In a statement on its website, the company reassured people with X-linked hypophosphatemia (XLH) that the manufacturing and distribution of Crysvita remains intact. Ultragenyx also said it has sufficient inventory of Crysvita and other medications on hand to account for all patient needs, in the rare event of a supply chain interruption.
Questions or concerns can be addressed by calling Ultragenyx’s phone help line, called UltraCare, at 888–756–8657 from 8 a.m. to 8 p.m. EDT, Monday to Friday. Requests for information also can be sent by email to [email protected]
Crysvita is approved in the U.S. for the treatment of XLH patients 6 months and older. In the European Union, the therapy was granted conditional marketing authorization by the European Medicines Agency for those 1 year and older.
Crysvita works by blocking a protein called fibroblast growth factor 23 (FGF23). Normally, FGF23 is involved in the regulation of phosphate levels in the blood by blocking the kidneys from reabsorbing phosphate.
In XLH patients, high activity of the PHEX gene leads to excess FGF23 activity, which causes the kidneys to excrete phosphate at an increased rate. This leads to low blood levels of phosphate, which results in symptoms that can affect bones, teeth and muscles.
By blocking FGF23, Crysvita enables the kidneys to reabsorb more phosphate and restore its levels in the blood to improve bone growth.
Ultragenyx also has taken steps to limit spread of COVID-19 by encouraging employees to work from home, conducting all meetings virtually, and cancelling all conference and travel engagements. The company said it has implemented a rigorous cleaning protocol in all offices.
Ultragenyx will continue monitoring the situation closely, but does not anticipate supply interruptions in ongoing clinical trials. Participating sites will receive continued support during the pandemic.
Besides XLH, the company is currently waiting for the U.S. Food and Drug Administration to decide whether to extend Crysvita’s indications to tumor-induced osteomalacia, and approve a therapy known as UX007 for people with long-chain fatty acid oxidation disorders.
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