Navigation Links
Amsterdam Molecular Therapeutics Receives Opinion on Glybera(R) Marketing Authorisation Application
Date:6/23/2011

AMSTERDAM, June 24, 2011 /PRNewswire/ --


 

- Dossier Re-Examination Process Initiated

Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, today announced that it has received an opinion on its Marketing Authorisation Application (MAA) for Glybera® (alipogene tiparvovec) as a potential therapy for Lipoprotein Lipase Deficiency ("LPLD"). Following a recent meeting with the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP), AMT has been notified that, at this time, Glybera is not approvable.

Subsequent to a review of the CHMP's letter, AMT believes that Glybera can receive a positive opinion subject to generating additional data from existing patients. AMT has therefore decided to ask for a re-examination of the clinical data package.

"From what we know today, despite the disappointment, we believe that there is an indication from the CHMP that Glybera could receive approval and that the current opinion at this time is a reflection of insufficient proof of clinical benefit of Glybera as a result of low patient numbers measured for chylomicron handling for at least 12 months post treatment. The CHMP also indicated that if certain additional data from already treated patients would confirm current results by the end of 2011, an approval may be possible," noted Jörn Aldag, CEO of AMT. "In the dossier, we provided important data showing Glybera is safe and prevents episodes of pancreatitis, the major clinical complication of LPLD. We appreciate the CHMP's responsibility for caution on such an advanced therapy, so we will work diligently to generate more information and, we hope, ensure that Glybera will still reach patients."

Next steps

As part of the MAA, AMT presented data to the CHMP on Glybera showing evidence in reducing the risk of pancreatitis. Also, the company demonstrated a clear signal of patients' ability to break down large chylomicron molecules, the accumulation of which, after intake of dietary fat, seems to be responsible for the occurrence of pancreatitis in LPLD patients. AMT is initiating study CT-AMT-011-04 to examine further how already treated patients handle chylomicrons over time. In addition, AMT will continue to collect follow up data on pancreatitis incidence. AMT's available funds will support its existing operations beyond the completion of the re-examination process into 2012.

"Based on communication from EMA, we understand that our technology platform using adeno-associated virus (AAV) vectors is approvable," Mr. Aldag added. "While we pursue the re-examination of Glybera, we will also continue development of other products in our pipeline such as hemophilia B and GDNF gene therapy for Parkinson's disease and Huntington's disease. We will also continue our preparations for registrations in Canada and the US."

For investors, AMT will conduct a conference call at 9:00 AM CET on June 24, 2011. To participate in the conference call, please call one of the following telephone numbers 15 minutes prior to the event, using access code 2788745: +44-(0)20-7136-2054 for the UK; +1-718-247-0881 for the US; and + 31-(0)20-201-5468 for the Netherlands. A pdf version of the presentation is available in the news and investor relations sections of the corporate website. Following the presentation, the lines will be opened for a question and answer session. A replay of the call will be available following the event.

About Glybera

AMT has developed Glybera as a treatment 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 status in the EU and US.

About Amsterdam Molecular Therapeutics

AMT is a world leader in the development of human gene based therapies. In addition to Glybera, AMT has a product pipeline of several gene therapy products in development for hemophilia B, Duchenne muscular dystrophy, 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 AMT to pursue its strategy of focusing on this sector of the industry.  AMT was founded in 1998 and is based in Amsterdam. Further information can be found at http://www.amtbiopharma.com.

Certain statements in this press release are "forward-looking statements" including those that refer to management's plans and expectations for future operations, prospects and financial condition. Words such as "strategy,""expects,"" plans,""anticipates,""believes, ""will,""continues," "estimates,""intends,"" projects,""goals,""targets " and other words of similar meaning are intended to identify such forward-looking statements. Such statements are based on the current expectations of the management of AMT only. Undue reliance should not be placed on these statements because, by their nature, they are subject to known and unknown risks and can be affected by factors that are beyond the control of AMT. Actual results could differ materially from current expectations due to a number of factors and uncertainties affecting AMT's business. AMT expressly disclaims any intent or obligation to update any forward-looking statements herein except as required by law. 

PRN NLD


'/>"/>
SOURCE Amsterdam Molecular Therapeutics B.V
Copyright©2010 PR Newswire.
All rights reserved

Related medicine technology :

1. Pharma and Food Thought Leaders Set to Converge in Amsterdam Next April 2011
2. Amsterdam Molecular Therapeutics Reports Promising Data From Cholesterol Lowering Gene Therapy Study
3. PharmacoFore, Inc. Announces Positive Results from a Phase I Clinical Study of Its Hydromorphone Bio-Activated Molecular Delivery™ System, and Upcoming Presentation at the 2011 BIO Business Forum During the Annual BIO International Convention in Was
4. Latest Molecular Diagnostics Technology Aids German Hospital in Management of European E. coli Crisis
5. Data from Clinical Studies of bioTheranostics CancerTYPE ID® and Breast Cancer Index(SM) Molecular Diagnostic Tests Presented at ASCO 2011 Annual Meeting
6. ImaginAb, Inc. Announces Availability of Custom Protein Radiolabeling Services for Molecular Imaging
7. Medco Partners With MolecularMD on Personalized Medicine Program for Chronic Myeloid Leukemia
8. The World Leader in Molecular Breast Imaging to Premiere Largest Detector in the Industry
9. Gen-Probe Files for US Regulatory Clearance of PANTHER™ System, Fully Automated and Integrated Molecular Testing Instrument
10. Latest Clinical Evidence Supports Promising Outlook for Low-Dose Molecular Breast Imaging (MBI)
11. Cepheid Extends Rapid Molecular Diagnostics Leadership with FDA Clearance of Xpert Clostridium difficile/Epi
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:5/27/2016)... Amarantus BioScience Holdings, Inc. (OTCQX: AMBS), ... Neurology and Orphan Diseases, today announced that President & CEO ... conferences: SeeThru Equity MicroCap Conference   ... City , NY When: Tuesday, May 31 st ... Where: Grand Hyatt Hotel, 109 East 42 nd ...
(Date:5/26/2016)... -- According to a new market report published ... Industry Analysis, Size, Share, Growth, Trends, and Forecast 2015 - ... valued at US$ 5.89 Bn in 2014 and is anticipated ... 2023 to reach US$ 7.99 Bn in 2023. ... free drug delivery devices and the market is estimated based ...
(Date:5/26/2016)... TARE (Transarterial Radio-embolization) Using ... and Overall Decreased Use of Hospital Resource ... specialist healthcare company, has today announced the publication ... of ISPOR (International Society for Pharmacoeconomics and Outcomes ... using yttrium-90 glass microspheres is associated with cost ...
Breaking Medicine Technology:
(Date:5/27/2016)... ... May 27, 2016 , ... Two director-level employees of ... Tribute to Women and Industry (TWIN) 2016 honorees. The award recognizes businesswomen who ... this year, Geri Boone, Director of the MLTSS (Managed Long-Term Services and Supports) Program ...
(Date:5/27/2016)... ... 27, 2016 , ... With over 60 percent of acute stroke survivors being ... product to aid in the rehabilitation process has steadily increased. Ekso Bionics had been ... hemiplegia due to stroke. , Ekso Bionics has now received clearance from the U.S. ...
(Date:5/27/2016)... ... May 27, 2016 , ... ... help educate the many who are unaware of the plight of aphasia. In ... run within the “Stroke Awareness” campaign. , The link between stroke and aphasia ...
(Date:5/27/2016)... ... , ... Aimed at nurses and employees in the health care world, this ... the nursing and health care industry. It also provides insight to the developing trends ... , As the nursing industry is coming out of one of the biggest recessions ...
(Date:5/26/2016)... (PRWEB) , ... May 26, 2016 , ... Despite last ... meeting, expect Janet Yellen and company to wait until March 2017 for an interest ... University’s J. Mack Robinson College of Business. , “The Federal Open Market Committee (FOMC) ...
Breaking Medicine News(10 mins):