Sanofi’s Riliprubart Gets Orphan Drug Designation from FDA for Organ Transplant Rejection

Riliprubart is Designated as an Orphan Drug in the US

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Announcement

The US Food and Drug Administration approved Rilpivirine, positioning it as an orphan drug designation. Rilipubart, an investigational treatment for antibody-mediated rejection (AMR) in solid organ transplantation. The designation is similar to one of Sanofi’s commitments to identify complex unmet needs in transplant medicine. Regarding which AMR remains challenging, as there is no FDA-approved treatment available yet. The FDA, to date, has awarded orphan drug designation to investigational therapies for rare medical conditions or diseases affecting 200,000 individuals in the US.

What New Development has entered the Healthcare Market?

Riliprubart, also called (SAR445088), has been found in various clinical studies in different indications in neurology and transplant. Currently, the phase 2 clinical study is in process considering its ability in kidney transplant recipients (NCT05156710). The study is segregated into two patient cohorts, one with a risk of facing rejection and the other consisting of an active form of antibody-mediated rejection.

Whereas, Sanofi is involved in two phase 3 studies identifying a possibility of riliprubart in chronic inflammatory demyelinating polyneuropathy (CIDP), which is a rare neurological disorder mainly found in IVIg-treated patients (VITALIZE, clinical study identifier: NCT06290141) and patients refractory to standard of care (MOBILIZE, clinical study identifier: NCT06290128). The clinical development program is organized for riliprubart, showing Sanofi’s efforts and commitment to explore riliprubart's ability to meet various immune-mediated conditions.

Progress

The continued clinical studies (trials) have been immensely contributing to science and the healthcare sector. The current US-approved and designated orphan drug has been introduced to support other studies that will lead to expansion with the performance in various other treatments. The ongoing phase 2 study is exploring an additional potential to treat and prevent active antibody-mediated rejection in kidney transplant recipients. Previously, riliprubart was a designated orphan drug in the EU and the US for investigational use in chronic inflammatory demyelinating polyneuropathy. The study is progressing and fostering innovation for this new designated orphan drug, spotlighting Sanofi’s ongoing commitment.

Views and Statements

Global Therapeutic Area Development Head, Immunology and Inflammation, Sanofi, Alyssa Johnsen said, “Riliprubart’s orphan drug designation is a milestone in our mission to identify intricate challenges in transplant medicine, accelerating our expertise in immunology. Antibody-mediated rejection is a serious condition to patient survival and transplanted organs. Riliprubart’s innovative mechanism will introduce a treatment option that will enhance the result for kidney transplant recipients.”