(Cipaglucosidase alfa + Miglustat) is a recombinant enzyme and small molecule commercialized by Amicus Therapeutics, with a leading Pre-Registration program in Pompe Disease. According to Globaldata, it is involved in 8 clinical trials, of which 1 was completed, and 7 are ongoing. GlobalData uses proprietary data and analytics to provide a complete picture of (Cipaglucosidase alfa + Miglustat)’s valuation in its risk-adjusted NPV model (rNPV). Buy the model here.

The revenue for (Cipaglucosidase alfa + Miglustat) is expected to reach an annual total of $543 mn by 2035 in the US based off GlobalData’s Expiry Model. The drug’s revenue forecasts along with estimated costs are used to measure the value of an investment opportunity in that drug, otherwise known as net present value (NPV). Applying the drug’s phase transition success rate to remaining R&D costs and likelihood of approval (LoA) to sales related costs provides a risk-adjusted NPV model (rNPV). The rNPV model is a more conservative valuation measure that accounts for the risk of a drug in clinical development failing to progress.

(Cipaglucosidase alfa + Miglustat) Overview

Cipaglucosidase alfa in combination with miglustat is under development for the treatment of Pompe disease. The drug candidate is a fixed dose of recombinant human alpha glucosidase (rhGAA , ATB200)  with a chaperone (AT2221). AT2221 is administered orally, formulated as capsule and ATB200 by intravenous route. It is developed based on Chaperone-Advanced Replacement Therapy (CHART) technology using engineered human alpha glucosidase enzyme as a replacement therapy.

Amicus Therapeutics Overview

Amicus Therapeutics is a biotechnology company, which focuses on the research, development and commercialization of small molecule drugs and orally administered drugs known as pharmacological chaperones for the treatment of rare and orphan diseases. The company’s lead product, migalastat HCl, a small molecule, is indicated for the treatment of Fabry disease. The other products in the pipeline include ATB200/AT2221 for the treatment of Pompe disease, Fabry disease, and other lysosomal storage disorders; migalastat for other conditions; and enzyme replacement therapies for other rare diseases. It has presence in the US, the UK, Ireland, France, the Netherlands, Germany, Spain, Italy and Japan. Amicus Therapeutics is headquartered in Philadelphia, Philadelphia, the US.

The company reported revenues of (US Dollars) US$305.5 million for the fiscal year ended December 2021 (FY2021), an increase of 17.1% over FY2020. The operating loss of the company was US$206.7 million in FY2021, compared to an operating loss of US$254.3 million in FY2020. The net loss of the company was US$250.5 million in FY2021, compared to a net loss of US$276.9 million in FY2020. The company reported revenues of US$81.7 million for the third quarter ended September 2022, an increase of 1.2% over the previous quarter.

For a complete picture of (Cipaglucosidase alfa + Miglustat)’s valuation, buy the drug’s risk-adjusted NPV model (rNPV) here.


GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article.

To create this model, GlobalData takes into account factors including patent law, known and projected regulatory approval processes, cash flows, drug margins and company expenses. Combining these data points with GlobalData’s world class analysis creates high value models that companies can use to help in evaluation processes for each drug or company.

The rNPV method integrates the probability of a drug reaching a clinical stage into the cash flow at that time, which provides a more accurate valuation, as it considers the probability that the drug never makes it through the clinical pathway to commercialization. GlobalData’s rNPV model uses proprietary likelihood of approval (LoA) and phase transition success rate (PTSR) data for the indication in the highest development stage, which can be found on GlobalData’s Pharmaceutical Intelligence Center.