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.

Emil Kakkis, Ultragenyx

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.

The drug will be sold as Crysvita for X-linked hypophosphatemia (XLH) in adult and pediatric patients 1 year of age and older. This is the first drug approved for XLH in the US, “and the only treatment that targets the underlying cause of the disease.”

In a call with analysts, the company said it would set a real-world price of about $160,000 for children and $200,000 a year for adults, depending on their weight.

The disease triggers high levels of phosphorous in the blood. In one study 94% of adults receiving Crysvita once a month achieved normal phosphorus levels — compared to only 8% of those receiving placebo.

Ultragenyx also wins its second priority review voucher with the approval after selling the first for $130 million.

Analysts like the win, but some are also offering caveats. From Joseph Schwartz at Leerink:

We believe that investors will welcome today’s update although there may remain some controversy about the attractiveness of the clinical profile, particularly in adults who did not benefit from a significant pain reduction in Phase 3.

“XLH differs from other forms of rickets in that vitamin D therapy is not effective,” stated Julie Beitz, director of the Office of Drug Evaluation III in the FDA’s Center for Drug Evaluation and Research. “This is the first FDA-approved medication for the treatment of XLH and a real breakthrough for those living with this serious disease.”

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Research Scientist - Immunology
Recursion Pharmaceuticals Salt Lake City, UT
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Atlas Venture Cambridge, MA

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