Shares of Amicus Therapeutics (FOLD) soared 20.4% after the company announced positive data from its second phase III study (Study 012) on the oral small molecule chaperone migalastat HCl in Fabry patients with amenable mutations.

The phase III study is evaluating the use of oral migalastat to standard-of-care enzyme replacement therapies (ERTs) − Sanofi’s (SNY) Fabrazyme and Shire’s (SHPG) Replagal − for Fabry disease. Eighteen-month data from the phase III study revealed that migalastat successfully met both the primary endpoints of comparability to ERT on key measures of kidney function.

Additionally, the candidate demonstrated low and stable plasma lyso-Gb3 levels in patients who switched from ERT to migalastat.

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In April this year, Amicus had announced positive results from its first phase III study (Study 011) on migalastat. Shares at that time had gained over 20% on the news. Migalastat had shown a statistically significant reduction in kidney interstitial capillary GL-3 at month 12 as compared to placebo.

Amicus plans to submit the marketing application for migalastat next year. We are encouraged by the positive data on migalastat considering that it is one of the key candidates at Amicus. We believe that post approval the drug could capture ERT’s market share which is valued at over $1 billion. Investor focus is expected to remain on migalastat going forward.

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Amicus’ (FOLD) shares jumped 20.4% with the company reporting positive data on its experimental Fabry disease treatment, migalastat. Although treatments for this rare inherited disease are already available, migalastat’s oral dosing regimen could prove to be a more convenient treatment option for patients

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