Navigation Links
EMA Starts Formal Review of Glybera(R) Dossier

AMSTERDAM, The Netherlands, January 25 /PRNewswire-FirstCall/ -- Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, has reached another important milestone in the official marketing authorisation process for its lead product Glybera(R), AMT's proprietary product for lipoprotein lipase deficiency (LPLD). The submission of the Glybera(R) Marketing Authorisation Application (MAA), announced earlier, has cleared the validation stage with The European Medicines Agency (EMA, formerly known as EMEA). The EMA will now commence its formal review of Glybera(R).

AMT has concluded two clinical studies for LPLD, in Europe and Canada, and long term follow-up from both of these is ongoing, as is a third clinical study in Canada. In these three studies Glybera(R) has shown a sizeable decrease in the incidence of pancreatitis, or acute inflammation of the pancreas, the most debilitating complication of LPLD. In addition, these studies indicate that Glybera(R) has an excellent safety profile.

"The acceptance of the Glybera(R) dossier by EMA is a significant step towards marketing approval for Glybera(R). Moreover, it demonstrates AMT's development capability. A future approval of the MAA for Glybera would fully validate our gene therapy approach and our adeno-associated viral (AAV) vector delivery platform. We believe this step offers hope to many patients, as gene therapy may become the therapeutic approach of choice for inherited disorders" said Jorn Aldag, Chief Executive Officer of AMT.

The EMA formal review will be conducted via the centralised procedure, which is the standard route for all advanced therapies. During 2010, AMT expects to provide further updates on the results from its follow-up and ongoing studies, in accordance with reporting regulations.

About the Disease

LPLD is a seriously debilitating, and potentially lethal, 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. This can result not only in pancreatitis, but also in difficult-to-treat diabetes, and is associated with significant morbidity and mortality.

About Amsterdam Molecular Therapeutics

AMT, founded in 1998 and based in Amsterdam, is a leader in the development of human gene based therapies. Using AAV as the delivery vehicle of choice for therapeutic genes, the company has been able to design and validate what is probably the first stable and scalable AAV production platform. This safe and efficacious proprietary platform offers a unique manufacturing capability which can be applied to a large number of rare (orphan) diseases that are caused by one faulty gene. Currently, AMT has a product pipeline with several AAV-based gene therapy products in LPL Deficiency, Hemophilia B, DMD, Acute Intermittent Porphyria and Parkinson's Disease at different stages of research or development.

For information

AMT will be presenting at the BioCEO & Investor Conference, Waldorf-Astoria, New York City, at 9:30 am (EST) on Tuesday, February 9, 2010.

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 Amsterdam Molecular Therapeutics 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, including, but not limited to, the timely commencement and success of AMT's clinical trials and research endeavors, delays in receiving U.S. Food and Drug Administration or other regulatory approvals (i.e. EMEA, Health Canada), market acceptance of AMT's products, effectiveness of AMT's marketing and sales efforts, development of competing therapies and/or technologies, the terms of any future strategic alliances, the need for additional capital, the inability to obtain, or meet, conditions imposed for required governmental and regulatory approvals and consents. AMT expressly disclaims any intent or obligation to update these forward-looking statements except as required by law. For a more detailed description of the risk factors and uncertainties affecting AMT, refer to the prospectus of AMT's initial public offering on June 20, 2007, and AMT's public announcements made from time to time.

SOURCE Amsterdam Molecular Therapeutics B.V

SOURCE Amsterdam Molecular Therapeutics B.V
Copyright©2010 PR Newswire.
All rights reserved

Related biology technology :

1. KBI Biopharma Starts Cell Line Generation Process under Vendor Network Agreement for PER.C6(R)
2. AMT Starts Preregistration Trial for Glybera(TM)
3. Pierre Fabre Dermatologie Starts Clinical Trials on a Beta-Blocker to Treat Severe Infantile Haemangioma
4. BioCis Pharma Starts Phase IIa Trial in Psoriasis
5. The Pittsburgh Life Sciences Greenhouse Starts 2009 With Investment Momentum
6. China Sky One Medical, Inc. Starts Production at Newly Acquired Facility and Reaffirms Guidance
7. Oncolytics Biotech Inc. Starts Patient Enrolment in U.S. Phase 2 Clinical Trial Investigating REOLYSIN(R) in Combination with Paclitaxel and Carboplatin
8. Gerresheimer Starts Process to Sell off its Business Segment of Technical Plastics
9. Roche starts acquisition process of ARIUS to gain access to new screening platform for antibody therapeutics
10. AMT Starts Collaboration With St. Jude Childrens Research Hospital on Gene Therapy for Hemophilia B. Company Accesses Exclusive Commercial Rights to Final Therapy
11. Anesiva Launches Needle-Free Zingo(TM) to Reduce Pain from IV Starts and Blood Draws in Children
Post Your Comments:
(Date:11/25/2015)... November 26, 2015 ... Market 2016 - 2020 report analyzes that automating ... and quality in long-term samples, minimizing manual errors, ... Automation minimizes manual errors such as mislabeling or ... Further, it plays a vital role in blood ...
(Date:11/25/2015)... HOLLISTON, Mass. , Nov. 25, 2015 ... HART ), a biotechnology company developing bioengineered organ implants ... McGorry will present at the LD Micro "Main ... 2:30 p.m. PT. The presentation will be webcast live ... Management will also be available at the conference for ...
(Date:11/25/2015)... 25, 2015 Orexigen® Therapeutics, Inc. (Nasdaq: ... a fireside chat discussion at the Piper Jaffray 27th ... . The discussion is scheduled for Wednesday, December 2, ... .  A replay will be available for 14 days ... , Julie NormartVP, Corporate Communications and Business Development , ...
(Date:11/24/2015)... ... , ... The United States Golf Association (USGA) today announced Dr. Bruce Clarke, ... annually since 1961, the USGA Green Section Award recognizes an individual’s distinguished service to ... Clarke, of Iselin, N.J., is an extension specialist of turfgrass pathology in the department ...
Breaking Biology Technology:
(Date:11/20/2015)... , November 20, 2015 ... company focused on the growing mobile commerce market and ... Gino Pereira , was recently interviewed on ... will air on this weekend on Bloomberg Europe ... America . --> NXTD ) ("NXT-ID" or ...
(Date:11/18/2015)... --> --> ... report titled  Gesture Recognition Market - Global Industry Analysis, ... to the report, the global gesture recognition market was valued at ... US$29.1 bn by 2021, at a CAGR of 20.3% ... dominated the global gesture recognition market in ...
(Date:11/17/2015)... 17, 2015 Pressure BioSciences, Inc. (OTCQB: PBIO) ... and sale of broadly enabling, pressure cycling technology ("PCT")-based ... today announced it has received gross proceeds of $745,000 ... Placement (the "Offering"), increasing the total amount raised to ... additional closings are expected in the near future. ...
Breaking Biology News(10 mins):