KYV-101 granted orphan drug status for MG in Europe
Cell therapy has equivalent designation in US
The European Medicines Agency granted orphan drug status to the experimental cell therapy KYV-101 to treat myasthenia gravis (MG).
The designation is given to medicines that aim to treat life-threatening or chronic debilitating conditions affecting no more than five in every 10,000 people. It provides companies with several benefits, including assistance with trial protocols, reduced regulatory fees, and 10 years of market exclusivity upon approval.
Similar benefits are awarded to therapies receiving the same status in the U.S., though the market exclusivity period upon approval lasts seven years. Kyverna Therapeutics’ KYV-101 has received orphan drug designation from the U.S. Food and Drug Administration (FDA) to treat MG.
“With KYV-101 advancing towards later stages of development, we are scaling Kyverna to bring the transformative impact of our differentiated CAR T therapies to patients with a range of B cell-driven autoimmune diseases,” Warner Biddle, Kyverna’s CEO, said in a company press release.
MG is an autoimmune disease caused by autoantibodies that target proteins at the neuromuscular junction, the site where nerve and muscle cells communicate to coordinate voluntary movements. Most commonly, these antibodies target acetylcholine receptors (AChRs), and less frequently, another protein called muscle-specific kinase (MuSK).
CAR T-cell therapy
KYV-101 is an investigational chimeric antigen receptor (CAR) T-cell therapy designed to reduce the levels of antibody-producing immune B-cells, which is expected to ease disease severity in people with MG.
It involves collecting a patient’s T-cells — immune cells that are able to kill other cells — and engineering them in the lab with a CAR targeting CD19, a protein found on the surface of B-cells. This CAR enables T-cells to specifically recognize and destroy B-cells once they are infused back into the body.
KYV-101 is designed to be given as a single intravenous (into-the-vein) infusion following a short course of chemotherapy to remove existing immune cells before the engineered cells are reinfused.
The therapy is being tested in a Phase 2 trial called KYSA-6 (NCT06193889), which is expected to enroll 20 patients, ages 18 to 75, with treatment-resistant generalized MG, who test positive for autoantibodies targeting AChR or MuSK. Patients are being recruited at sites in the U.S. and Germany.
The trial’s main goals include assessing the therapy’s safety and efficacy, as evaluated by changes in the scores of the MG Activities of Daily Living (MG-ADL) scale, a patient-reported measure of MG severity that assesses the disease’s impact on a patient’s ability to perform daily functions, for about six months. Safety assessments will be conducted for up to two years.
KYV-101 has received FDA fast track and regenerative medicine advanced therapy (RMAT) designations. Both are intended to support and accelerate the therapy’s development and regulatory review.
The therapy is also being investigated as a potential treatment for other autoimmune conditions, including lupus nephritis, systemic sclerosis, multiple sclerosis, and stiff person syndrome.
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