Verdict on AMT’s gene therapy expected soon
pharmafile | May 11, 2011 | News story | Research and Development, Sales and Marketing | AMT, Amsterdam Molecular Therapeutics, gene therapy, genes
Amsterdam Molecular Therapeutics says it will shortly receive the regulatory verdict on its gene therapy Glybera.
Glybera (alipogene tiparvovec) is in development to treat lipoprotein lipase deficiency (LPLD), a group of rare genetic disorders in which a person lacks a protein needed to break down fat molecules.
The disorder causes large amounts of fat to build up in the blood, and is caused by a specific mutation in the gene which codes lipoprotein lipase.
AMT says it ‘continues to work diligently’ with the EMA and is confident a decision on whether or not to approve Glybera will come in mid-2011.
“Our interactions with the EMA continue to be positive and we are encouraged as we hit each milestone in the path towards a regulatory decision. If approved, Glybera would be the first gene therapy product to reach the market in Europe, said Jörn Aldag, chief executive of AMT.
“We will continue to work with the EMA so that the review of our data remains on track as much as possible from our side and look forward to a decision in mid-2011,” he concluded.
The company will present additional data at the forthcoming American Society of Gene and Cell Therapy (ASGCT) annual meeting in Seattle.
In particular, the company will provide an update on a long-term clinical study of Glybera that showed improved chylomicron metabolism could be used as a biomarker in LPLD patients.
Several posters will report advances made by AMT in the field of RNAi delivery including additional data relating to Apoliprotein B silencing in hypercholesterolemia and delivery of miRNA molecules into the brain for development in Huntington’s disease.
AMT scientists and collaborators will also present data from several other gene therapy programmes.
The company has a phase I candidate for haemophilia B and pre-clinical products for Duchenne muscular dystrophy, acute intermittent porphyria, and Parkinson disease.
AMT’s technology uses adeno-associated viral (AAV) derived vectors to deliver therapeutic genes to correct the faulty gene. The company has designed and validated what it calls “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.
Andrew McConaghie
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