Grifols Doses First Patients in Two Phase 3 Trials to Expand Labels for Gamunex-C and Xembify
核心洞察
Grifols has dosed the first patients in two separate Phase 3 trials, SIGMA and XPERT, aiming to expand U.S. indications for its immunoglobulin products Gamunex-C and Xembify.
The SIGMA trial will evaluate Gamunex-C for preventing infections in patients with secondary immunodeficiency (搜索) due to blood cancers including CLL, multiple myeloma (搜索), and non-Hodgkin lymphoma (搜索).
The XPERT trial will assess whether Xembify is non-inferior to Gamunex-C as maintenance therapy for chronic inflammatory demyelinating polyradiculoneuropathy (搜索) (CIDP).
Grifols has announced that the first patients have been dosed in two separate Phase 3 clinical trials designed to expand the U.S. labels for its flagship immunoglobulin therapeutics: Gamunex-C, an intravenous immunoglobulin (IVIg), and Xembify, a subcutaneous immunoglobulin (SCIg). The trials represent a strategic effort to broaden the approved indications for the company's plasma-derived immunoglobulin portfolio.
SIGMA Trial: Targeting Secondary Immunodeficiency (搜索) in Hematologic Cancers
The SIGMA trial (NCT07582432) will evaluate the pharmacokinetics, efficacy, and safety of Gamunex-C in combination with standard of care treatment to prevent infections in patients with secondary immunodeficiency (搜索) (SID) characterized by low immunoglobulin levels. Unlike primary immunodeficiency, which is genetic, SID occurs when a previously healthy immune system is compromised by factors such as blood cancer and treatments like chemotherapy or B-cell-targeted therapies.
This open-label, multicenter, single-arm prospective study will focus specifically on patients with common blood cancers including chronic lymphocytic leukemia (搜索) (CLL), multiple myeloma (搜索) (MM), and non-Hodgkin lymphoma (搜索) (NHL) — malignancies that together account for more than 1 million patients in the United States. Fifty participants diagnosed with B-cell CLL, MM, or NHL with hypogammaglobulinemia will take part in the trial.
The primary efficacy objective of the SIGMA trial is to demonstrate that the rate of serious bacterial infections is less than 1 per participant per year when treated with Gamunex-C plus standard of care medical treatment. Expanding the label to include hematologic cancer–associated SID would broaden the approved indications for Gamunex-C in the U.S.
XPERT Trial: Evaluating Xembify in CIDP
The second clinical trial, XPERT (NCT07540221), will determine whether Xembify's pharmacokinetic profile is non-inferior to that of Gamunex-C for treating patients with chronic inflammatory demyelinating polyradiculoneuropathy (搜索) (CIDP). The study will also evaluate Xembify's safety profile and efficacy based on its ability to keep patients' condition under control.
Approximately 60,000 patients in the U.S. suffer from CIDP, a chronic illness with symptoms including progressive weakness in the arms and legs and changes in sensation. Immunoglobulin therapy, including Gamunex-C, is a first-line treatment with immunomodulatory properties that can improve neuromuscular disability and prevent relapse. Gamunex-C is already indicated to treat this autoimmune disease.
The XPERT trial is an open-label, multicenter study that will enroll approximately 40 CIDP patients across 20 sites in the U.S. and Europe. Favorable results showing non-inferiority between the two immunoglobulin therapeutics, which share similar manufacturing processes, may support future regulatory discussions, including a potential application to seek expansion of Xembify's U.S. label to include CIDP.
This trial builds on Grifols' longstanding history in CIDP research. In the mid-2000s, a pivotal trial centered on Gamunex-C demonstrated its short-term and long-term efficacy in reducing disability, including improvement in grip strength, in patients with CIDP compared to placebo.
Building on a Growing Immunoglobulin Portfolio
These two new trials add to a third immunoglobulin trial already underway. Announced at the end of 2022, the EXCELL trial (NCT05645107) is evaluating the efficacy, safety, and pharmacokinetics of Xembify plus standard medical treatment compared to placebo plus standard medical treatment to prevent infections in participants with hypogammaglobulinemia and recurrent or severe infections associated with B-cell CLL, multiple myeloma (搜索), and non-Hodgkin lymphoma (搜索).
"We're excited about the potential to provide patients with more immunoglobulin treatment options to help manage their conditions with flexibility and convenience," said Eduardo Herrero, Grifols' executive vice president biopharma industrial and scientific innovation. "Whether it's our IVIg or SCIg treatments, Grifols' Igs are backed by decades of deep expertise on this protein."
Lisa Butler, president and CEO of The GBS | CIDP Foundation, added: "The GBS I CIDP Foundation International embraces Grifols' commitment to innovate and to support the patient community with additional choice in treatment and access options."
Grifols' suite of immunoglobulin products continues to perform strongly, driven by increasing patient need worldwide. Xembify, in particular, has benefited from robust demand, growing nearly 60% year over year in 2025. In July 2024, the U.S. FDA approved an expanded label for Xembify to include treatment-naïve patients with primary humoral immunodeficiencies, making it the first 20% subcutaneous immunoglobulin with dosing approved for this population and introducing a convenient biweekly dosing option.
Gamunex-C is currently indicated for primary humoral immunodeficiency (搜索) disease in patients 2 years of age and older, idiopathic thrombocytopenic purpura in adults and children, and CIDP in adults. Xembify is a 20% immune globulin indicated for primary humoral immunodeficiency disease in patients 2 years of age and older, for subcutaneous administration only.