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news.Amicus Therapeutics has submitted a marketing authorization application (MAA) to the EU seeking full approval for the oral small molecule pharmacological chaperone Galafold (migalastat HCl) to treat Fabry patients who have amenable genetic mutations.
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Both the European Medicines Agency (EMA) and the US Food and Drug Administration (FDA) have approved the brand name Galafold.
Amicus Therapeutics chairman and CEO John Crowley said: "The MAA submission for Galafold is the first marketing application for Amicus."
"Our goal now is crystal clear: to bring this novel small molecule, orally bioavailable personalized medicine to as many people living with this devastating genetic disease as quickly as possible."
Galafold has secured accelerated assessment in the EU, under which the EMA’s Committee for Medicinal Products for Human Use (CHMP) may shorten the MAA review period from 210 days, under standard review, to 150 days under Accelerated Assessment.
The company intends to submit a new drug application (NDA) for Galafold in the US under Subpart H1 in the second half of this year.
Currently, around 90 individuals across the world are being treated with Galafold as their only therapy for Fabry disease in ongoing long-term extension studies.
Earlier, the company had reported positive Phase III data for Galafold in both treatment naive and enzyme replacement therapy (ERT) switch patients.
The results showed that treatment with Galafold has resulted in reductions in disease substrate, stability of kidney function, reduction in cardiac mass, and a positive impact on patient-reported outcomes in patients with amenable mutations.