FDA Clears Taliglucerase Alfa for Kids With Gaucher Disease

Megan Brooks


September 02, 2014

The US Food and Drug Administration (FDA) has approved taliglucerase alfa (Elelyso, Protalix BioTherapeutics, Pfizer) as a long-term enzyme replacement therapy for children with type 1 Gaucher disease, the companies announced August 28.

The FDA approved taliglucerase alfa for use in adults with type 1 Gaucher disease in 2012.

"While Type 1 Gaucher disease can manifest in childhood or adulthood, the disease more often presents during childhood," Paige Kaplan, MB BCh, from the Children's Hospital of Philadelphia, Pennsylvania, said in a news release. "It is important that children with this disease have access to a range of FDA-approved treatment options that are effective."

Gaucher disease is a rare disease involving a deficiency in glucocerebrosidase, which leads to the accumulation of lipids in various organs, including the spleen, liver, and kidneys. Type 1 Gaucher disease affects about 6000 people in the United States.

The safety and efficacy of taliglucerase alfa were demonstrated in 14 children aged 2 to 16 years with type 1 Gaucher disease in 2 separate clinical trials. One of the trials involved 9 treatment-naive children, and the other involved 5 children who switched from another enzyme replacement therapy (imiglucerase) to taliglucerase alfa.

The recommended dosage of taliglucerase alfa for treatment-naive adult and pediatric patients 4 years of age and older is 60 U/kg body weight administered every other week as a 60- to 120-minute intravenous infusion, the companies say.

They recommend that patients previously receiving stable-dose imiglucerase begin taliglucerase alfa at that same dose when they switch from imiglucerase to taliglucerase alfa. Dosage adjustments can be made on the basis of achievement and maintenance of each patient's therapeutic goals.

Serious hypersensitivity reactions, including anaphylaxis, have occurred in some patients treated with taliglucerase alfa, the companies note.

The most common adverse reactions for taliglucerase alfa in clinical trials were itching, flushing, headache, joint pain, pain in extremity, abdominal pain, vomiting, fatigue, back pain, dizziness, nausea, and rash. Vomiting occurred more often in children than adults.


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