Navigation Links
AMT Receives EMEA Orphan Drug Designation for Acute Intermittent Porphyria
Date:5/27/2009

AMSTERDAM, May 28 /PRNewswire-FirstCall/ -- Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, announced today that the European Medicines Agency has granted Orphan Drug Designation to AMT's gene therapy product AMT-021 for the treatment of acute intermittent porphyria (AIP).

Orphan Drug Designation for AIP entitles AMT to ten year market exclusivity in Europe following marketing approval for AMT-021 if this product candidate is the first new drug with a major medical benefit receiving marketing approval for the European Union. The designation also provides for special benefits, including research support, eligibility for protocol assistance and possible exemptions or reductions in certain regulatory fees during development or at the time of application for marketing approval.

"We are proud to have received this Orphan Drug Designation for the treatment of acute intermittent porphyria," said Sander van Deventer, Chief Executive Officer of AMT. "This designation is an important step in the development of a treatment for this seriously debilitating and potentially lethal disease."

About Acute Intermittent Porphyria

Acute intermittent porphyria is a rare genetic disease in which mutations in the PBGD gene result in insufficient activity of a protein necessary for the synthesis of heme. This leads to an accumulation of toxic intermediate metabolites resulting in a wide variety of problems including acute, severe abdominal pains, psychiatric, neurologic illnesses, and muscular weakness. Long-term consequences may be irreversible nerve damage, liver cancer and kidney failure. Acute porphyric attacks can be life-threatening.

AIP affects 1 per 10.000 people in the European Union.

Currently available therapies do not prevent the symptoms and consequences of acute porphyric attacks. AMT has demonstrated that its product, AMT-021, results in normalization of the PBGD protein in an animal model of AIP. In this model, the therapy completely prevented the occurrence of attacks and significantly ameliorated the neuropathy that develops in untreated mice. AMT-021 is intended to provide long-term normalization of the PBGD protein in order to prevent acute porphyric attacks and their complications.

About Amsterdam Molecular Therapeutics

AMT has a unique gene therapy platform that to date appears to circumvent many if not all of the obstacles that have prevented gene therapy from becoming a mainstay of clinical medicine. Using adeno-associated viral (AAV) vectors 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. As such, AMT's proprietary platform holds tremendous promise for thousands of rare (orphan) diseases, especially those that are caused by one faulty gene. Currently, AMT has a product pipeline with nine products at different stages of development.

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
Copyright©2009 PR Newswire.
All rights reserved

Related medicine technology :

1. Reclast(R) Receives US FDA Approval as First and Only Once-Yearly Treatment for Women With Postmenopausal Osteoporosis
2. Indevus Receives Approvable Letter From FDA for VALSTAR(R) for Bladder Cancer Therapy
3. Auxilium Pharmaceuticals, Inc. Receives Clearance to Resume Clinical Trials for XIAFLEX(TM)
4. Abbott Receives Supplemental FDA Approval for its Best-in-Class in Sensitivity RealTime HIV-1 Viral Load Test
5. GlaxoSmithKline Receives New HHS Order for H5N1 Bulk Antigen. First North American Clinical Trials with GSKs Candidate Pre-pandemic Flu Vaccine to Start.
6. CryoCor Receives FDA Approval for Right Atrial Flutter
7. Ranbaxy Receives Approval to Manufacture and Market Hydrocodone Bitartrate and Acetaminophen Tablets USP
8. Dimericine(R) Receives FDA Fast Track Designation for Treatment Of Photosensitivity in XP Patients
9. Poniard Pharmaceuticals Receives FDA Fast Track Designation for Picoplatin for Treatment of Small Cell Lung Cancer
10. Kosan Receives Completed Special Protocol Assessment for TIME-1 Pivotal Phase 3 Trial of Tanespimycin in Multiple Myeloma
11. The Female Health Company Receives USAID DELIVER PROJECT Order for 5 Million Units
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:6/23/2016)... 2016 Research and Markets has ... Global Analysis (United States, China, Japan, Brazil, United Kingdom, ... their offering. Surgical ... business planners, provides surgical procedure volume data in a ... an in-depth analysis of growth drivers and inhibitors, including ...
(Date:6/23/2016)... June 23, 2016  The National Pharmaceutical Council ... the health policy research organization as its newest ... MD, senior vice president and chief scientific officer, ... on the NPC Board of Directors. ... Mallinckrodt has joined us in support of our ...
(Date:6/23/2016)... , June 23, 2016 Research ... Distribution in Europe 2016 Edition: The New Global Agenda " ... OTC Distribution in Europe ... that explores and tracks change. It identifies the driving factors ... for the consumer healthcare industry now and in the future. ...
Breaking Medicine Technology:
(Date:6/24/2016)... ... 24, 2016 , ... Strategic Capital Partners, LLC (SCP) in ... investment capital for emerging technology companies. SCP has delivered investment events and ... more than a million dollars of capital investment for five companies. ...
(Date:6/24/2016)... Washington, D.C. (PRWEB) , ... June 24, 2016 , ... ... Pro-Am Heroes Golf Classic Tournament held on June 20th at the Woodmont Country ... local charity, Luke’s Wings, an organization dedicated to helping service members that have been ...
(Date:6/24/2016)... ... 24, 2016 , ... Advanced Plastic Surgery Institute ( http://www.advancedplasticsurgeryinstitute.com ... its official Medspa Sponsor. Dr. Josh Olson, a board-certified plastic surgeon, owns the ... Olson says the decision to support the pageant in an official capacity is ...
(Date:6/24/2016)... ... June 24, 2016 , ... To succeed under value-based ... however, are unsure how to move forward, given the need to sustain current ... forward tailored to an organization’s specific needs. , PYA Principal Martie Ross states, ...
(Date:6/24/2016)... ... ... Today, InhaleLabs.com (Inhale) offilially launched its site, which aims to ... high quality water pipes within an ideal price range. The site is completely free ... two brothers, Nick and Mike Hunter, who use medical cannabis to heal ailments of ...
Breaking Medicine News(10 mins):