndrome enables healthcare professionals to provide tailored management plans and support, contributing to the overall demand for solutions in the market.
The growing awareness of Alagille Syndrome among healthcare professionals, patients, and their families has also driven demand in the US market. As medical knowledge expands and information about rare genetic disorders becomes more accessible, there is an increasing recognition of the specific challenges posed by Alagille Syndrome. This heightened awareness has led to earlier and more accurate diagnoses, creating a greater demand for therapeutic interventions and supportive care to address the complex and varied symptoms associated with the syndrome.
The demand for solutions in the Alagille Syndrome market is further fueled by the development of therapeutic options aimed at managing the various manifestations of the disorder. While there is currently no cure for Alagille Syndrome, treatment approaches focus on alleviating specific symptoms and improving overall quality of life. Therapies targeting liver, heart, and other affected organs are being explored, and the market is witnessing ongoing research and clinical trials to expand the range of available treatment options.
Moreover, the emphasis on multidisciplinary care has become a central aspect of meeting the demand in the Alagille Syndrome market. Given the multi-organ nature of the disorder, collaboration among healthcare professionals from various specialties, including hepatologists, cardiologists, and genetic counselors, is essential. This comprehensive and coordinated approach ensures that individuals with Alagille Syndrome receive holistic care that addresses the diverse medical and developmental challenges associated with the disorder.
The supportive community and advocacy efforts for Alagille Syndrome have played a significant role in raising awareness, fostering research, and addressing the unique needs of individuals and families affected by the disorder. Support organizations provide valuable resources, connect affected individuals with expert medical care, and contribute to ongoing research initiatives. This collective effort has not only increased visibility but has also driven demand for improved diagnostic tools, therapeutic options, and supportive services within the Alagille Syndrome market.
The COVID-19 pandemic has highlighted the importance of telemedicine and remote healthcare services, especially for individuals with rare disorders like Alagille Syndrome. The adoption of virtual consultations, telehealth platforms, and remote monitoring tools has facilitated ongoing care and support for individuals with Alagille Syndrome, ensuring continuity of services during challenging times.
The US Food and Drug Administration (FDA) has broadened the approved use of odevixibat (Bylvay) to treat cholestatic pruritus linked to Alagille syndrome (ALGS) in patients as young as 12 months. ALGS is an uncommon hereditary condition characterized by the accumulation of bile in the liver due to an insufficient number of bile ducts, resulting in liver injury. ALGS can also impact several organ systems, such as the cardiovascular system, musculoskeletal system, visual system, and renal system. Almost all individuals with the illness exhibit persistent cholestasis, typically occurring within the initial months of birth. Additionally, up to 88% of these individuals also experience intense and uncontrollable itching. Odevixibat is a powerful, non-systemic inhibitor of bile acid transport in the ileum. The FDA granted approval for the medication in 2021 to treat individuals suffering from cholestatic pruritus caused by progressive familial intrahepatic cholestasis. The FDA approval for odevixibat in ALGS was granted based on the findings of the phase 3 ASSERT study. This study assessed the safety and effectiveness of administering 120 µg/kg/day of odevixibat for a duration of 24 weeks. The study aimed to determine the extent to which odevixibat might alleviate pruritus in individuals ranging from newborns to teenagers, aged from birth to 17 years, who have genetically-confirmed ALGS. The research was carried out at 32 locations spanning North America, Europe, the Middle East, and the Asia Pacific region.
March 2024:
Mirum Pharmaceuticals Inc. has declared that the United States. The Food and Drug Administration (FDA) has granted approval for the use of LIVMARLI® (maralixibat) oral solution in the treatment of cholestatic pruritus in patients who are at least five years old and have progressive familial intrahepatic cholestasis (PFIC). Mirum has filed an additional supplemental new drug application (sNDA) to provide a more concentrated formulation of LIVMARLI, which was utilized in the MARCH research. The purpose of this application is to expand the approved uses of LIVMARLI to include younger patients with PFIC. This label extension is expected to occur later this year. LIVMARLI is authorized for the management of cholestatic pruritus in patients diagnosed with Alagille syndrome (ALGS) in the United States (for individuals aged three months and older), Europe (for individuals aged two months and older), Canada, and other regions worldwide. LIVMARLI has the capacity to bring about a significant and profound change in the treatment of patients suffering from cholestatic pruritus linked to PFIC. It is particularly noteworthy that LIVMARLI provides a viable choice for patients with the most uncommon subtypes of this condition.
Key players
Geographically, the Americas is anticipated to dominate the global alagille syndrome market owing to a well-developed healthcare sector, rising prevalence of rare genetic disease and growing healthcare expenditure. According to the Global Genes Project, rare and genetic diseases affect 1 in 10 people in the United States Additionally, increasing awareness among the people regarding the disease and well-developed technology is likely to contribute to the growth of the market.
Europe is expected to hold the second largest position in the alagille syndrome global market. The market growth in this region is attributed to the growing prevalence of several liver diseases, increasing prevalence of genetic disease, availability of funds for research, and increasing healthcare expenditure. According to the Public Health England, approximately 11,597 number of people died with an underlying cause of liver disease in England in 2014. In addition, according to the Office for National Statistics, gross domestic expenditure on research and development (R&D) was Euro 33.1 billion (USD 35.2 billion) in 2016.
Asia-Pacific is anticipated to be the fastest growing region in the market due to the presence of a huge patient population, continuously developing economies, growing occurrences of rare diseases and increasing government funding for the healthcare sector.
On the other hand, the Middle East & Africa has the least share of the market. Majority of the market of this region is expected to be held by the Middle East region due to a well-developed healthcare sector and growing government initiatives for the healthcare sector.
Intended Audience
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