Fresh from securing FDA approval for its combination treatment targeting Pompe disease, Amicus Therapeutics has attracted a significant investment. Amicus announced a substantial $430 million financing deal with Blackstone Life Sciences and Blackstone Credit on Monday. The agreement entails a $400 million loan provided by the asset manager to Amicus for the purpose of refinancing existing debt, as stated in Amicus’ press release. In addition to the loan, Blackstone has committed a strategic investment of $30 million in Amicus’ common stock.
This strategic move is strategically aligned with Amicus’ objectives of increasing revenue and progressing towards profitability. The recent FDA approval of Pombility and Opfolda for the treatment of adults with late-onset Pompe disease, who do not respond to current enzyme replacement therapy (ERT), marked a significant milestone for Amicus. Pombility represents a long-term infused ERT, while Opfolda serves as an oral stabilizer that reduces the loss of enzyme activity in the blood during infusion.
Also Read: Moderna Axes Four Programs, Including Two Former AstraZeneca Collaborations
Amicus envisions substantial potential in the Pompe disease market, estimating it could reach $1.8 billion by 2027. The company also anticipates peak sales of its combination therapy to reach $1.2 billion. It has announced its readiness to launch in the United States immediately, pricing the treatment at $650,000 per year for patients of average weight.
Beyond its newly approved Pompe combination therapy, Amicus boasts another commercial product: Galafold, a treatment for Fabry disease. Galafold received FDA approval in August 2018. In 2022, Amicus reported full-year revenues totaling $329.2 million, reflecting an 8% year-over-year increase. The company had previously indicated its potential to achieve profitability by the second half of 2023 as part of its strategic outlook.
