FDA Approves Carvykti™ CAR T-Cell Therapy for Relapsed/Refractory Multiple Myeloma

The U.S. Food and Drug Administration (FDA) has approved Carvykti™ (ciltacabtagene autoleucel) CAR T-Cell therapy for relapsed/refractory multiple myeloma in adults.^1,2

People must have tried at least 4 other treatments before using Carvykti. These other treatments must include a proteasome inhibitor (PI), an immunomodulatory agent (IMid), and an anti-CD38 monoclonal antibody.^1,2

The application for Carvytki was granted:¹

• Priority review – Granted when a new drug seems promising and may greatly help a disease. Priority Review means the FDA will decide whether to approve new drugs in 6 months. It is only for drugs that seem to work better or are safer than current treatments.
• Breakthrough designation – This makes a drug that treats a serious condition eligible for faster approval from the FDA. It is only for drugs that seem to work better or are safer than current treatments.
• Orphan drug designation – Orphan drugs prevent, diagnose, or treat a rare disease or condition. A rare disease or condition affects fewer than 200,000 people in the United States. The U.S. government provides incentives and support for companies to develop orphan drugs. This encourages them to develop medicines for rare diseases or conditions.

What are the ingredients in Carvykti?

The active ingredient in Carvykti is ciltacabtagene autoleucel.²

How does Carvykti work?

Carvykti is a type of treatment called chimeric antigen receptor (CAR) T-cell therapy. This is a new type of treatment that uses the body’s immune system to fight cancer. It uses the person's own T-cells, which are important cells in the immune system. Carvykti works by helping the T-cells identify and fight abnormal cancer cells that cause multiple myeloma.²

Evidence for Carvykti

Carvykti was approved based on the results from a clinical trial. The trial included 97 people with relapsed or refractory multiple myeloma who:^1,2

• Received at least 3 prior treatments that included a PI, IMiD, and an anti-CD38 monoclonal antibody, and
• Their cancer worsened on or after their last chemotherapy treatment

The study found that 98 percent of the people who received Carvykti had their cancer shrink or disappear. About 78 percent of this group did achieve a complete response. This means a doctor was not able to find any remaining signs of the cancer. ^1,2

What are the possible side effects of Carvykti?

The most common side effects of Carvykti include:²

• Fever, chills
• Dizziness or lightheadedness
• Headache
• Muscle or joint pain
• Tiredness
• Confusion
• Infections
• Low levels of antibodies (immunoglobin) in the blood
• Cough, shortness of breath
• Diarrhea
• Nausea, decreased appetite
• Fast or irregular heartbeat
• Problems with blood clotting

Carvykti has a boxed warning, the strictest warning from the FDA. It has this warning because it may cause severe, life-threatening side effects. They include:³

• Cytokine Release Syndrome (CRS) – An immune reaction that begins with flu-like symptoms and can cause serious illness
• Immune Effector Cell-Associated Neurotixicity Syndrome (ICANS) – A syndrome that causes neurologic side effects such as confusion, disorientation, loss of consciousness, or seizures
• Parkinsonism and Guillain-Barré syndrome – A disorder where your immune system starts to attack your nerves
• Hemophagocytic Lymphohistiocytosis/Macrophage Activation Syndrome (HLH/MAS) – A disorder where white blood cells start to attack other healthy blood cells
  Low red blood cell level that may require blood transfusions to treat

Because of these risks, Carvykti can only be given through a restricted program called Risk Evaluation and Mitigation Strategies (REMS). This means it is only given at REMS-certified healthcare facilities. The REMS program ensures the drug is used under controlled circumstances and helps monitor safety while using it.²

Call your doctor right away if you have any signs of severe side effects, including:²

• Fever
• Chills
• Fast or irregular heartbeat
• Breathing problems
• Very low blood pressure
• Dizziness, lightheadedness
• Confusion, slurred speech, memory loss
• Movement or balance problems
• Personality changes
• Tingling, numbness, or pain in hands and feet
• Facial numbness

These are not all the possible side effects of Carvykti]. Talk to your doctor about what to expect when taking Carvykti. You also should call your doctor if you have any changes that concern you when taking Carvykti.

Things to know about Carvykti

Before you receive Carvykti, your doctor will give you chemotherapy for 3 days to prepare your body.²

People receiving Carvykti will be monitored each day for the first 10 days following the infusion at a REMS-certified facility. They should also stay near a REMS-certified facility for at least 4 weeks following the infusion.²

Your doctor will perform blood tests to monitor your blood counts before and after treatment with Carvykti.²

You should not donate blood, organs, tissues, or cells for transplantation after receiving Carvykti.²

Carvykti may increase the risk of infection or alter immunity after getting certain vaccines. Before starting Carvykti, ask your doctor if you need to receive any vaccines.²

You should not drive, operate heavy machinery, or do activities that could be dangerous if you are not mentally alert for at least 8 weeks after treatment with Carvykti. This is because Carvykti can cause memory problems, sleepiness, confusion, and other neurological side effects.²

Carvykti may harm an unborn baby. If you or your partner can become pregnant, you should use birth control during treatment and for some time after the last dose of Carvykti. You should also not breastfeed during treatment with Carvykti and for some time after the last dose. Talk to your doctor about your options for birth control and breastfeeding while taking Carvykti.²

Before beginning treatment for multiple myeloma, tell your doctor about all your health conditions and any other drugs, vitamins, or supplements you are taking. This includes over-the-counter drugs.

For more information, read the full prescribing information of Carvykti.