Amicus Therapeutics, Inc. - Common Stock (FOLD)
Frequently Asked Questions About Amicus Therapeutics, Inc. - Common Stock (FOLD)
How does Amicus Therapeutics approach research and development?
Amicus Therapeutics adopts a patient-focused approach in its research and development (R&D) initiatives. The company emphasizes collaboration with academic institutions, patient advocacy groups, and industry partners. Its R&D strategy is centered on innovation in drug development, employing advanced technologies to design therapies that effectively target specific genetic disorders.
How does Amicus Therapeutics support patients and healthcare providers?
Amicus Therapeutics is committed to supporting patients and healthcare providers through comprehensive educational programs, awareness campaigns, and resources. The company offers information on disease management, treatment options, and access programs to ensure that patients can benefit from available therapies. They also engage with healthcare professionals to provide guidance on the best practices for managing rare diseases.
How does Galafold work?
Galafold (migalastat) is an oral medication that acts as a pharmacological chaperone for individuals with Fabry disease. It works by stabilizing the alpha-galactosidase A enzyme, enabling it to function more effectively in breaking down glycosphingolipids. This helps alleviate the symptoms associated with the accumulation of these lipids in the body and is intended for patients who have a specific genetic mutation.
Is Amicus Therapeutics publicly traded?
Yes, Amicus Therapeutics is publicly traded on the Nasdaq under the ticker symbol 'FOLD.' The company went public in 2014, and being listed on a major stock exchange allows it to access capital markets for funding its research and development initiatives.
What are the challenges faced by Amicus Therapeutics?
Like many biotechnology firms, Amicus Therapeutics faces several challenges, including the complexities of drug development, competition within the biopharmaceutical industry, and navigating regulatory pathways. Additionally, securing funding for continued R&D and successfully bringing products to market while managing operational costs also presents ongoing challenges.
What are the key products developed by Amicus Therapeutics?
Amicus Therapeutics has developed several key products, including Galafold (migalastat) which is an oral therapy approved for the treatment of Fabry disease. In addition, the company is also working on AT-GAA, a dual-enzyme therapy for Pompe disease, which is currently in late-stage clinical development. They continually strive to expand their product pipeline for patients with rare genetic diseases.
What does Amicus Therapeutics, Inc. do?
Amicus Therapeutics, Inc. is a biotechnology company that focuses on developing innovative therapies for rare and devastating diseases, particularly those related to genetic disorders. Their primary focus is on the treatment of lysosomal storage disorders like Fabry disease and Pompe disease. They utilize next-generation pharmacological chaperone technology and other advanced approaches to address unmet medical needs.
What is Fabry disease?
Fabry disease is a rare genetic disorder caused by the deficiency of an enzyme called alpha-galactosidase A. This deficiency leads to the accumulation of certain types of fats in the body's cells, which can result in severe symptoms affecting multiple organs, including pain, kidney dysfunction, heart problems, and skin rashes. Early diagnosis and treatment are crucial to manage the disease and its complications.
What is Pompe disease?
Pompe disease is a rare inherited disorder caused by the deficiency of the enzyme acid alpha-glucosidase. This enzyme is essential for the breakdown of glycogen, leading to its accumulation and resulting in muscle weakness, respiratory issues, and progressive disability. The severity of Pompe disease varies widely, and it can manifest in infancy, childhood, or adulthood.
What is the financial outlook for Amicus Therapeutics?
The financial outlook for Amicus Therapeutics can fluctuate depending on factors such as clinical trial successes, regulatory approvals, and market performance. Analysts often review revenue forecasts based on product launches and adoption. Investors should stay informed about the company’s financial reports and operational progress to gauge its future performance.
What is the mission of Amicus Therapeutics?
The mission of Amicus Therapeutics is to develop and deliver innovative therapies for patients facing rare and devastating diseases. The company strives to transform the lives of patients by advancing research and creating effective treatments while maintaining a commitment to the highest standards in clinical development and patient care.
What is the significance of Amicus Therapeutics' pipeline?
The pipeline of Amicus Therapeutics is significant as it reflects the company's commitment to addressing unmet medical needs in rare diseases. By developing a range of therapies, including both enzyme replacement treatments and small-molecule pharmacological chaperones, Amicus aims to provide comprehensive care options for patients suffering from lysosomal storage disorders and expand its impact in the field of rare diseases.
What partnerships does Amicus Therapeutics have?
Amicus Therapeutics actively seeks partnerships and collaborations with other biopharmaceutical companies, academic institutions, and research organizations. These partnerships enhance their research capabilities, expand their pipeline, and help to accelerate the development and commercialization of their therapies for rare diseases.
What recent developments have occurred at Amicus Therapeutics?
Amicus Therapeutics remains active in advancing its clinical and commercial efforts. Recent developments include updates on ongoing clinical trials, new data on their pipeline products, potential new partnerships or collaborations, and regulatory submissions. Keeping abreast of these developments is essential for investors and stakeholders to understand the company's trajectory and impact.
What role do clinical trials play at Amicus Therapeutics?
Clinical trials at Amicus Therapeutics play a critical role in evaluating the safety and efficacy of new therapies. These trials allow the company to gather essential data on its products, as well as to ensure compliance with regulatory requirements. The trials are crucial for obtaining approvals from health authorities, thus enabling the company to bring new therapies to patients who need them.
When was Amicus Therapeutics founded?
Amicus Therapeutics was founded in 2002 by John F. Crowley, who was motivated by the struggles of his children, who suffered from Pompe disease. Since its founding, the company has been dedicated to advancing therapies for rare diseases and improving the lives of patients and families affected by these conditions.
Where is Amicus Therapeutics headquartered?
Amicus Therapeutics is headquartered in Cranbury, New Jersey, USA. This location is strategically chosen to leverage the region's strong biotechnology and pharmaceutical ecosystem, facilitating collaborations, research, and development efforts in the biotech arena.
Who are the key executives at Amicus Therapeutics?
The key executives at Amicus Therapeutics include John F. Crowley, who serves as the CEO and has been instrumental in the company's strategic vision and growth. Other executives typically include leaders in research, development, operations, and finance, bringing together a diverse skill set to drive the company's mission forward.
What is the current price of Amicus Therapeutics, Inc. - Common Stock?
The current price of Amicus Therapeutics, Inc. - Common Stock is 7.990
When was Amicus Therapeutics, Inc. - Common Stock last traded?
The last trade of Amicus Therapeutics, Inc. - Common Stock was at 4:00 pm EDT on April 2nd, 2025
What is the market capitalization of Amicus Therapeutics, Inc. - Common Stock?
The market capitalization of Amicus Therapeutics, Inc. - Common Stock is 2.13B
How many shares of Amicus Therapeutics, Inc. - Common Stock are outstanding?
Amicus Therapeutics, Inc. - Common Stock has 266.56M shares outstanding.