Self-administered Crysvita receives positive CHMP opinion

pharmafile | May 10, 2021 | News story | Sales and Marketing CHMP, EMA 

Kyowa Kirin’s drug for a rare metabolic bone disease, Crysvita, has received a positive opinion from the EMA’s Committee for Medicinal Products for Human Use (CHMP), for self-administration.

The CHMP indicated that, in addition to healthcare professionals, some patients or carers may be suitable to administer Crysvita (burosumab), for the treatment of X-linked hypophosphataemia (XLH) in children and adolescents aged 1 to 17 years with radiographic evidence of bone disease, and in adults.

Abdul Mullick, President of Kyowa Kirin International, said: “The global pandemic has seen huge demands placed on healthcare systems, which has greatly impacted patient care. COVID-19 has also made some patients reluctant to visit healthcare facilities for fear of infection or of interrupting critical care of patients diagnosed with coronavirus.

“One of the ways in which Kyowa Kirin can help is by giving healthcare professionals increased flexibility to help patients receive treatment in the way that best suits their needs. The self-administration of Crysvita is a great example of meeting physician and patient needs and is one of the ways in which we are delivering on our purpose, to make people smile.”

Crysvita is administered by subcutaneous injection and treatment will still need to be initiated by a physician experienced in the management of patients with metabolic bone diseases. Subsequently, if the patient is receiving a stable dose, the physician may recommend that administration can be performed by the patient or carer following appropriate training.

XLH is a rare, genetic disease that causes abnormalities in the bones, muscles, and joints. It is not life-threatening, but its burden is life-long and progressive, and it may reduce a person’s quality of life.

People with XLH have a genetic defect on the X-chromosome, which causes an excessive loss of phosphate through the urine and poor absorption from the gut, resulting in chronically low levels of phosphate in the blood. Phosphate is a key mineral needed for maintaining the body’s energy levels, muscle function, and the formation of healthy bones and teeth.

While there is no cure for XLH, therapies aimed at helping to restore phosphate to normal levels within the body may help to improve the symptoms of the disease.

Dr Raja Padidela, Consultant Paediatric Endocrinologist at Royal Manchester Children’s Hospital, said: “Self-administration of medicines can help some patients maintain their independence and also empower them to take better control of their condition. In addition to XLH patients with mobility issues, this news could also be of benefit to parents of younger children with XLH, who may have less time to attend appointments at health centres or accommodate visits from home care nurses for administering injections.

“Many injectable treatments for chronic diseases, such as insulin for the treatment of diabetes mellitus, are successfully self-administered by patients. For XLH patients, the treating physician will assess which patients are appropriate, ensuring those self-administering are competent and comfortable doing so.”

The European Commission will review the CHMP recommendation and a final decision on the variation application to add the option of self-administration of Crysvita to the current approved SmPC is expected in the coming months.

Kat Jenkins

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