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Amicus (FOLD) Initiates Rolling BLA Submission for AT-GAA

Amicus Therapeutics Ltd. FOLD initiated a rolling biologics license application (BLA) submission to the FDA for its investigational two-component therapy,AT-GAA, for the treatment of late onset Pompe disease (LOPD).Pompe disease is an inherited lysosomal disorder caused by the deficiency of the enzyme acid alpha-glucosidase (GAA).

Shares of the company have surged 132.4% year to date compared with the industry’s growth of 4 7%.

 The rolling submission allows the company to submit portions of the regulatory application to the FDA for review as they are completed, rather than waiting until every section of the BLA is complete.

The company remains on track to complete the BLA submission in the first half of 2021.

AT-GAA is a novel treatment paradigm that consists of ATB200, a unique recombinant human acid alpha-glucosidase (rhGAA) enzyme to enhance uptake, co-administered with AT2221, which is a pharmacological chaperone. In February 2019, the FDA granted a Breakthrough Therapy designation (“BTD”) to AT-GAA. The candidate is the first-ever investigational product for Pompe disease to receive BTD.

Amicus is also conducting an ongoing phase III study (ATB200-03, or PROPEL) of AT-GAA in adult patients with LOPD. The study is designed to assess the efficacy, safety and tolerability of AT-GAA compared to the current standard of care, alglucosidase alfa, an enzyme replacement therapy (ERT). The primary endpoint is a six-minute-walk distance, an integrated measure of disease progression.

If approved,AT-GAA has the potential to become the new standard of care for individuals living with Pompe disease.

Zacks Rank & Stocks to Consider

Amicus currently carries a Zacks Rank #3 (Hold).

Some better-ranked stocks in the healthcare sector include ASLAN Pharmaceuticals Ltd. ASLN, Aptose Biosciences Inc. APTO and Alimera Sciences Inc. ALIM. All of them carry a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

ASLAN’s loss per share estimates have narrowed from 42 cents to 40 cents for 2020 and from 60 cents to 57 cents for 2021 over the past 60 days.

Aptose’s loss per share estimates have narrowed from 77 cents to 69 cents for 2020 and from 85 cents to 81 cents for 2021 over the past 60 days.

Alimera’s loss per share estimates have narrowed from $1.31 to 96 cents for 2020 over the past 60 days.


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