Amicus Therapeutics: A Global Biotechnology Leader in Rare Diseases

Strategic Insights -

Amicus Therapeutics is a patient-dedicated biotechnology company focused on discovering, developing, and delivering novel medicines for rare diseases. The company's cornerstone product, Galafold(R) (also referred to as "migalastat"), has achieved widespread global approval as the first and only approved oral precision medicine for people living with Fabry disease who have amenable genetic variants. Additionally, the company is leading the development of a differentiated biologic for Pompe disease in the clinic and has an industry-leading rare disease gene therapy portfolio.

Key Takeaways:

  • Amicus Therapeutics has a robust pipeline of product opportunities, including Galafold(R) for Fabry disease, AT-GAA for Pompe disease, and gene therapy programs for various rare metabolic diseases.
  • The company has established an industry-leading gene therapy portfolio through a license with Nationwide Children's Hospital and an expanded collaboration with the University of Pennsylvania.
  • Galafold(R) has achieved widespread global approval, including in the United States, European Union, United Kingdom, and Japan, with multiple additional approvals pending.
  • The company has seen strong commercial momentum and expansion into additional geographies for Galafold(R), with a high rate of compliance and adherence among patients.
  • Amicus Therapeutics has completed enrollment in its global Phase 3 pivotal study of AT-GAA for Pompe disease and has received Breakthrough Therapy designation from the U.S. FDA.
  • The company is working closely with strategic partners to support its clinical manufacturing capabilities for its gene therapy programs.
  • Amicus Therapeutics has maintained operations and supply chains during the COVID-19 pandemic, with minimal disruptions and continued access to medicines for those in need of treatment.

Statistics:

  • Galafold(R) revenue totaled $60.5 million for the three months ended March 31, 2020, an increase of $26.5 million compared to the same period in the prior year.
  • The FDA has approved Galafold(R) for 348 amenable GLA variants.
  • The company has completed enrollment in its global Phase 3 pivotal study of AT-GAA for Pompe disease with 123 participants at 59 global sites.
  • Amicus Therapeutics has secured Fast Track designation for its CLN3 Batten disease gene therapy program.

Sources:

  • Amicus Therapeutics (10-K filing)
  • U.S. FDA press releases
  • European Medicines Agency (EMA) press releases
  • University of Pennsylvania press releases
  • Nationwide Children's Hospital press releases
  • Comtex SmarTrend Alert (dated 03-02-2012 for FOLD @ $5.84)