First up is Ultragenyx, a commercial- and clinical-stage biopharma company with a focus on rare diseases. These diseases are defined by the FDA as affecting fewer than 1 in 50,000 people; as such, they have a small patient base and limited prospects for drug sales to reimburse the high costs of development. Ultragenyx spreads out those costs by operating several of its development lines in partnerships with other pharmaceutical researchers, and works assiduously at commercialization of its approved products.
On that front, the company has had a measure of success. In 4Q21, Ultragenyx showed $83.4 million in total revenue, which was in-line with consensus expectations. The total included $55.5 million in revenue from burosumab (branded as Crysvita), as well as smaller sums from two other commercial-stage products. Triheptanoin (Dojovi) saw product sales of $11.8 million, while vestronidase alfa (trade name Mepsevii) brought in $3.1 million.
