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Amicus Therapeutics Inc's Business Segments
Amicus Therapeutics Inc's reported revenue by business segment and by geographic region, quarterly and annual, normalized against the consolidated income statement. Free below: the top 3 rows per table, this quarter and this fiscal year. Subscriber access adds the full segment history and operating income by segment.
Segment Data As of FY
Reportable Segments
4
Largest Segment
Galafold
Total Revenue
$ 633
Regions Reported
2
Revenue Share by Reportable Segment - FY
- Galafold82.1%
- Non-US Galafold48.1%
- Pombiliti Opfolda18.2%
- Non-US Pombiliti Opfolda10.5%
Revenue by Reportable Segment - FY
| Segment | Revenue (Millions) | % of Total |
|---|---|---|
| Galafold | $ 138 | 82.1% |
| Non-US Galafold | $ 81 | 48.1% |
| Pombiliti Opfolda | $ 31 | 18.2% |
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Revenue Share by Region - FY
- United States Galafold33.9%
- United States Pombiliti Opfolda7.8%
Revenue by Geographic Region - FY
| Region | Revenue (Millions) | % of Total |
|---|---|---|
| United States Galafold | $ 57 | 33.9% |
| United States Pombiliti Opfolda | $ 13 | 7.8% |
Annual Results
Revenue by Product & Service Category - FY
- Galafold34.1%
- Pombiliti + Opfolda7.6%
Revenue by Product & Service Category - FY
| Category | Revenue (Millions) | % of Total |
|---|---|---|
| Galafold | $ 216 | 34.1% |
| Pombiliti + Opfolda | $ 48 | 7.6% |
Product and service categories are a supplemental disclosure and are not required to sum to consolidated revenue or to the reportable segments above.
Description of Amicus Therapeutics Inc
Amicus Therapeutics is an American biopharmaceutical company founded in 2002, based in Cranbury, New Jersey. The company is focused on developing and commercializing new treatments for rare and orphan diseases, including lysosomal storage disorders (LSDs) of genetic origin.
The company's mission is to unlock the potential of genetic medicine to improve the lives of people affected by rare and devastating diseases. They are dedicated to conducting rigorous research to uncover new therapeutic approaches to these diseases, and to bringing safe and effective treatments to market.
Amicus Therapeutics has a diverse portfolio of small molecule and gene therapy drug candidates that are designed to address various LSDs. They currently have three approved products: Galafold, a small molecule medication for the treatment of Fabry disease, which is a genetic disorder that leads to the accumulation of a type of fat in the body called globotriaosylceramide (Gb3); Migalastat, an oral medication that is used in combination with enzyme replacement therapy (ERT) for patients with Fabry disease; and Jivi, a recombinant factor VIII therapy used for the treatment of hemophilia A.
In addition to their approved products, Amicus Therapeutics also has several promising drug candidates in development, such as AT-GAA, a gene therapy for Pompe disease that is currently in Phase 3 clinical trials.
Amicus Therapeutics has a strong commitment to patient advocacy and engagement, working closely with patient organizations and advocacy groups to ensure that the needs and concerns of patients are taken into account in their research and development efforts.
Overall, Amicus Therapeutics is a leading company in the field of rare and orphan diseases, with a strong focus on developing innovative and effective treatments to improve the lives of patients affected by these devastating conditions.
The company's mission is to unlock the potential of genetic medicine to improve the lives of people affected by rare and devastating diseases. They are dedicated to conducting rigorous research to uncover new therapeutic approaches to these diseases, and to bringing safe and effective treatments to market.
Amicus Therapeutics has a diverse portfolio of small molecule and gene therapy drug candidates that are designed to address various LSDs. They currently have three approved products: Galafold, a small molecule medication for the treatment of Fabry disease, which is a genetic disorder that leads to the accumulation of a type of fat in the body called globotriaosylceramide (Gb3); Migalastat, an oral medication that is used in combination with enzyme replacement therapy (ERT) for patients with Fabry disease; and Jivi, a recombinant factor VIII therapy used for the treatment of hemophilia A.
In addition to their approved products, Amicus Therapeutics also has several promising drug candidates in development, such as AT-GAA, a gene therapy for Pompe disease that is currently in Phase 3 clinical trials.
Amicus Therapeutics has a strong commitment to patient advocacy and engagement, working closely with patient organizations and advocacy groups to ensure that the needs and concerns of patients are taken into account in their research and development efforts.
Overall, Amicus Therapeutics is a leading company in the field of rare and orphan diseases, with a strong focus on developing innovative and effective treatments to improve the lives of patients affected by these devastating conditions.
Amicus Therapeutics Inc. is a biopharmaceutical company dedicated to the development and commercialization of innovative therapies for rare and orphan diseases. The organization’s mission is to provide hope and improved quality of life for patients suffering from these challenging conditions through cutting-edge research, development efforts, and supportive patient services.
Segments:
1. Research and Development:
Amicus Therapeutics emphasizes extensive investment in research and development (R&D) to discover and address unmet medical needs associated with rare diseases. The R&D pipeline includes potential therapies for lysosomal storage disorders, which are genetic conditions that result in the accumulation of toxic materials in cells. Key areas of focus include:
- Fabry Disease: An inherited condition caused by the absence or malfunction of the enzyme alpha-galactosidase A.
- Pompe Disease: A rare genetic disorder characterized by the buildup of glycogen in the body’s cells due to an enzyme deficiency.
- Mucopolysaccharidosis Type IIIA: A rare lysosomal storage disorder leading to the accumulation of complex carbohydrates in the body.
2. Commercialization:
This segment is critically focused on the market introduction and sustainability of Amicuss therapies. The commercialization team collaborates with healthcare providers, patients, and payers to facilitate access to treatments, manage resources effectively, and ensure patient education. The company actively pursues international approvals and market expansion for its therapies to maximize reach and patient impact.
Products:
1. Galafold:
Galafold (Migalastat) is an oral medication specifically approved for the treatment of Fabry disease in patients who have a specific genetic mutation. By stabilizing the mutated form of the enzyme α-galactosidase A, it aids in the effective breakdown of certain fats that accumulate due to enzyme deficiency. The unique oral formulation provides an alternative treatment option compared to traditional infusions and has shown to improve renal function and cardiac health among its users.
2. Migalastat:
While the name is often interchangeably used with Galafold, it specifically refers to the investigational aspect before obtaining approval for the marketed product. As an oral pharmacological chaperone, Migalastat enhances the processing of enzymes in patients with Fabry disease, enabling the mutant enzymes to function more effectively and reduce glycosphingolipid accumulation, which can lead to severe health complications.
3. AT-GAA:
AT-GAA is an investigational therapy designed for the treatment of Pompe disease. It acts by providing a recombinant form of the enzyme acid α-glucosidase, which is deficient in Pompe patients. By restoring the activity of this enzyme, AT-GAA aims to decrease glycogen accumulation in muscle tissues, potentially enhancing muscle strength and overall function, and improving the patients quality of life.
Services:
1. Patient Support:
Amicus Therapeutics recognizes the importance of comprehensive support for patients and their families. The company provides patient support programs that include genetic counseling, educational resources, and advocacy initiatives. These programs are aimed at empowering patients with knowledge about their disorders and treatment options, as well as connecting them to communities of care.
2. Research Collaboration:
In its endeavor to discover and develop groundbreaking therapies, Amicus Therapeutics collaborates with academic institutions, industry partners, and health systems. These partnerships enhance research capabilities and accelerate innovative solutions for rare diseases, with a focus on improving patient outcomes.
3. Clinical Trials:
Committed to maintaining rigorous standards of safety and efficacy, Amicus Therapeutics conducts comprehensive clinical trials to evaluate the effectiveness of its therapies. The company coordinates with clinical trial sites across various geographies, ensuring ethical practices and patient safety while also providing opportunities for eligible patients to participate in trials that may offer access to novel therapies that are not yet widely available.
Overall, Amicus Therapeutics Inc. is committed to the advancement of treatment options for patients with rare diseases through its focused segments on R&D and commercialization, innovative product offerings, and dedicated services aimed at patient support and education. This multi-faceted approach positions the company as a leader in the biopharmaceutical industry focused on enhancing the lives of individuals affected by rare conditions.
Segments:
1. Research and Development:
Amicus Therapeutics emphasizes extensive investment in research and development (R&D) to discover and address unmet medical needs associated with rare diseases. The R&D pipeline includes potential therapies for lysosomal storage disorders, which are genetic conditions that result in the accumulation of toxic materials in cells. Key areas of focus include:
- Fabry Disease: An inherited condition caused by the absence or malfunction of the enzyme alpha-galactosidase A.
- Pompe Disease: A rare genetic disorder characterized by the buildup of glycogen in the body’s cells due to an enzyme deficiency.
- Mucopolysaccharidosis Type IIIA: A rare lysosomal storage disorder leading to the accumulation of complex carbohydrates in the body.
2. Commercialization:
This segment is critically focused on the market introduction and sustainability of Amicuss therapies. The commercialization team collaborates with healthcare providers, patients, and payers to facilitate access to treatments, manage resources effectively, and ensure patient education. The company actively pursues international approvals and market expansion for its therapies to maximize reach and patient impact.
Products:
1. Galafold:
Galafold (Migalastat) is an oral medication specifically approved for the treatment of Fabry disease in patients who have a specific genetic mutation. By stabilizing the mutated form of the enzyme α-galactosidase A, it aids in the effective breakdown of certain fats that accumulate due to enzyme deficiency. The unique oral formulation provides an alternative treatment option compared to traditional infusions and has shown to improve renal function and cardiac health among its users.
2. Migalastat:
While the name is often interchangeably used with Galafold, it specifically refers to the investigational aspect before obtaining approval for the marketed product. As an oral pharmacological chaperone, Migalastat enhances the processing of enzymes in patients with Fabry disease, enabling the mutant enzymes to function more effectively and reduce glycosphingolipid accumulation, which can lead to severe health complications.
3. AT-GAA:
AT-GAA is an investigational therapy designed for the treatment of Pompe disease. It acts by providing a recombinant form of the enzyme acid α-glucosidase, which is deficient in Pompe patients. By restoring the activity of this enzyme, AT-GAA aims to decrease glycogen accumulation in muscle tissues, potentially enhancing muscle strength and overall function, and improving the patients quality of life.
Services:
1. Patient Support:
Amicus Therapeutics recognizes the importance of comprehensive support for patients and their families. The company provides patient support programs that include genetic counseling, educational resources, and advocacy initiatives. These programs are aimed at empowering patients with knowledge about their disorders and treatment options, as well as connecting them to communities of care.
2. Research Collaboration:
In its endeavor to discover and develop groundbreaking therapies, Amicus Therapeutics collaborates with academic institutions, industry partners, and health systems. These partnerships enhance research capabilities and accelerate innovative solutions for rare diseases, with a focus on improving patient outcomes.
3. Clinical Trials:
Committed to maintaining rigorous standards of safety and efficacy, Amicus Therapeutics conducts comprehensive clinical trials to evaluate the effectiveness of its therapies. The company coordinates with clinical trial sites across various geographies, ensuring ethical practices and patient safety while also providing opportunities for eligible patients to participate in trials that may offer access to novel therapies that are not yet widely available.
Overall, Amicus Therapeutics Inc. is committed to the advancement of treatment options for patients with rare diseases through its focused segments on R&D and commercialization, innovative product offerings, and dedicated services aimed at patient support and education. This multi-faceted approach positions the company as a leader in the biopharmaceutical industry focused on enhancing the lives of individuals affected by rare conditions.