Welcome to our dedicated page for Amicus Therapeut news (Ticker: FOLD), a resource for investors and traders seeking the latest updates and insights on Amicus Therapeut stock.
Amicus Therapeutics, Inc. (symbol: FOLD) is a global biotechnology company that specializes in discovering, developing, and delivering high-quality medicines for individuals suffering from rare metabolic diseases. The company's central focus revolves around addressing lysosomal storage disorders, such as Fabry disease and Pompe disease, through its innovative platform technologies and medicines.
Amicus Therapeutics operates with a patient-centric approach, ensuring that the needs of those in the rare disease community are at the forefront of their scientific research, commercial activities, and clinical programs. The company is dedicated to creating significant impacts on the lives of patients and their caregivers, striving to provide advanced therapeutic solutions where none previously existed.
The company's product pipeline includes:
- Galafold® (migalastat): An oral chaperone therapy approved for the treatment of adults with Fabry disease who have amenable genetic variants. Galafold works by stabilizing the body’s own dysfunctional enzyme to clear the accumulated substrate.
- Pombiliti™ and Opfolda™: A novel, two-component therapy designed for the treatment of late-onset Pompe disease, showcasing Amicus's commitment to addressing the complexities of metabolic disorders.
- Rare Disease Gene Therapy Portfolio: A set of advanced gene therapies aimed at providing long-lasting solutions for various rare metabolic diseases.
Amicus Therapeutics is focused on expanding its pipeline with first-in-class or best-in-class treatments, underscoring its commitment to innovative medical solutions. Financially, the company maintains a strong position, actively pursuing strategic priorities to sustain growth and enhance shareholder value.
With a presence in 27 countries, Amicus Therapeutics leverages a global footprint to address unmet medical needs across diverse patient populations. The company continuously seeks opportunities to broaden its impact and foster collaborations that advance its mission.
Recent developments include regulatory approvals and strategic partnerships aiming to enhance the reach and effectiveness of their therapies. By building strong relationships with stakeholders, Amicus Therapeutics ensures a collaborative effort in combating rare diseases.
Overall, Amicus Therapeutics Inc. embodies a profound dedication to transforming the lives of those affected by rare diseases through innovative science, patient-focused care, and robust therapeutic development.
Amicus Therapeutics (FOLD) announced that the UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) granted a positive scientific opinion for AT-GAA under the Early Access to Medicines Scheme. This allows eligible adults with late-onset Pompe disease to access AT-GAA prior to marketing authorization. The decision underscores the high unmet medical need in this patient group. AT-GAA, a two-component therapy, has shown promising results in clinical studies. Marketing Authorization Applications for AT-GAA are expected in the UK and EU in late 2021.
Amicus Therapeutics (Nasdaq: FOLD) will participate in the Goldman Sachs 42nd Annual Global Healthcare Conference on June 9, 2021, at 5:30 p.m. E.T. The event will include a fireside chat featuring members of Amicus' senior leadership team. Investors can listen to the live audio webcast via the company’s investor relations page. Amicus focuses on developing innovative medicines for rare metabolic diseases, underscoring its commitment to patient care and advancing a strong pipeline of therapies.
Amicus Therapeutics (Nasdaq: FOLD) announced the presentation of two posters at the virtual 16th International Congress on Neuromuscular Diseases on May 21-22 and 28-29, 2021. The posters focus on its development program for Pompe disease, including:
- Enhancing Delivery of Acid Alpha-Glucosidase to Skeletal Muscle by Nithya Selvan, Ph.D.
- Efficacy and Safety of Cipaglucosidase Alfa/Miglustat versus Alglucosidase Alfa/Placebo in Late-Onset Pompe Disease presented by Benedikt Schoser, MD.
The posters will be available on the Amicus website after the presentations.
Amicus Therapeutics reported Q1 2021 revenue of $66.4 million for Galafold, a 9.8% increase year-over-year, and remains on track to meet its full-year guidance of $300M-$315M. Patient demand for Galafold has risen, and compliance exceeds 90%. The company is progressing with the rolling BLA submission for AT-GAA in Pompe disease, anticipating completion in Q2 2021. Amicus maintains a cash position of $417.4 million, sufficient for operations through to self-sustainability.
Amicus Therapeutics (Nasdaq: FOLD) announced participation in two upcoming virtual investor conferences in May 2021. The Bank of America 2021 Health Care Conference is scheduled for May 12 at 1:15 p.m. E.T., followed by the UBS Global Healthcare Virtual Conference on May 24 at 10:00 a.m. E.T.. Investors can access live webcasts of these presentations on the company’s corporate website. Amicus Therapeutics is dedicated to developing innovative therapies for rare metabolic diseases.
Amicus Therapeutics (Nasdaq: FOLD) has appointed Sébastien Martel as Senior Vice President of Strategy and Business Development. With 25 years in the pharmaceutical and biotechnology sectors, Martel previously led Global Rare Diseases at Sanofi. His expertise encompasses corporate strategy and commercialization in rare diseases. Amicus CEO John F. Crowley highlighted Martel's experience as crucial for advancing the company's growth strategies and enhancing its portfolio of rare disease therapeutics.
Amicus Therapeutics (Nasdaq: FOLD) has announced the acceptance of two abstracts for the American Society of Gene & Cell Therapy 24th Annual Meeting, occurring virtually from May 11-14, 2021. The presentations will feature preclinical data from the company's gene therapy programs targeting Fabry and Pompe diseases. The Pompe disease abstract, presented by Ali Ramezani, focuses on muscle pathology reversal, while the Fabry disease abstract, by CJ Song, discusses the efficacy of a new transgene. These insights underscore Amicus's commitment to rare metabolic diseases.
Amicus Therapeutics (NASDAQ:FOLD) announced the successful completion of a Type B Pre-Biologics License Application (BLA) meeting with the FDA for its investigational therapy AT-GAA, aimed at treating Pompe disease. They are on track to complete the rolling BLA submission by the end of Q2 2021, including data from Phase 1/2 and Phase 3 studies. AT-GAA has previously received Breakthrough Therapy Designation from the FDA. The company plans to submit marketing applications to the EMA in the latter half of 2021, focusing on improving the lives of Pompe disease patients.
Amicus Therapeutics (Nasdaq: FOLD) will host a conference call and live audio webcast on May 10, 2021, at 8:30 a.m. ET, to discuss its financial results for Q1 2021, ended March 31, 2021. Investors can join by dialing 877-303-5859 (U.S./Canada) or 678-224-7784 (international), using conference ID: 5767104. A replay will be available for seven days starting at 11:30 a.m. ET. Amicus focuses on developing medicines for rare metabolic diseases, emphasizing a patient-centric approach.
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