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

Jan 29, 2021 9:45am
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.

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