Amicus Therapeutics, Inc. FOLD announced that it recently met with regulatory authorities in Europe and the U.S. in order to discuss the approval pathways for pipeline candidate — oral small molecule pharmacological chaperone migalastat HCl.
The candidate is being developed as a monotherapy for Fabry patients who have amenable genetic mutations.
As per the discussions with the officials from the European Medicines Agency (EMA), Amicus may submit its marketing authorization application (MAA) for migalastat monotherapy under the full approval pathway in the EU.
As a result of positive feedback from the officials from the EMA, Amicus is accelerating the timing of the MAA submission for full approval for migalastat monotherapy from mid -2015 to the second quarter of 2015.
Meanwhile, Amicus also held a meeting with the FDA earlier in the week to review results from both phase III studies (Study 011 and Study 012) along with discussing the approval pathway for migalastat in the U.S.
Based on its meetings, Amicus plans to submit a new drug application (NDA) for accelerated approval to the FDA. Accelerated approval is generally granted to therapies for severe and life-threatening conditions that address significant unmet medical needs and migalastat has to meet quite a few endpoints (substrate reduction, reduction in cardiac mass, stabilization of kidney function) to be able to achieve so.
Amicus currently plans to schedule a pre-NDA meeting and consequently submit an NDA in the second half of 2015. The company is required to conduct a post-approval (phase IV) confirmatory study to be able to obtain approval under accelerated approval.
The pipeline progress at Amicus is encouraging. Migalastat is a key candidate in Amicus’ pipeline and hence we expect investor focus on regulatory filings for the candidate. Amicus does not have any approved product in its kitty. The company is also developing next-generation enzyme replacement therapy (ERT) products for Fabry disease, Pompe disease, and MPS-1.
We note that prescribed treatments for Fabry disease include Sanofi’s SNY Fabrazyme among others.
Amicus currently carries a Zacks Rank #3 (Hold). Some better-ranked stocks in the health care sector are ANI Pharmaceuticals, Inc. ANIP and Horizon Pharma plc HZNP. Both carry a Zacks Rank #1 (Strong Buy).
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