Amicus (FOLD) Gets Approval in Europe for Pompe Disease Drug
Amicus Therapeutics FOLD announced that the European Commission approved miglustat 65 mg capsules, under the brand name Opfolda, for treating late-onset Pompe disease (LOPD) in adults. Miglustat is an enzyme stabilizer component, used in combination with Pombiliti (cipaglucosidase alfa) — a long-term enzyme replacement therapy (ERT).
Approval was expected in April 2023 as the Committee for Medicinal Products for Human Use had previously granted a positive opinion on the same. Pombiliti received approval from European Commission (EC) in March 2023.
Two components of Amicus’ lead pipeline AT-GAA, developed for treating Pompe disease, are Cipaglucosidase alfa and miglustat.
The approval presents a significant commercial opportunity for FOLD. Management will move forward with the launch of Pombiliti + Opfolda in Europe.
Shares of Amicus have nosedived 0.3% year to date compared with the industry’s 3.5% decline.
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The EC approval was based on positive data from the phase III PROPEL study, the only randomized, controlled trial in LOPD. The study included patients in the high unmet need ERT-experienced population, in addition to ERT-naïve patients.
Pompe disease is a lysosomal disorder, resulting from the deficiency of the enzyme acid alpha-glucosidase (GAA). The disease is characterized by severe muscle weakness that worsens over time.
Amicus is also working with the FDA to get biologics license application (BLA) approval for cipaglucosidase alfa in the United States. Earlier in October 2022, the FDA had deferred action on the BLA for cipaglucosidase alfa, citing FOLD’s inability to complete the manufacturing facility inspection before the PDUFA action date.
In the second quarter of 2023, the FDA completed the required pre-approval inspection of the WuXi Biologics manufacturing site in China.
Management expects regulatory approval of AT-GAA in the U.K. and the United States in the third quarter of 2023.
FOLD currently has one marketed medicine in its portfolio, Galafold (migalastat), the first and only approved oral precision medicine for patients with Fabry disease with amenable genetic variants. In the first quarter of 2023, Galafold generated revenues of $86.3 million.
Amicus Therapeutics, Inc. Price and Consensus
Amicus Therapeutics, Inc. price-consensus-chart | Amicus Therapeutics, Inc. Quote
Zacks Rank & Stocks to Consider
Amicus currently carries a Zacks Rank #3 (Hold).
Some better-ranked stocks in the overall healthcare sector are Novartis NVS, Akero Therapeutics AKRO and Omega Therapeutics OMGA, each carrying a Zacks Rank #2 (Buy) at present. You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
The Zacks Consensus Estimate for Novartis’ 2023 earnings has gone up from $6.56 per share to $6.74 in the past 90 days. Shares of Novartis have risen 9.3% year to date.
NVS’ earnings beat estimates in each of the trailing four quarters, delivering an average surprise of 5.15%.
The consensus estimate for Akero Therapeutics has narrowed from a loss of $2.96 per share to a loss of $2.80 for 2023 in the past 90 days. Shares of Akero Therapeutics have nosedived 17.1% year to date.
AKRO’s earnings beat estimates in each of the trailing four quarters, delivering an average surprise of 19.13%.
The consensus mark for Omega Therapeutics has narrowed from a loss of $2.49 per share to a loss of $2.05 for 2023 in the past 90 days. Shares of the company have rallied 5% year to date.
OMGA’s earnings beat estimates in two of the trailing four quarters, met the mark in one and missed in another, delivering an average surprise of 8.24%.
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