AMT Receives Orphan Drug Designation From the U.S. Food and Drug Administration Duchenne Muscular Dystrophy Gene Therapy

AMSTERDAM, September 24, 2010 /PRNewswire-FirstCall/ -- Amsterdam Molecular Therapeutics (AMT) Holding N.V. (Euronext: AMT), a leader in the field of human gene therapy, announced today that the U.S. Food and Drug Administration (FDA) has designated AMT-080, a gene therapy for Duchenne muscular dystrophy (DMD) as an orphan drug. In October 2009, the Committee for Orphan Medical Products of the European Medicines Agency granted AMT-080 orphan designation for the same indication in the European Union.

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This one sounds wonderful. But with them planning clinical trials to start late 2012 I'm not sure who we will lose between now and then. Plus, I don't understand something, this article stated AMT has already done clinical trials, so why the wait for further trials? Can't FDA accept the info from the first batch of clinical trials held outside the USA? Orphan Status is nice but I really don't have any faith that that status makes things move faster...Maybe I should be packing for Amsterdam...

Thanks Raktim for finding this and sharing it though!!
I don't think they did any clinical trials in DMD, only preclinical.
Yes, I re-read it and you are right Ofelia. And furthermore, it appears as though this is a one shot "treatment", ie it seems as though one treatment fixes things for good. No lifetime treatments involved. Certainly worth waiting for.

Ofelia Marin said:
I don't think they did any clinical trials in DMD, only preclinical.
You are welcome Cheryl.
My understanding is as of now, AMT-080 gene therapy focusses on exon 51 and improves the delivery efficiency( One shot treatment).
Last november, I had received this information from AMT
Our hope is that with our gene therapy product we can help DMD patients for a long time with one administration of the product. AMT has started with the preclinical studies of this gene therapy programme, after which testing in humans can begin. Therefore, we do not have a cure for DMD available yet and it will take some time before such a solution will in practice be available. Our gene therapy is currently focused on exon 51 only, however the technique might be generalised to less frequent and multiple mutations in the future.

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