Amicus Therapeutics, Inc. (FOLD - Free Report) issued an update on the regulatory pathway for migalastat for the treatment of Fabry disease in the U.S. Subsequent to several collaborative discussions with the FDA, including the receipt of final written minutes from an in-person type B meeting, the company has decided to collect additional data on gastrointestinal (GI) symptoms in Fabry patients who have an amenable mutation.
Additional data is expected to support full approval of migalastat for Fabry disease in the U.S.
Amicus will conduct a randomized, 12-month, placebo-controlled, pivotal, cross-over study in treatment-naïve Fabry patients (n=35) who have an amenable mutation and GI symptoms. The primary endpoint of the study will be diarrhea based on the FDA’s established irritable bowel syndrome guidance. An intermediate expanded access program (EAP) to warrant short-term access to migalastat for patients in the U.S. who are currently on enzyme replacement therapy is expected to begin in 2017.
Amicus is working with the FDA to finalize the study protocol, with enrollment expected to begin in 2017 and data anticipated in 2019. A new drug application filing will be based upon these new data and the totality of data from all prior studies on migalastat.
On the flip side, the FDA has declared that kidney globotriaosylceramide reduction cannot be considered as the basis for accelerated approval under Subpart H. Amicus’ share price tanked 26.7% on the news in afterhours trading yesterday.
We note that migalastat gained full approval in the EU under the trade name Galafold in May 2016 as a first-line therapy for the long-term treatment of adults and adolescents aged 16 years and older with a confirmed diagnosis of Fabry disease, who have an amenable mutation. The company expects the EU approval to serve as the basis for regulatory approvals in more than two-thirds of the global Fabry market outside the U.S.
Meanwhile, outside the U.S., Amicus has initiated the commercial launch of Galafold in Germany and is undergoing the EU country-by-country processes to launch in the majority of EU countries throughout 2016 and 2017. The company has also commenced intermediate expanded access programs (EAP) in the EU and other territories outside the U.S.
Zacks Rank & Key Picks
Amicus currently carries a Zacks Rank #3 (Hold). Some better-ranked stocks in the health care sector include Heska Corp. (HSKA - Free Report) , Cambrex Corp. (CBM - Free Report) and Vanda Pharmaceuticals, Inc. (VNDA - Free Report) . Each of these stocks sports a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
Heska’s earnings estimates have increased from $1.13 to $1.35 for 2016 and from $1.38 to $1.53 for 2017 over the last 60 days. The company has posted a positive earnings surprise in all of the four trailing quarters with an average beat of 301.64%. Its share price has increased 69.6% year to date.
Cambrex’s earnings estimates increased from $2.46 to $2.55 for 2016 and from $2.91 to $3.06 for 2017 over the last 60 days. The company posted a positive earnings surprise in three of the four trailing quarters with an average beat of 19.78%. Its share price has increased 4.3% year to date.
Vanda’s loss estimates have narrowed from 68 cents to 56 cents for 2016 while its earnings estimates have increased from 16 cents to 17 cents for 2017 over the last 60 days. The company has posted a positive earnings surprise in three of the four trailing quarters with an average beat of 56.65%. Its share price surged 83.7% year to date.
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