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
| Report Attribute/Metric | Details |
| Â Â Market Size | Â Â 0.75 Billion |
| Â Â CAGR | Â Â 2024-2032: 6.32% CAGR |
| Â Â Base Year | Â Â 2022 |
| Â Â Forecast Period | Â Â 2032 |
| Â Â Historical Data | Â Â 2018 |
| Â Â Forecast Units | Â Â Value (USD Billion) |
| Â Â Report Coverage | Â Â Revenue Forecast, Competitive Landscape, Growth Factors, and Trends |
| Â Â Segments Covered | Â Â Symptom, Treatment, Diagnosis, And End-User |
| Â Â Geographies Covered | Â Â North America, Europe, Asia-Pacific, and Rest of the World (RoW) |
| Â Â Key Vendors | Â Â Sanofi AG, Teva Pharmaceutical Industries Ltd., Allergen Plc, Lannett, Albireo Pharma, Inc., AstraZeneca Plc, Novartis AG, Pfizer, Inc., Shire, Bristol-Myers Squibb Company, GlaxoSmithKline Plc, Merck & Co., Inc |
| Â Â Key Market Opportunities | Â Â Increasing government funding for the healthcare sector |
|   Key Market Drivers | · Increasing healthcare expenditure · Rising prevalence of genetic diseases |
Alagille Syndrome Market Highlights:
Frequently Asked Questions (FAQ) :
Alagille market may suffer a setback due to a lack of awareness.
Symptoms included in the alagille syndrome market are nutrition problems, liver problems, distinctive facial features, neurologic problems, heart problems, and others.
The segments in alagille syndrome market report are biliary diversion procedures, medication, liver transplantation, vitamin supplements, heart surgery, kidney surgery, and others.
The segments included in alagille syndrome market report are urinalysis, prenatal DNA testing, blood test, slit-lamp exam, X-ray imaging, cardiology exam, genetic testing, liver biopsy, and others.
The end users involved in alagille syndrome market report are diagnostic centers, hospitals & clinics, and others.
Leading companies partner with us for data-driven Insights.
Kindly complete the form below to receive a free sample of this Report
Assistant Manager He holds an experience of about 7+ years in market research and business consulting, working under the spectrum of life sciences and healthcare domains. rahul conceptualizes and implements a scalable business strategy and provides strategic leadership to the clients. his expertise lies in market estimation, competitive intelligence, pipeline analysis, customer assessment, etc. in addition to the above, his other responsibility includes strategic tracking of high growth markets & advising clients on the potential areas of focus they could direct their business initiatives
Get in Touch
© 2024 Market Research Future ® (Part of WantStats Reasearch And Media Pvt. Ltd.)
