FDA approves Xembify for biweekly dosing, treatment-naïve patients

Key takeaways:

• The company expects Xembify’s expanded use to be available in the U.S. by the end of September.
• The expanded label aims to minimize the burden of care for patients with primary immunodeficiencies.

The FDA approved an expanded label for Xembify, a 20% subcutaneous immunoglobulin used to treat primary immunodeficiencies produced by Grifols, according to a company press release.

The expansion allows patients to begin subcutaneous immunoglobulin (SCIG) therapy without previously having intravenous therapy, as well as biweekly dosing. The drug was first approved in 2019 for weekly dosing for patients with primary immunodeficiencies (PI). The expansion aims to improve treatment flexibility and convenience.

“The need for treatments is growing due to an increased prevalence of these conditions,” Joerg Schuettrumpf, MD, chief scientific innovation officer at Grifols, told Healio. “This is driven by better diagnosis. Grifols wants to ensure patients have access to the medicines they depend on.”

The expansion is supported by a phase 4 clinical trial, a multicenter, single-sequence, open-label study that included 33 patients in 18 U.S. locations, according to Schuettrumpf.

The trial showed that patients treated with Xembify every 2 weeks vs. every week achieved non-inferiority in total Ig levels. The trial did not identify any safety issues and the tolerability profiles were consistent in both doses.

“In addition to including treatment-experienced patients already treated with immunoglobulin, the study also included treatment-naïve patients initiating IgG replacement therapy for the first time,” Schuettrumpf said.

“Additionally, previous data showed that with Xembify, patients reach a therapeutic level of IgG quickly and remain above the commonly accepted therapeutic threshold between doses, regardless of age,” he continued.

Current PI treatments involve immunoglobulin replacement therapy (IGRT), as 75% of patients with this condition cannot make antibodies normally, according to Richard Wasserman, MD, managing partner of Allergy Partners of North Texas, explained. IGRT, which Wasserman called lifesaving treatment, works by administering antibodies derived from plasma to replace those antibodies that patients cannot make themselves.

“Regular treatment is needed to reduce the frequency and severity of infections, enhance quality of life, and manage the symptoms associated with PIs,” he said. “Patients typically require replacement therapy for the rest of their lives.”

He further added that IGRT can be given intravenously or subcutaneously, but the type of administration depends on medical considerations and the need to minimize burden of care.

“From my point of view as a doctor, the most important advantage of the Xembify expanded label is that it is now the first and only 20% SCIG available that provides dosage and administration guidance for starting patients directly on subcutaneous administration without the need to begin first on IVIG therapy,” Wasserman said.

“The expanded label reduces the burden of care for patients with immunodeficiencies by eliminating the need for IV treatment. This is a significant advancement for patients, especially pediatric patients who are often challenging to treat with IV infusions,” he continued.

While Xembify is already available in its expanded label within some countries in Europe, it is expected to launch in the U.S. in the third quarter of this year.