Naxitamab, sold under the brand name Danyelza, is an

anti-cancer medication Chemotherapy (often abbreviated chemo, sometimes CTX and CTx) is the type of cancer treatment that uses one or more anti-cancer drugs ( chemotherapeutic agents or alkylating agents) in a standard regimen. Chemotherapy may be given with a cu ...

. It is a

monoclonal antibody A monoclonal antibody (mAb, more rarely called moAb) is an antibody produced from a cell lineage made by cloning a unique white blood cell. All subsequent antibodies derived this way trace back to a unique parent cell. Monoclonal antibodie ...

used in combination with granulocyte-macrophage colony-stimulating factor (GM-CSF) for people one year of age and older with relapsed or refractory high-risk

neuroblastoma Neuroblastoma (NB) is a type of cancer that forms in certain types of nerve tissue. It most frequently starts from one of the adrenal glands but can also develop in the head, neck, chest, abdomen, or Vertebral column, spine. Symptoms may include ...

in the bone or bone marrow demonstrating a partial response, minor response, or stable disease to prior therapy. The most common adverse reactions include

injection site reaction Injection site reactions (ISRs) are reactions that occur at the site of injection of a drug. They may be mild or severe and may or may not require medical intervention. Some reactions may appear immediately after injection, and some may be delayed ...

s or infusion-related reactions, pain,

tachycardia Tachycardia, also called tachyarrhythmia, is a heart rate that exceeds the normal resting rate. In general, a resting heart rate over 100 beats per minute is accepted as tachycardia in adults. Heart rates above the resting rate may be normal ...

(fast heart beats), vomiting, cough, nausea, diarrhea, decreased appetite, hypertension, fatigue, erythema multiforme, peripheral neuropathy, urticaria, pyrexia, headache, injection site reaction, edema, anxiety, localized edema, and irritability. The U.S.

Food and Drug Administration The United States Food and Drug Administration (FDA or US FDA) is a List of United States federal agencies, federal agency of the United States Department of Health and Human Services, Department of Health and Human Services. The FDA is respo ...

(FDA) granted the application for naxitamab

priority review Priority review is a program of the United States Food and Drug Administration (FDA) to expedite the review process for drugs that are expected to have a particularly great impact on the treatment of a disease. The priority review voucher program ...

breakthrough therapy Breakthrough therapy is a United States Food and Drug Administration designation that expedites drug development that was created by Congress under Section 902 of the 9 July 2012 Food and Drug Administration Safety and Innovation Act. The FDA's "b ...

, and,

orphan drug An orphan drug is a medication, pharmaceutical agent that is developed to treat certain rare medical conditions. An orphan drug would not be profitable to produce without government assistance, due to the small population of patients affected by th ...

designations. The FDA issued a priority review voucher for this rare pediatric disease product application and was later granted a priority approval.

Medical uses

Naxitamab is used in combination with granulocyte-macrophage colony-stimulating factor (GM-CSF) to treat people one year of age and older with high-risk neuroblastoma in bone or bone marrow whose tumor did not respond to or has come back after previous treatments and has shown a partial response, minor response, or stable disease to prior therapy.

History

The application for naxitamab was approved based on two clinical trials (Trial 1/NCT03363373 and Trial 2/NCT01757626) of 97 participants with high-risk neuroblastoma in bone or bone marrow. The trials were conducted at four centers in the United States and in Spain. Both trials enrolled participants who were previously treated for high-risk neuroblastoma in the bone or bone marrow. Some participants were not responding to the previous therapies anymore and some participants experienced the return of the cancer. Participants with cancer that was actively growing after their last therapy were not included in the trial. All participants received naxitamab in combination with granulocyte-macrophage colony-stimulating factor (GM-CSF) according to the trial schedule.

Medical uses

History

Society and culture

Legal status

References

External links