26. Galafold

FDA
(FDA)

Active ingredient: migalastat
Disease: Fabry disease
Peak sales estimate: $534 million by 2024
Approved: Aug. 10
Company: Amicus Therapeutics

The scoop: Galafold got a faster-than-expected nod this summer—a now-vacated FDA demand for additional safety trials once pushed its anticipated timeline to 2020—but with the approval bumps up against Sanofi's standard and well-established Fabry disease treatment Fabrazyme. Amicus’ lower list price of $315,000 versus Fabrazyme’s average price of $344,000 price tag may help its cause, but it’s fighting Sanofi’s 15-year history with Fabrazyme, which recorded sales last year of $824 million. That said, Galafold is an oral drug versus the IV-infused Fabrazyme, making it more convenient for patients, although not every Fabry patient will be eligible. Galafold specifically treats adults with certain variants of a rogue gene and works by boosting the body’s missing enzyme rather than replacing it in patients. An estimated 35% to 50% of Fabry’s disease patients with an amenable galactosidase alpha gene mutation would be eligible for the treatment. Amicus, which has promised to not raise the price of Galafold higher than the rate of consumer inflation, expects total global sales of the drug to hit about $90 million this year. — Beth Snyder Bulik 

26. Galafold

Suggested Articles

German researchers uncovered 28 antibodies that neutralize COVID-19 and are working with Boehringer Ingelheim to advance them into clinical testing.

Oragenics is ending a phase 2 study of its oral mucositis drug, yanking its IND application and switching its focus to a COVID-19 vaccine.

The vehicle, which Blackstone claims is the largest life sciences private fund, has committed close to $1 billion to companies including Alnylam.