European Medicines Agency rejects MAA for Amsterdam Molecular Therapeutics’ Glybera


Amsterdam – The European Medicines Agency (EMA) has rejected a market approval application for Amsterdam Molecular Therapeutics’ (AMT) gene therapy Glybera (Alipogene tiparvovec, AMT-011), the first potential cure for the inherited rare disease lipoprotein lipase deficiency (LPLD). Patients with LPLD cannot hydrolyse the triglyceride component of circulating chylomicrons and very low density lipoproteins (VLDL) and accumulate chylomicrons within the bloodstream, which can cause abdominal pain, an enlarged spleen and liver, eruptive xanthomas and potentially lethal pancreatitis. After having evaluated the results of three clinical trials on 27 LPLD patients provided by Amsterdam Molecular Therapeutics, the EMA’s CHMP said that the firm did not provide sufficient evidence of persistence in lowering blood fats in a clinically relevant manner and that there were too few patients for whom sufficiently long-term data were available. Additionally, the Committee said there was also insufficient evidence of a reduction in the rate of pancreatitis. In the current stage, the data would be not sufficient to outweigh the risks associated with injection of the non-replicating, non-integrating adeno-associated virus carrying LPLS447X, a gain of function mutation of LPL. Additionally, it would not outweigh the risks associated with immunosuppression administered to boost tolerance of the therapy. Amsterdam Molecular Therapeutics said it believes that Glybera can receive a positive opinion subject to generating additional data from existing patients. The firm asked the EMA to re-examine the clinical data package. The company announced it will initiate a study to examine further how already treated patients handle chylomicrons over time. In addition, AMT will continue to collect follow up data on pancreatitis incidence.



Brussels – Promethera Biosciences has completed a Series B financing round, generating proceeds of €23.6m. The Belgian cell therapy company secured a capital increase of €17m from its new investors, among them the venture arms...



Leuven – Biopharmaceutical company ThromboGenics has entered into an agreement with Alcon Inc., a division of Novartis for the commercialisation of its product ocriplasmin in all markets outside the U.S. ThromboGenics said it...



Amsterdam Molecular Therapeutics has announced a financial manoeuvre aimed at getting the biotech out of a hopeless situation. The assets and – more importantly – the liabilities of the drug developer will be acquired by a newly...



Amsterdam - Amsterdam Molecular Therapeutics has announced a financial manoeuvre which is aimed to get the biotech out of a dead end. The assets and – more importantly – the liabilities of the drug developer will be acquired by...



Glasgow/Leuven - With a little help from the EU, two biotech companies are banding together to develop the first stem cell therapy against rheumatoid arthritis. UK-based Sistemic will work with TiGenix to develop the Belgian's...



Amsterdam – Two elephants in the biofuels business join forces. Dutch food and chemicals group Royal DSM and POET, LLC, a US-based ethanol producer, announced a plan to build a commercial cellulosic ethanol plant. POET–DSM...



Heerlen - Royal DSM N.V will be the industrial partner to the Panoptes consortium, which aims to develop novel drug delivery technology for the posterior eye segment. The four year project is supported by EUR4m in funding from...



Leiden - Dutch Pharming Group NV has taken a further step to secure sound financing even beyond the recent milestone payments. The drug developer announced that it has entered into a financing of approximately €8.4 million by...



Ghent – Just days after US pharmaceuticals giant Pfizer pulled out of a rheumatoid arthritis pact with Ablynx NV, the Belgian biopharmaceutical company expanded its relationship with Merck Serono. Ablynx has now entered into a...

Displaying results 1 to 10 out of 304

1-10 Next >

© 2007-2015 BIOCOM


Product of the week



All Events

Stock list

All quotes


  • THERAMETRICS (CH)0.07 CHF16.67%
  • MEDIGENE (D)9.35 EUR10.65%


  • CHRONTECH PHARMA (S)0.01 SEK-50.00%
  • NORDIC NANOVECTOR (N)24.40 NOK-9.29%
  • EVOTEC (D)3.33 EUR-6.20%


  • DBV Technologies (F)55.76 EUR31.0%
  • VERONA PHARMA (UK)5.20 GBP24.7%
  • MEDIGENE (D)9.35 EUR20.2%


  • CHRONTECH PHARMA (S)0.01 SEK-50.0%
  • NORDIC NANOVECTOR (N)24.40 NOK-30.5%
  • TRANSGENE (F)3.75 EUR-24.2%


  • ADOCIA (F)69.25 EUR398.9%
  • 4SC (D)4.88 EUR369.2%
  • WILEX (D)3.54 EUR321.4%


  • CHRONTECH PHARMA (S)0.01 SEK-87.5%
  • ACTIVE BIOTECH (S)9.20 SEK-68.1%
  • NEOVACS (F)1.13 EUR-66.9%

No liability assumed, Date: 01.07.2015

Current issue

All issues