The Global Alagille Syndrome Market size was USD 361.89 Million in 2021 and is expected to register a revenue CAGR of 5.8% during the forecast period, according to latest analysis by Emergen Research. Increasing prevalence of alagille syndrome, rising number of clinical studies and trials in the field of alagille syndrome-based treatments, and introduction of novel and advanced diagnostic platforms for early diagnosis are crucial factors driving market revenue growth.
Alagille syndrome is a genetically inherited condition that is similar to other forms of liver disease frequently seen in infants and young children. According to the National Institute of Diabetes and Digestive and Kidney Diseases, Alagille Syndrome affects one in every 30,000 live newborns. Clinically, Alagille Syndrome shows as a variety of phenotypes, but is commonly distinguished by five distinct symptoms, impaired bile flow, congenital heart disease, bone issues, a thickening of the crease of eyelid, and particular facial characteristics. The diagnostics can be supported by genetic testing. ALGS is a multisystem disorder with a wide range of clinical variability, from preclinical symptoms to modestly abnormal liver enzymes, a heart issue, butterfly vertebrae, and posterior embryotoxicity. In addition to supporting biomarker tests and molecular genetic analysis, clinical diagnosis of Alagille syndrome requires proof of a lysosomal acid alpha-glucosidase enzyme deficiency, which can be confirmed in dried blood spots or liquid blood samples.
Alagille syndrome may be diagnosed by means of other tests in addition to a liver biopsy, which include eye exams, spine x-rays to look for distinctive changes such as butterfly vertebrae, abdominal ultrasounds of hepatobiliary tree (including the liver, pancreas, gall bladder, and spleen) to look for abnormalities or rule out other conditions, and blood tests to assess liver function and find fat-soluble vitamin deficiencies. Personalized molecular screening and genetic testing of those who are at risk can result in early diagnosis and treatment beginning, potentially improving clinical results of Alagille syndrome patients.
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Key Companies Profiled in the Report are:
Albireo Pharma, Inc., Bioarray S.L., Mirum Pharmaceuticals, Inc., AbbVie Inc., CANbridge Life Sciences Ltd., Teva Pharmaceutical Industries Ltd., Takeda Pharmaceutical Company Limited., Endo International plc, Mylan N.V., Novartis AG, Amneal Pharmaceuticals LLC. and Epic Pharma, LLC.
Many researchers are trying to better understand the earliest features and manifestations of Alagille syndrome as it has drawn the attention of many researchers for many years, and they have seen the field advance from understanding disease’s pathology and natural history to comprehending possibilities for treating its different clinical disease symptoms, such as liver disease. The European Medicines Agency (EMA) and Food and Drug Administration have granted permission to investigational drugs including Maralixibat and Odevixibat, which are key treatment alternatives for ALGS (FDA). However, their mode of action, is limited to blocking bile acid reabsorption in the intestine and is used to reduce clinical signs of uncommon cholestatic liver diseases, such as Bile Atresia and Progressive Familial Intrahepatic Cholestasis (PFIC).
Rapid scientific advancements in research & development initiatives for treatment of alagille syndrome, have facilitated sparking significant efforts for their translation into the clinical field. 4 clinical research trials for the treatment of alagille syndrome are currently listed on ClinicalTrials.gov, 3 of which are in Phase II stage and 1 of which is in the Phase III stage. In recent decades, various possible treatments for alagille syndrome have begun preclinical and clinical development and are currently in late-stage clinical trials. Subject of ongoing study in the worldwide pharmaceutical industry is the inherited disease alagille syndrome. Major corporations, such as Mirum Pharmaceuticals Inc. and Albireo Pharma Inc., actively support and work on clinical studies for research into possible treatments for Alagille Syndrome. These are the key factors which are significantly contributing to market revenue growth.
However, insufficient research funding for such rare diseases as well as delayed clinical diagnosis of Alagille Syndrome, which can take months or even years, are hindering growth of this market. Furthermore, traditional screening methods present a number of drawbacks, including false-positive tests and delayed results, to name a few. These are some of the factors hindering market growth.
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Some Key Highlights From the Report
The genetic testing segment accounted for a significant revenue share in 2021. Genetic testing is a preferred approach for validating diagnosis of alagille syndrome (ALGS), which is brought on by genetic changes in the Jagged 1 (JAG1), Notch 2 (NOTCH2), or 20p12 chromosomal deletion. Nearly 95% of patients with clinical symptoms and an ALGS-related mutation are found through accurate genetic testing. This procedure, carried out by a medical expert in a lab setting, entails cellular DNA extraction and analysis to find genetic abnormalities that might be the disease’s etiology. Genetic testing can help to predict patient outcomes, recurrence risk, and medical care by identifying gene mutations related to ALGS. Prenatal genetic testing (DNA samples taken during pregnancy) or preimplantation genetic testing (DNA samples taken from embryos created through in vitro fertilization) can aid in early detection of ALGS in fetus if one or both parents have the condition. These are major factors driving growth of this segment.
The hospitals segment accounted for a significant revenue share in 2021. Many hospitals are working on novel and improved ways to facilitate in the early detection of alagille syndrome, with the goal of quickly transferring patients with mild symptoms to expert centers. Patients would be able to be properly tested and diagnosed, allowing them to begin treatment as soon as possible. In addition, hospital doctors and medical specialists are providing individualized methods for all forms of alagille syndrome and additional supportive drugs that address the condition’s symptoms and problems.
The Europe market accounted for a moderate revenue share in 2021. Experimental drugs prepared for alagille syndrome have been gaining an increasing number of approvals in the recent years. For instance, on 30 November, 2020, Mirum Pharmaceuticals, Inc., announced that the validation status for company’s Marketing Authorization Application (MAA) for its investigational drug maralixibat, for treatment of patients with progressive familial intrahepatic cholestasis type 2 (PFIC2), also known as Bile Salt Export Pump (BSEP) deficiency. The biopharmaceutical company is devoted to development and commercialization of novel treatments for crippling liver diseases (EMA). These are major factors driving revenue growth in this region.
On 29 March 2021, Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) announced that its New Drug Application (NDA) for maralixibat, an oral Apical Sodium dependent Bile acid Transporter (ASBT) inhibitor for treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) one year of age and older, has been accepted for filing and priority review by the U.S. Food and Drug Administration (FDA).
Emergen Research has segmented the global alagille syndrome market based on diagnostic method, treatment type, treatment centers, and region:
Diagnostic Method Outlook (Revenue, USD Million; 2019-2030)
Heart and Blood Vessel Tests
Kidney Function Tests
Treatment Type Outlook (Revenue, USD Million; 2019-2030)
Ileal Bile Acid Transporter (IBAT) inhibitors
Late Stage Pipeline Drugs
Treatment Centers Outlook (Revenue, USD Million; 2019-2030)
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Regional Outlook (Revenue, USD Million; 2019-2030)
Rest of Europe
Rest of APAC
Rest of LATAM
Middle East & Africa
Rest of Middle East & Africa
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