Ultragenyx earns 2 rare disease drug approvals—and a validated commercial strategy

Ultragenyx logo
Ultragenyx nabbed its third and fourth FDA approvals in 2020 and pushed through the pandemic by reaching out to patients directly. (Ultragenyx)

Rare disease pharma companies spend a lot of time looking for patients and investing in keeping them on their meds.

But what happens during a pandemic lockdown? At Ultragenyx, patient support teams kicked into high gear, making thousands of proactive outbound calls to find out what patients needed to continue their treatment.

It had just nabbed a second approval for FGF23 hormone blocker Crysvita in June 2020 for tumor-induced osteomalacia. The first nod for the drug, developed by Ultragenyx and partner Kyowa Kirin, came in April 2018 for X-linked hypophosphatemia.

And while Ultragenyx support teams worked with those patients, the biopharma's management reached out to the FDA to seek approval for temporary home administration of the injection.

RELATED: Ultragenyx's Crysvita scores FDA nod in 2nd ultra-rare bone disorder

As it turned out, the pandemic helped Ultragenyx solidify its rare disease commercial model. One important strategy it validated was its practice of maintaining equal-sized sales teams in the field and patient diagnostic teams in medical affairs because “patient diagnosis is critical,” Chief Commercial Officer Erik Harris said.

“The key to success for any rare disease business, and it has been the key for us, is the ability to identify and confirm diagnosis of rare disease patients," Harris said. "What you find with rare disease (drug) launches is they often stall after the first year or so because of the inability to find patients."

The consistent strategy has been important for Ultragenyx's other 2020 approval, also in June, for Dojolvi, a metabolic supplement for a rare group of disorders; those patients cannot convert certain fatty acids into energy. Ultragenyx is now just rolling out Dojolvi but is using lessons learned from its commercialization of Crysvita.

RELATED: Ultragenyx scores 2nd FDA approval in as many weeks for ultra-rare metabolic disease drug Dojolvi

The focus is again on finding patients. For Dojolvi, that means locating and contacting the estimated 2,000 to 3,500 people affected, starting with the 160 metabolic centers where a concentration of patients are treated. Ultragenyx plans to eventually move that model outside those centers to reach more patients and physicians, Harris said, much as it's done with Crysvita.

Ultragenyx did get the FDA's go-ahead for home injections and now will take that into the future with plans to discuss extending use beyond the pandemic, Harris said.

Crysvita sales for 2020 are expected to hit between $137 million and $139 million. Company revenue estimates for 2021 fall in the $180 million to $190 million range. Piper Sandler analysts pointed out in an investor note that Ultragenyx revenue guidance is in line with its expectations, adding that its "pipeline progress continues on track, and we continue to like the setup on this name."

Ultragenyx's two 2020 approvals represent the third and fourth since Ultragenyx began in 2010. Up next? A pipeline and a plan to reach $1 billion in sales in the next five years.

“The momentum generated by those new approvals, along with the clinical pipeline, form our foundation for the next five years,” Harris said.