Pediatrics Xagena

New indication for Humira approved by FDA: Crohn’s disease in pediatric patients

The FDA ( Food and Drug Administration ) has approved a new indication for Adalimumab ( Humira ) for the treatment of moderate to severe Crohn’s disease n pediatric patients 6 years of age and older.

Humira can be used to reduce signs and symptoms of Crohn’s disease, as well as achieve and maintain clinical remission in children who experienced treatment failure on other therapies.
Adalimumab is the only biologic approved for use in this patient population that can be administered at home.

Crohn’s disease commonly involves the end of the small intestine and the large intestine. The disease can appear at any age, but is most often diagnosed in adolescents and young adults between 15 and 35 years old.
An estimated 38,000 children and adolescents have Crohn’s disease in the United States.

Symptoms of pediatric Crohn’s disease are similar to those in adults and can include abdominal pain, weight loss and diarrhea. Additionally, when Crohn’s disease is diagnosed in childhood, it may be more extensive than when it is diagnosed in adulthood.

Adalimumab works by inhibiting tumor necrosis factor-alpha ( TNF-alpha ) and can be self-administered after proper injection training and with appropriate physician monitoring.

Because there is no cure, one of the treatment goals in pediatric Crohn’s disease is to induce and maintain clinical remission.

The new indication was supported by the phase III IMAgINE-1 trial, which has evaluated multiple dosing strategies of Adalimumab to induce and maintain clinical remission in children with moderately to severely active disease for whom certain other treatments had not been efficacious.
In the multicenter, randomized safety and efficacy study, the researchers studied 192 patients with Pediatric Crohn’s Disease Activity Index ( PCDAI ) scores of greater than 30, who were not responding to conventional treatment.
The children received two doses of open-label induction therapy two weeks apart with subcutaneous Adalimumab ( 160 and 80 mg, or 80 and 40 mg, for body weight greater than or equal to 40 or less than 40 kg ).

At four weeks, 188 patients were assigned to groups based on achievement of clinical response ( defined as decrease in PCDAI greater than or equal to 15 points from baseline; 155/188 [ 82.4% ] ) and prior exposure to Infliximab ( 82/188 [ 43.6% ] ).

Maintenance therapy was double-blinded, with Adalimumab at high dose ( 40 or 20 mg for body weight greater than or equal to 40 or less than 40 kg; n=93 ) or low dose ( 20 or 10 mg for body weight greater than or equal to 40 or less than 40 kg; n=95 ) every other week for 48 weeks.
One hundred fifty-two patients ( 80.9% ) completed all 26 weeks of therapy.
At end of treatment, 63 patients ( 33.5% ) were in clinical remission ( PCDAI less than or equal to 10 ), with no significant difference between high- and low-dose groups ( 36/93 [ 38.7% ] vs 27/95 [ 28.4% ]; P=0.075 ) ( Xagena )

Source: FDA, 2014