Keyword: Amicus Therapeutics
The study has generated early evidence that the gene therapy may stabilize the motor and language function of children with the rare genetic disorder.
Amicus plans to build an R&D center in the heart of Philadelphia’s University City, not long after inking a $100 million deal for 10 gene therapies.
Galafold is an oral drug versus the IV-infused Fabrazyme, making it more convenient for patients, although not every Fabry patient will be eligible.
The takeover gives Amicus a ready-made pipeline of 10 gene therapies, including two that are in clinical trials.
Amicus must now continue adding to its existing phase 1/2 data with a view to sitting down again with the agency next year.
Amicus needs to gather data on more patients and track the progress of previously treated people before seeking conditional approval.
A drug for rare skin disease epidermolysis bullosa that Amicus Therapeutics acquired in a $847 million deal 2 years ago has failed a key phase 3 trial.
Amicus can now submit its rare disease drug for review, a year after being blocked.
In a typically fiery speech, President Donald Trump in his first joint address to Congress last night tore into the FDA’s “slow and burdensome approval process,” and gave vague assurances that this would be “eliminated … so more lives could be saved.”
Amicus saw its shares down by 26% after news that its Fabry disease candidate migalastat did not meet the FDA’s requirements for a speedy review, and the biotech will also see any approval delayed until at least the end of the decade as it needs to create more data for the drug.