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Amicus Therapeutics FOLD announced that the European Medicines Agency (“EMA”) has validated the marketing authorization applications (MAA) for AT-GAA, the company’s two investigational two-component therapy for the treatment of Pompe disease.
The EMA’s validation of MAA confirms that its filings for AT-GAA have been accepted. Following validation, the EMA’s centralized procedure with Committee for Medicinal Products for Human Use’s (CHMP) assessment begins.
Amicus Therapeutics’ stock has plunged 54.7% this year so far in comparison with the industry’s 21.7% rise.
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The EMA’s acceptance of the MAAs is based on data from phase I/II and phase III PROPEL studies as well as the long-term open-label extension study evaluating AT-GAA in adults with Pompe disease.
Pompe Disease is a debilitating disease marked by severe muscle weakness that worsens over time. It is caused by a deficiency of the enzyme acid alpha-glucosidase (GAA) whose reduced or absent levels lead to accumulation of glycogen in cells, resulting in clinical manifestations of the indication. Per management, an estimated 5,000-10,000 people worldwide are affected by the disease.
AT-GAA consists of two components – cipaglucosidase alfa, a recombinant human acid alpha-glucosidase (rhGAA) enzyme with optimized carbohydrate structures, particularly bis-phosphorylated mannose-6 phosphate (bis-M6P) glycans, to enhance uptake into cells; and miglustat, a stabilizer of cipaglucosidase alfa.
In September 2021, Amicus announced that the FDA’s acceptance of the company’s biologics license application (BLA) for cipaglucosidase alfa and new drug application (NDA) for miglustat for AT-GAA for the treatment of Pompe disease. The FDA has set target action dates of May 29, 2022 for the NDA and Jul 29, 2022 for the BLA. The FDA has also granted the Breakthrough Therapy designation (“BTD”) to AT-GAA for the indication.
Currently, Amicus has only one marketed medicine in its portfolio, Galafold, approved for patients living with Fabry disease, having amenable genetic variants.
If approved, AT-GAA will face stiff competition from pharma giant Sanofi SNY, which already has two approved products for Pompe disease. Sanofi’s Myozyme/Lumizyme (alglucosidase alfa) is an enzyme replacement therapy approved to treat both Infantile Onset and Late Onset Pompe disease (IOPD and LOPD).
Earlier in August, Sanofi announced that the FDA has granted approval to its enzyme replacement therapy, avalglucosidase alfa-ngpt, for the treatment of LOPD. The drug is marketed under the trade name Nexviazyme.
Sanofi also markets Fabrazyme, an enzyme replacement therapy approved for the treatment of Fabry disease.
Zacks Rank & Stocks to Consider
Amicus currently carries a Zacks Rank #3 (Hold). A couple of better-ranked stocks in the overall healthcare sector include Endo International ENDP and Precision BioSciences DTIL, each sporting a Zacks Rank #1 (Strong Buy) at present. You can see the complete list of today’s Zacks #1 Rank stocks here.
Endo International’s earnings per share estimates for 2021 have increased from $2.32 to $2.84 in the past 30 days. The same for 2022 has increased from $2.25 to $2.47 in the past 30 days.
Earnings of Endo International beat estimates in all the last four quarters, with the average being 57.7%.
Precision BioSciences’ loss per share estimates for 2021 have narrowed from $1.17 to $0.65 in the past 30 days. The same for 2022 has narrowed from $2.39 to $1.91 in the past 30 days.
Earnings of Precision BioSciences beat estimates in all the last four quarters, delivering a surprise of 76.9%, on average.
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