Welcome!

News Feed Item

Albireo Has Been Granted Orphan Drug Designation for A4250 from the US FDA for the Treatment of Progressive Familial Intrahepatic Cholestasis (PFIC) and for Primary Biliary Cirrhosis (PBC)

GOTHENBURG, Sweden, November 8, 2012 /PRNewswire/ --

Albireo AB (Gothenburg, Sweden; "Albireo"), a biopharmaceutical company specializing in gastroenterology, today announced that A4250 (an inhibitor of the ileal bile acid transporter, IBAT/ASBT) has been granted orphan-drug designation by the U.S Food and Drug Administration ("FDA") for the treatment of

  • Progressive Familial Intrahepatic Cholestasis (PFIC)
  • Primary Biliary Cirrhosis (PBC)

Orphan-drug designation is granted by the FDA to novel drugs that seek to treat a rare disease or condition and provides seven years of market exclusivity for the product upon regulatory approval. Albireo has previously received similar orphan drug designation for the same disease areas from the European Commission.

Orphan designation was granted based on an extensive preclinical data package showing that A4250 has a high potency and a minimal systemic exposure. In addition, substantial benefits were shown in an animal model of cholestasis; the predominant feature of PFIC and PBC.

"We are very pleased with the FDA´s recognition of our technology with A4250 as an orphan medicinal product for the treatment of these liver diseases," said Dr. Hans Graffner, Chief Medical officer at Albireo. "This designation is an important milestone in our efforts to provide a treatment for patients with cholestatic liver diseases; a disease entity often leading to severe symptoms, liver transplantation and with reduced survival. A4250 is an inhibitor of the bile acid transport mechanism and will decrease the toxic levels of bile acids in the liver cells. The approvals of the orphan drug designation for A4250 both in Europe and in the US provide evidence of the quality of the A4250 research and give us confidence to move forward with the development of A4250 in these liver diseases."

About A4250
A4250 belongs to a class of inhibitors of the ileal bile acid transporter (IBAT, syn. apical sodium-dependent bile acid transporter ASBT). A4250 decreases the re-absorption of bile acids and will reduce the toxic levels of bile acids in the liver cells of patients with PFIC and PBC. In preclinical models of cholestasis, treatment with A4250 has been shown to be a very potent inhibitor of IBAT/ASBT with only minimal systemic exposure; thereby reducing the risk for systemic side effects and studies have shown that A4250 decreases the damage in the liver cells and the development of fibrosis/cirrhosis of the liver known to occur in PFIC and PBC. Plans are to move A4250 into clinical studies in 2013.

About Progressive Familial Intrahepatic Cholestasis (PFIC)
PFIC is a rare disease (approximately 5-10,000 patients in the United States) caused by a genetic defect impairing the transport of bile acids thereby inducing toxic levels of bile acid products in the liver inducing severe symptoms such as  intractable itching and scarring of the liver (cirrhosis) early in life. Although milder forms exist, most patients will develop severe symptoms in early childhood. Because medical therapy is ineffective, patients require surgical treatment; otherwise, the disease progresses to liver cirrhosis and failure in the first decade of life. Surgical therapy consists of diversion the bile to a stoma bag or into the large bowel.

If the patient does not get better, or if there is evidence of liver cancer, then liver transplantation may be needed.

About Primary Biliary Cirrhosis (PBC)
PBC is a slowly progressive autoimmune disease of the liver, primarily affecting women; average age when symptoms start is around 40-50 years of age. It is characterized by destruction of bile ducts resulting in an increased bile acid concentration in the liver inducing inflammation and cirrhosis. There are more than 100,000 patients with PBC in the United States. Main symptoms are fatigue, severe itching and symptoms of cirrhosis. There is no cure for PBC, some therapeutic alternatives may slow disease progression and relieve symptoms but some patients may need liver transplantation.

About Albireo
Albireo AB is a Swedish biotechnology company focused on the development of novel therapeutic alternatives in the gastrointestinal area. In addition to A4250, Albireo has a pipeline of drug candidates for the development in areas such as IBD and functional gastrointestinal diseases. The lead program, elobixibat, is ready to enter Phase III in Chronic constipation and is also in development for IBS with constipation. Elobixibat is partnered with Ferring and with Ajinomoto. The Albireo management team has a broad experience in drug development, in particular in the GI area and has an extensive network in the international scientific and clinical communities. Albireo was created as a spin-out of AstraZeneca in 2008, financed by a syndicate of leading healthcare investors, led by Phase4 Ventures, and joined by TPG Biotech, TVM Capital and Scottish Widows Partnership.

SOURCE Albireo AB

More Stories By PR Newswire

Copyright © 2007 PR Newswire. All rights reserved. Republication or redistribution of PRNewswire content is expressly prohibited without the prior written consent of PRNewswire. PRNewswire shall not be liable for any errors or delays in the content, or for any actions taken in reliance thereon.

Latest Stories
"Cloud Academy is an enterprise training platform for the cloud, specifically public clouds. We offer guided learning experiences on AWS, Azure, Google Cloud and all the surrounding methodologies and technologies that you need to know and your teams need to know in order to leverage the full benefits of the cloud," explained Alex Brower, VP of Marketing at Cloud Academy, in this SYS-CON.tv interview at 21st Cloud Expo, held Oct 31 – Nov 2, 2017, at the Santa Clara Convention Center in Santa Clar...
In his session at 21st Cloud Expo, Carl J. Levine, Senior Technical Evangelist for NS1, will objectively discuss how DNS is used to solve Digital Transformation challenges in large SaaS applications, CDNs, AdTech platforms, and other demanding use cases. Carl J. Levine is the Senior Technical Evangelist for NS1. A veteran of the Internet Infrastructure space, he has over a decade of experience with startups, networking protocols and Internet infrastructure, combined with the unique ability to it...
The question before companies today is not whether to become intelligent, it’s a question of how and how fast. The key is to adopt and deploy an intelligent application strategy while simultaneously preparing to scale that intelligence. In her session at 21st Cloud Expo, Sangeeta Chakraborty, Chief Customer Officer at Ayasdi, provided a tactical framework to become a truly intelligent enterprise, including how to identify the right applications for AI, how to build a Center of Excellence to oper...
"IBM is really all in on blockchain. We take a look at sort of the history of blockchain ledger technologies. It started out with bitcoin, Ethereum, and IBM evaluated these particular blockchain technologies and found they were anonymous and permissionless and that many companies were looking for permissioned blockchain," stated René Bostic, Technical VP of the IBM Cloud Unit in North America, in this SYS-CON.tv interview at 21st Cloud Expo, held Oct 31 – Nov 2, 2017, at the Santa Clara Conventi...
Gemini is Yahoo’s native and search advertising platform. To ensure the quality of a complex distributed system that spans multiple products and components and across various desktop websites and mobile app and web experiences – both Yahoo owned and operated and third-party syndication (supply), with complex interaction with more than a billion users and numerous advertisers globally (demand) – it becomes imperative to automate a set of end-to-end tests 24x7 to detect bugs and regression. In th...
In his session at 21st Cloud Expo, James Henry, Co-CEO/CTO of Calgary Scientific Inc., introduced you to the challenges, solutions and benefits of training AI systems to solve visual problems with an emphasis on improving AIs with continuous training in the field. He explored applications in several industries and discussed technologies that allow the deployment of advanced visualization solutions to the cloud.
Widespread fragmentation is stalling the growth of the IIoT and making it difficult for partners to work together. The number of software platforms, apps, hardware and connectivity standards is creating paralysis among businesses that are afraid of being locked into a solution. EdgeX Foundry is unifying the community around a common IoT edge framework and an ecosystem of interoperable components.
Agile has finally jumped the technology shark, expanding outside the software world. Enterprises are now increasingly adopting Agile practices across their organizations in order to successfully navigate the disruptive waters that threaten to drown them. In our quest for establishing change as a core competency in our organizations, this business-centric notion of Agile is an essential component of Agile Digital Transformation. In the years since the publication of the Agile Manifesto, the conn...
"MobiDev is a software development company and we do complex, custom software development for everybody from entrepreneurs to large enterprises," explained Alan Winters, U.S. Head of Business Development at MobiDev, in this SYS-CON.tv interview at 21st Cloud Expo, held Oct 31 – Nov 2, 2017, at the Santa Clara Convention Center in Santa Clara, CA.
Large industrial manufacturing organizations are adopting the agile principles of cloud software companies. The industrial manufacturing development process has not scaled over time. Now that design CAD teams are geographically distributed, centralizing their work is key. With large multi-gigabyte projects, outdated tools have stifled industrial team agility, time-to-market milestones, and impacted P&L stakeholders.
"ZeroStack is a startup in Silicon Valley. We're solving a very interesting problem around bringing public cloud convenience with private cloud control for enterprises and mid-size companies," explained Kamesh Pemmaraju, VP of Product Management at ZeroStack, in this SYS-CON.tv interview at 21st Cloud Expo, held Oct 31 – Nov 2, 2017, at the Santa Clara Convention Center in Santa Clara, CA.
Enterprises are adopting Kubernetes to accelerate the development and the delivery of cloud-native applications. However, sharing a Kubernetes cluster between members of the same team can be challenging. And, sharing clusters across multiple teams is even harder. Kubernetes offers several constructs to help implement segmentation and isolation. However, these primitives can be complex to understand and apply. As a result, it’s becoming common for enterprises to end up with several clusters. Thi...
"Space Monkey by Vivent Smart Home is a product that is a distributed cloud-based edge storage network. Vivent Smart Home, our parent company, is a smart home provider that places a lot of hard drives across homes in North America," explained JT Olds, Director of Engineering, and Brandon Crowfeather, Product Manager, at Vivint Smart Home, in this SYS-CON.tv interview at @ThingsExpo, held Oct 31 – Nov 2, 2017, at the Santa Clara Convention Center in Santa Clara, CA.
"Akvelon is a software development company and we also provide consultancy services to folks who are looking to scale or accelerate their engineering roadmaps," explained Jeremiah Mothersell, Marketing Manager at Akvelon, in this SYS-CON.tv interview at 21st Cloud Expo, held Oct 31 – Nov 2, 2017, at the Santa Clara Convention Center in Santa Clara, CA.
"Codigm is based on the cloud and we are here to explore marketing opportunities in America. Our mission is to make an ecosystem of the SW environment that anyone can understand, learn, teach, and develop the SW on the cloud," explained Sung Tae Ryu, CEO of Codigm, in this SYS-CON.tv interview at 21st Cloud Expo, held Oct 31 – Nov 2, 2017, at the Santa Clara Convention Center in Santa Clara, CA.