Ultragenyx wins a blockbuster OK for its leading rare disease drug burosumab -- priced at $200,000
Ultragenyx $RARE has won the big FDA approval they were looking for.
The biotech — allied with Kyowa Hakko Kirin — reported Tuesday afternoon that the agency came through on burosumab, an inherited form of rickets. This is its second OK and the one that the company believes will put them on the map for commercialization work.
This drug is central to Ultragenyx’s narrative. The biotech recently scored an approval for Mepsevii, a drug that treats rare cases of mucopolysaccharidosis type VII . But it’s the burosumab launch that has everyone’s attention on Wall Street. The biotech has been setting up a small, 30-person sales force as it begins to handle what Ultragenyx execs hope will be a substantial portfolio of rare disease drugs. And burosumab has peak sales figures of $1 billion tied to it.
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