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Amicus (FOLD) Gets Pompe Disease Treatment Approval in Europe
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Amicus (FOLD - Free Report) announced that the European Commission approved cipaglucosidase alfa, commercialized under the brand name Pombiliti, for treating late-onset Pompe disease (LOPD) in adults. Cipaglucosidase alfa is a long-term enzyme replacement therapy (ERT), used in combination with miglustat, a stabilizer of the engineered enzyme.
An approval was mostly expected as the Committee for Medicinal Products for Human Use had previously granted a positive opinion on the same.
The 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.
Shares of Amicus have gained 12% in the past year against the industry’s 16.5% decline.
Image Source: Zacks Investment Research
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. In 2019, the FDA granted Breakthrough Therapy designation to AT-GAA – the first ever granted to an investigational product for Pompe disease.
Given the approval, it presents a significant commercial opportunity for Amicus. Management will be moving forward with the launch of Pombiliti in Europe.
The company 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 Amicus’ inability to complete the manufacturing facility inspection before the PDUFA action date.
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.
Loss per share estimates for CRISPR Therapeutics have narrowed from $8.21 to $7.54 for 2023 in the past 60 days.
CRSP's earnings beat estimates in two of the last four quarters and missed the mark in the other two, the average surprise being 3.19%. CRSP’s shares have plunged 29.4% in the past year.
Loss per share estimates for Kala Pharmaceuticals have narrowed from $19.67 to $15.35 for 2023 and from $14.41 to $13.12 for 2024 in the past 60 days. KALA’s shares have plunged 82.7% in the past year.
KALA’s earnings beat estimates in two of the last four quarters and missed the mark in the other two, the average surprise being 11.56%.
Loss per share estimates for Jasper Therapeutics have narrowed from $1.42 to 61 cents for 2023 and from $1.6 to 59 cents for 2024 in the past 60 days. JSPR's shares have plunged 46.9% in the past year.
JSPR's earnings beat estimates in three of the last four quarters and met the mark in one, the average surprise being 3.51%.
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Amicus (FOLD) Gets Pompe Disease Treatment Approval in Europe
Amicus (FOLD - Free Report) announced that the European Commission approved cipaglucosidase alfa, commercialized under the brand name Pombiliti, for treating late-onset Pompe disease (LOPD) in adults. Cipaglucosidase alfa is a long-term enzyme replacement therapy (ERT), used in combination with miglustat, a stabilizer of the engineered enzyme.
An approval was mostly expected as the Committee for Medicinal Products for Human Use had previously granted a positive opinion on the same.
The 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.
Shares of Amicus have gained 12% in the past year against the industry’s 16.5% decline.
Image Source: Zacks Investment Research
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. In 2019, the FDA granted Breakthrough Therapy designation to AT-GAA – the first ever granted to an investigational product for Pompe disease.
Given the approval, it presents a significant commercial opportunity for Amicus. Management will be moving forward with the launch of Pombiliti in Europe.
The company 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 Amicus’ inability to complete the manufacturing facility inspection before the PDUFA action date.
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.
Amicus Therapeutics, Inc. Price and Consensus
Amicus Therapeutics, Inc. price-consensus-chart | Amicus Therapeutics, Inc. Quote
Zacks Rank & Stocks to Consider
Currently, Amicus has a Zacks Rank #3 (Hold).
Some better-ranked stocks in the same sector are CRISPR Therapeutics (CRSP - Free Report) , Kala Pharmaceuticals (KALA - Free Report) and Jasper Therapeutics (JSPR - Free Report) , 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.
Loss per share estimates for CRISPR Therapeutics have narrowed from $8.21 to $7.54 for 2023 in the past 60 days.
CRSP's earnings beat estimates in two of the last four quarters and missed the mark in the other two, the average surprise being 3.19%. CRSP’s shares have plunged 29.4% in the past year.
Loss per share estimates for Kala Pharmaceuticals have narrowed from $19.67 to $15.35 for 2023 and from $14.41 to $13.12 for 2024 in the past 60 days. KALA’s shares have plunged 82.7% in the past year.
KALA’s earnings beat estimates in two of the last four quarters and missed the mark in the other two, the average surprise being 11.56%.
Loss per share estimates for Jasper Therapeutics have narrowed from $1.42 to 61 cents for 2023 and from $1.6 to 59 cents for 2024 in the past 60 days. JSPR's shares have plunged 46.9% in the past year.
JSPR's earnings beat estimates in three of the last four quarters and met the mark in one, the average surprise being 3.51%.