Amicus Therapeutics’ Fabry disease drug gets European regulatory support

pharmafile | April 4, 2016 | News story | Research and Development, Sales and Marketing Amicus Therapeutics, EMA, Fabry disease, genetic disorder, rare disease 

Shares in biotechnology firm Amicus Therapeutics (Nasdaq: FOLD) rose to close up nearly 3% after the European regulators announced backing for its drug to treat Fabry disease.  

The therapy called Galafold (migalastat) will provide additional treatment option to the rare genetic disorder, Fabry disease.

The European Medicines Agency (EMA) has recommended a marketing authorisation in the European Union (EU) for Galafold. The European Committee for Medicinal Products for Human Use has adopted a positive opinion to approve the company’s trial drug. A final decision from the European Commission is expected in the second quarter, the company said.

John Crowley, chief executive officer of Amicus Therapeutics, says: “Oral migalastat represents a ground breaking approach to personalized medicine, adding, “These amenable genetic mutations are represented in an estimated 35% to 50% of the diagnosed Fabry population today, which is a significant opportunity for Amicus to deliver a therapy option for many patients in need.”

In 2013, UK drug major GlaxoSmithKline (LSE: GSK) returned the rights to migalastat and dissolved its stake in Amicus after a failed late-stage study.

In October, the firm’s shares witnessed a sharp drop after it reported the filing for US marketing would be delayed for migalastat.

Fabry disease is an inherited lysosomal storage disorder caused by deficiency of an enzyme called alpha-galactosidase A (alpha-Gal A), which are the result of mutations in the GLA gene. The primary biological function of alpha-Gal A is to degrade specific lipids in lysosomes, including globotriaosylceramide (referred to here as GL-3 and also known as Gb3).

Reduced or absent levels of alpha-Gal A activity lead to the accumulation of GL-3 in the affected tissues, including the central nervous system, heart, kidneys, and skin. Progressive accumulation of GL-3 is believed to lead to the morbidity and mortality of Fabry disease, including pain, kidney failure, heart disease, and stroke. All Fabry disease is progressive and leads to organ damage regardless of the time of symptom onset.

Shares closed up nearly 3% at $8.69 Friday, on the Nasdaq.

Anjali Shukla

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