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news.The European Medicines Agency (EMA) has validated Amicus Therapeutics' marketing authorization application (MAA) and started centralized procedure for the oral small molecule pharmacological chaperone, Galafold (migalastat HCl), designed to treat Fabry patients who have amenable genetic mutations.
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Galafold is the first investigational Fabry drug to be granted accelerated assessment in the European Union (EU).
In the US, the company plans to carry out a pre-NDA meeting with the US Food and Drug Administration (FDA) and to submit a new drug application (NDA) for Galafold under Subpart H1 in the second half of this year.
Following the MAA validation, the company is also starting the regulatory submission process in several additional geographies.
Amicus Therapeutics chairman and chief executive officer John Crowley said: "With a majority of Fabry patients residing outside the United States, the MAA validation also allows us to prepare for the regulatory process in additional geographies where the MAA may serve as the basis for submission.
"In the United States, we remain on track to hold a pre-NDA meeting with the FDA and to submit an NDA for Galafold under Subpart H in the second half of 2015.
"Our global strategy is to bring this novel orally bioavailable personalized medicine to as many people living with Fabry disease as quickly as possible."
Under accelerated assessment, the EMA’s Committee for Medicinal Products for Human Use (CHMP) may shorten the MAA standard review period from 210 days to 150 days.
Currently, around 90 individuals around the world are being treated with Galafold as their only therapy for Fabry disease in ongoing long-term extension trials.