Navigation Links
First Gene Therapy in Western World Receives Positive Opinion in Europe from CHMP
Date:7/20/2012

AMSTERDAM, The Netherlands, July 20, 2012 /PRNewswire/ --

  • First gene therapy in the Western world to reach important regulatory approval milestone, culminating 40 years of research
  • First therapy for LPL deficient patients, a severe disease with no alternative treatment
  • Validates uniQure's unique AAV-based gene therapy platform, consisting of a modular, plug-and-play vector system and unrivaled GMP manufacturing capabilities on a commercial scale
  • Heralds new phase in uniQure's development, including potential revenues from sales and partnerships
  • Technology platform can now be leveraged to find solutions for many more severe genetic and other disorders

uniQure announced today that the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion that recommends marketing authorization of Glybera® (alipogene tiparvovec) as a treatment for lipoprotein lipase deficiency (LPLD) under exceptional circumstances. LPLD is a very rare, inherited disease. Patients with the disease are unable to handle fat particles in their blood plasma, which leads to recurring severe abdominal pain and pancreatitis.

The European Commission (EC) generally follows the recommendations of the CHMP. "We expect final approval from the EC within 3 months after the CHMP decision," says Jörn Aldag, CEO of uniQure.  "After today's positive recommendation, Glybera is poised to become the first in a class of gene therapy products approved in Europe to treat orphan diseases, rare conditions with a very high unmet medical need." Marketing authorization covers all 27 European Union member states.

Mr. Aldag continued: "Patients with LPLD are afraid of eating a normal meal because it can lead to acute and extremely painful inflammation of the pancreas, often resulting in a visit to intensive care. Now, for the first time, a treatment exists for these patients that not only reduces this risk of getting severely sick, but also has a multi-year beneficial effect after just a single injection. The positive recommendation from the CHMP for Glybera therefore represents a major breakthrough for both LPLD patients and for medicine as a whole. Restoring the body's natural ability to break down fat particles in the blood in order to prevent pancreatitis and excruciating abdominal pain suffered by patients, is what gene therapy is all about: curing disease at the genetic level."

"At uniQure we are developing treatments for a number of other rare diseases as well, such as acute intermittent porphyria and Sanfilippo B. But the potential of gene therapy stretches far beyond rare diseases. As shown recently in a publication in the New England Journal of Medicine (N Engl J Med 2011; 365:2357-2365, December 22, 2011), hemophilia patients treated with our proprietary gene are showing a sustained clinical effect over several years, which has allowed prophylaxis treatment to be stopped. In addition, we are advancing programs in degenerative diseases such as Parkinson's. We believe that just like antibodies, gene therapy will one day be a mainstay in clinical practice," Mr Aldag added.

As part of the approval, treatment with Glybera will be offered through dedicated centers of excellence with expertise in treating LPLD and by specially trained doctors to ensure ongoing safety of this novel treatment paradigm. uniQure has also committed to building a patient registry for continued understanding of this devastating, under-researched disease. The Company is now preparing to apply for regulatory approval in the US, Canada, and other markets.

Glybera has been tested in three interventional clinical studies conducted in the Netherlands and in Canada, in which a total of 27 LPLD patients participated. In all three clinical trials, Glybera was well tolerated, with no relevant safety issues observed. Data from these clinical trials indicate that a single dose administration of Glybera resulted in a long-term biological activity of the LPL protein.

About Glybera®

uniQure has developed Glybera as a therapy for patients with the genetic disorder lipoprotein lipase deficiency. LPLD is an orphan disease for which no treatment exists today. The disease is caused by mutations in the LPL gene, resulting in highly decreased or absent activity of LPL protein in patients. This protein is needed in order to break down large fat-carrying particles that circulate in the blood after each meal. When such particles, called chylomicrons, accumulate in the blood, they may obstruct small blood vessels. Excess chylomicrons result in recurrent and severe acute inflammation of the pancreas, called pancreatitis, the most debilitating complication of LPLD. Glybera has orphan drug designation in the EU and US. LPL Deficiency affects 1-2 persons per million. For further information on LPLD visit http://www.lpldeficiency.com.

About uniQure

uniQure is a world leader in the development of human gene based therapies. uniQure has a product pipeline of gene therapy products in development for hemophilia B, acute intermittent porphyria, Parkinson's disease and SanfilippoB. Using adeno-associated viral (AAV) derived vectors as the delivery vehicle of choice for therapeutic genes, the company has been able to design and validate probably the world's first stable and scalable AAV manufacturing platform. This proprietary platform can be applied to a large number of rare (orphan) diseases caused by one faulty gene and allows uniQure to pursue its strategy of focusing on this sector of the industry.  uniQure's largest shareholders are Forbion Capital Partners and Gilde Healthcare, two of the leading life sciences venture capital firms in the Netherlands. Further information can be found at http://www.uniqure.com.


'/>"/>
SOURCE uniQure B.V.
Copyright©2012 PR Newswire.
All rights reserved

Related biology technology :

1. First detailed timeline established for brains descent into Alzheimers
2. Ethical Corporation Names Life Technologies First CSO Sustainability Executive of the Year
3. Janssen Research & Development, LLC, Sponsors First of its Kind Research Initiative to Advance Multiple Sclerosis Science
4. infirst HEALTHCARE Established to Launch Novel Consumer Medicines
5. Hark! Group demonstrates first heralded single photon source made from silicon
6. First 3-D nanoscale optical cavities from metamaterials
7. First Genetic Cure For Cancer Anchors Provocative New Thriller
8. Prosperous Agriculture Reports Outstanding Sales Result for First Half of 2012
9. Nourish the World! Terra Biotech Receives Thanks and Praise After First Shipment of 10,000 Servings of Nectr Core Vitamin Powder to Orphans in Ghana, Africa
10. Beyond Batten Disease Foundation and American Brain Foundation Launch First Clinical Research Fellowship to Treat and Cure Juvenile Batten Disease
11. NDIA, NTSA Will Be On Hand At Seventh Annual Capitol Hill Modeling and Simulation Expo, Set To Focus On Homeland Security, First Response
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:2/11/2016)... 11, 2016  Wellcentive today announced it has ... Oregon -based community care organization (CCO) with ... analytics, quality reporting and care management solutions and ... team of quality managers, analysts and care managers ... groups serving FamilyCare members. ...
(Date:2/11/2016)... , February 11, 2016 ... Corporation ("PositiveID" or "Company") (OTCQB: PSID), a life ... today that its Thermomedics subsidiary, which markets the ... its growth plan in January 2016, including entering ... increasing sequential monthly sales growth, and establishing several ...
(Date:2/11/2016)... ... February 11, 2016 , ... ... a new agreement with Bankok,Thailand-based Global Stem Cells Network (GSCN) to distribute exosome ... Latin American countries, including Mexico, Costa Rica, Dominican Republic, Colombia, Argentina, Nicaragua, Panama, ...
(Date:2/10/2016)... Early-career researchers from Indonesia ... Uganda and Yemen ... Indonesia , Nepal , ... Yemen are being honored for their accomplishments in nutrition, psychiatry, ... mentoring young women scientists who are pursuing careers in agriculture, biology and ...
Breaking Biology Technology:
(Date:2/3/2016)... Feb. 3, 2016 ... of the "Emotion Detection and Recognition ... and Others), Software Tools (Facial Expression, Voice ... Users,and Regions - Global forecast to 2020" ... --> http://www.researchandmarkets.com/research/d8zjcd/emotion_detection ) has announced the ...
(Date:2/2/2016)... 2016 Checkpoint Inhibitors for Cancer – ... Are you interested in the future of cancer ... inhibitors. Visiongain,s report gives those predictions to 2026 ... level. Avoid falling behind in data or ... revenues those emerging cancer therapies can achieve. There ...
(Date:2/2/2016)... Feb. 2, 2016  Based on its recent ... Sullivan recognizes US-based Intelligent Retinal Imaging Systems (IRIS) ... Award for New Product Innovation. IRIS, a prominent ... North America , is poised to set ... diabetic retinopathy market. The IRIS technology presents superior ...
Breaking Biology News(10 mins):