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

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.