The global progressive familial intrahepatic cholestasis type 2 treatment market size was valued at USD 35 million in 2021 and is projected to reach around USD 550 million in 2030 exhibiting a CAGR of 39.0% in the forecasted period. The rise in the number of drug approvals from the regulatory bodies is anticipated to boost the growth of the market during the forecasted period. Furthermore, rise in the demand from developing nations will elevate the market growth, also increase in the prevalence of the disease, upsurge in the healthcare affordability in the US and increase in the knowledge and awareness between the people are some of the critical factors boosting the market during the expected period. Conversely, rise in the cost of research and development activities in the market acts as the hindrance in the market in the forecasted period.
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Growth in research and development of new drugs for progressive familial intrahepatic cholestasis (PFIC) type 2 treatment is anticipated to fuel the market throughout the forecasted period. For instance, in August 2020, Mirum Pharmaceuticals, Inc. offered outline from its phase 2 indigo study at the International Liver Congress 2020. The five-year analysis characterized that patients with Progressive familial intrahepatic cholestasis (PFIC) type 2, also known as bile salt export pump (BSEP) deficiency, who accomplished long-term maralixibat treatment have a substantial enhancement in the transplant-free survival.
Increase in drug approvals from regulatory bodies is projected to boost the global progressive familial intrahepatic cholestasis type 2 treatment market during the forecasted period. For example, in November 2020, Mirum Pharmaceuticals, Inc. declared that the corporation’s Marketing Authorization Application (MAA) for its investigational medicine, Maralixibat, for the management of patients with progressive familial intrahepatic cholestasis type 2 (PFIC2), received approval for analysis from the European Medicines Agency (EMA).
Progressive familial intrahepatic cholestasis (PFIC) type 2 is an infrequent genetic disorder that has no basic treatment and needs R&D for new treatment drugs. There is no remedial therapy for progressive familial intrahepatic cholestasis (PFIC) type 2. Hence, absence of new therapies is anticipated to obstruct the progressive familial intrahepatic cholestasis (PFIC) type 2 treatment market.
Patients with moderate familial intrahepatic cholestasis type 2 are anticipated to receive special attention throughout the coronavirus pandemic since the liver conditions can be enormously comorbid throughout the coronavirus disease. The symptoms of liver injury have revealed in as many as half the individuals contaminated with the coronavirus. The list of possible followers of hepatic injury in COVID-19 patients incorporates infection, though fundamental ailment, and the ischemic, safe, medication, and liquor-induced stresses of liver injury, and the several medicines for these conditions. Owing to the COVID-19 pandemic, important concern visits have reduced significantly, and non-pressing and elective methods are put off. Liver transplantation is a management choice for extreme examples of reasonable familial intrahepatic cholestasis type 2. In any situation, during the coronavirus pandemic, all liver transfer exercises were temporarily suspended owing to a flood of clinic confirmations, ICU bed shortage, and organ attainment restrictions carried out by the organ gift authority.
The increasing number of ground-breaking work exercises, clinical beginnings, and medicine authorizations by vital applicants for advanced work are the central facts that are anticipated to drive the growth of the global progressive familial intrahepatic cholestasis type 2 treatment market during the forecasted period.
Drug Class Insights
Among drugs, the ursodeoxycholic acid portion is depended on to hold a substantial share in 2021, as the interest in ursodeoxycholic acid is high as it is a secure medicine with no substantial consequences and is convincing for an extensive range of modest domestic intrahepatic cholestasis (PFIC), as would be measured normal to boost the section’s development through the estimated timeframe. Ursodeoxycholic acid is hydrophilic bile, which is not dangerous to hepatocytes. Ursodeoxycholic acid replaces the hydrophobic bile salts, which are lethal for the human body, and it is deliberated an introductory restorative administration for kids with a widespread range of reasonable familial intrahepatic cholestasis (PFIC).
Distribution Channel Insights
On the basis of distribution channel, the progressive familial intrahepatic cholestasis type 2 treatment market has been categorized into retail pharmacies, hospital pharmacies, and online pharmacies. The hospital pharmacies category is to observe higher adoption of progressive familial intrahepatic cholestasis type 2 treatment during the forecasted period. This is mostly owing to the accessibility of experienced healthcare professionals & the safety & efficiency throughout the treatment procedures also increasing hospital administration of patients globally are the major aspects for the growth of the segment.
Region Insights
North America region is predicted to hold a prominent share of the global progressive familial intrahepatic cholestasis type 2 treatment market during the forecasted period, owing to an upsurge in clinical trials intervention for progressive familial intrahepatic cholestasis type 2 treatment. For example, in September 2020, Albireo Pharma, Inc. declared positive top line results from PEDFIC 1, a global Phase 3 clinical trial calculating the security of odevixibat conducted for PFIC1 and PFIC2. Odevixibat reduce bile acids in both PFIC1 and PFIC2 patients and validated a clinically proven better results for pruritus.
However, the market in Europe is projected to expand at a fast pace during the forecasted period. Furthermore, significant market players are dedicated on the growth of novel drugs to broaden their product portfolio, which is estimated to boost the market in the region during the forecasted period. For example, in December 08, 2020, Albireo Pharma, Inc. submitted Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) to obtain derive approval for odevixibat, useful for the treatment of patients experiencing the progressive familial intrahepatic cholestasis (PFIC) type 2.
Key Companies Insights
The market for progressive familial intrahepatic cholestasis type 2 treatment is moderately competitive. With the rising applications of Progressive familial intrahepatic cholestasis type 2 treatment, new players are considering to enter the market. The companies are also involved in events like joint ventures, acquisitions, partnerships, mergers, and collaborations. These activities aid in growing the effect of the players in the market, ultimately boosting the market growth. Some of the key companies working in the global market include:
• Teva Pharmaceutical Industries Ltd.
• AbbVie, Inc.
• Glenmark Pharmaceuticals Limited
• Par Pharmaceuticals, Inc.
• Mylan Pharmaceuticals, Inc.
• Sanofi S.A.
• Novartis International AG
• Akorn, Inc.
• Albireo Pharma, Inc.
• Mirum Pharmaceuticals
• Other players
Some of the Recent Developments:
• In November 2020, Xencor, MorphoSys and Incyte entered into a clinical collaboration to examine the combination of tafasitamab, enalidomide, and plamotamab in patients with progressive familial intrahepatic cholestasis type 2 treatment (DLBCL).
• In July 2020, MorphoSys AG and Incyte got approval from the U.S. Food and Drug Administration (FDA) for Monjuvi along with lenalidomide for the management of adult patients with progressive familial intrahepatic cholestasis type 2 treatment (DLBCL) not otherwise specified, including DLBCL rising from low grade lymphoma, and who are not entitled for autologous stem cell transplant (ASCT).
Segments
By Drug Class
• Ursodeoxycholic Acid
• Cholestyramine
• Rifampicin
• Late Stage Pipeline Drugs
• Others
By Distribution Channel
• Retail Pharmacies
• Hospital Pharmacies
• Online Pharmacies
By Geography
• North America
o U.S.
o Canada
o Mexico
• Europe
o U.K.
o Germany
o France
o Italy
o Spain
o Russia
• Asia-Pacific
o Japan
o China
o India
o Australia
o South Korea
o ASEAN
• Latin America
o Brazil
o Argentina
o Colombia
• MEA
o South Africa
o Saudi Arabia
o UAE
o Egypt
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Key Questions answered with an analysis in this report:
- What are the current market trends? Will there be an increase or decrease in market demand in the following years?
- Which product categories are expected to have the highest demand? What kind and applications might we expect in the future?
- What Economic Predictions are Available for the Industry Considering Capacity, Production, and Production Value?
- Which costs and profits are projected? What Will the Market Share, Consumption, and Supply Be? How do imports and exports operate?
- In light of the strategic advancements, where will the industry be in the mid- to long-term?
- What are the primary raw materials employed in the production?
- What is the value of the global market? How much was the market worth in 2022?
- Which companies are the main players in the market? Which businesses are leading the pack?
- What are the current business trends that can be used to create new sources of income?
- What Entry Strategies, Economic Impact Countermeasures, and Marketing Channels Should the Industry Use?
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