Regulatory Decision Summary for Genotropin
Review decision
The Regulatory Decision Summary explains Health Canada’s decision for the product seeking market authorization. The Regulatory Decision Summary includes the purpose of the submission and the reason for the decision.
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What was the purpose of this submission?
The purpose of this Supplemental New Drug Submission is to add the treatment of pediatric patients with Prader-Willi Syndrome (PWS) who have growth failure as an indication for Genotropin. The diagnosis of PWS should be confirmed by appropriate genetic testing. In addition, the sponsor south an indication for Genotropin for improvement of body composition in children with PWS.
At the time of this submission, Genotropin was authorized for long-term treatment for children with growth disturbance due to insufficient secretion of GH, Turner syndrome, small for gestational age (SGA) and idiopathic short stature (ISS). It is also indicated for replacement therapy in adults with growth hormone deficiency (GHD).
Why was the decision issued?
There is an unmet clinical need for treatment options for Children with Prader-Willi Syndrome (PWS). Although PWS is a rare genetic disorder, it is recognised as the most common genetic cause of life-threatening obesity. Children with PWS usually have a growth hormone deficiency which can be treated with growth hormone. But there are currently no growth hormone therapies approved in Canada for the treatment of PWS.
The safety and efficacy of Genotropin, a growth hormone therapy, for PWS was demonstrated in this submission by a combination of pivotal trials (CTN 94-8129-007 and TRN 91-019), three supportive trials in infants and children with PWS, and long-term information from a number of registries. Additional supportive information was obtained from clinical evidence from real use of Genotropin internationally over the last 20 years. Taken together, children with PWS who received growth hormone therapy experienced improvement in growth (height velocity) and body composition (distribution of fat and non-fat mass) which are expected to reduce the development of life-threatening obesity.
No new safety signals were identified in the studies and registries; and the adverse event profile are in line with the safety results described for growth hormone treatment of growth hormone deficient children.
Based on the evaluation of the submitted information, BRDD considered that the submission for Genotropin "for the treatment of pediatric patients, who have growth failure due to Prader-Willi syndrome (PWS diagnosed by appropriate genetic testing)" and "for improvement of body composition in children with PWS", complied with the Food and Drug Regulations and a Notice of Compliance (NOC) was recommended.
Decision issued
Approved; issued a Notice of Compliance in accordance with the Food and Drug Regulations.