FDA Approves Expanded Use of Xembify for Immunodeficiency Treatment

The FDA has approved an expanded label for XEMBIFY®, permitting direct subcutaneous administration without prior IV therapy for patients with primary immunodeficiencies. This first-of-its-kind approval includes a biweekly dosing option, shown to maintain effective immunoglobulin levels, enhancing convenience and flexibility for patients.

Grifols announced that the US Food and Drug Administration (FDA) has approved an expanded label for XEMBIFY®, the first 20% subcutaneous immunoglobulin (SCIg), to include treatment-naïve patients with primary humoral immunodeficiencies (PI). This approval marks a pivotal shift, allowing treatment-naïve patients to initiate SCIg therapy directly without the prerequisite of intravenous administration.

Joerg Schuettrumpf, Chief Scientific Innovation Officer at Grifols, highlighted the patient-centric benefits of the new labeling, stating, “The XEMBIFY label expansion eliminates the need for patients to have initial intravenous treatment, which differentiates XEMBIFY from other SCIg therapies, plus offers patients greater convenience and flexibility with biweekly dosing.”

XEMBIFY® is now the first and only 20% SCIg product to receive FDA approval for biweekly dosing under the Supplemental Biologics Application (sBLA). This unique option has proven effective in maintaining total immunoglobulin levels comparably to the traditional weekly regimen. This finding comes from robust data obtained in a phase 4 clinical trial (NCT04566692), which demonstrated non-inferiority in total Ig levels when administered every two weeks versus weekly.

The study, a multicenter, single-sequence, open-label trial, encompassed 27 subjects across 18 U.S. sites, confirming the safety and efficacy of XEMBIFY® under its new dosing protocol. Notably, there were no unique safety issues identified, and the tolerability profiles remained consistent between the two dosing schedules.

The expansion is a strategic component of Grifols' broader Ig business strategy, aimed at addressing a growing market need. Immunodeficiencies, which include both primary and secondary forms, currently represent over half of the Ig market, with expectations to grow significantly in the coming years. The market for Ig treatments, particularly for immunodeficiencies, is anticipated to outpace all other indications.

This label expansion is set to launch in the U.S. in the third quarter of 2024, enhancing Grifols' already robust portfolio of immunoglobulin solutions and reinforcing its commitment to innovate and adapt to patient needs and lifestyles. XEMBIFY® is also approved for PI in various other regions, including Europe, Canada, and Australia, with plans underway to extend its availability in additional European countries.