BY OUR PHARMA CORRESPONDENT
2 September, 2005: Increlex, new drug to treat dwarfish kids has been approved in US. The Brisbane-based Tercica’s Increlex will be the first new drug in 30 years for the treatment of abnormally short stature in children.
The approval was granted based on the clinical evidence submitted by the biotechnology company to the Food and Drug Administration. Data showed that
Increlex prompted growth in children who did not respond to injections of growth hormone, the standard treatment.
In clinical trials, patients given the drug through twice-a-day injections grew an average of about an inch more a year than patients not given the treatment.
The active ingredient of Increlex, mecasermin [rDNA origin] is identical to the natural hormone, IGF-1, which the body produces in response to stimulation by growth hormone. Insulin-like growth factor-1, or IGF-1, is the direct mediator of growth hormone's effect on statural growth and must be present in order for children's bones, cartilage, and organs to grow normally. Severe Primary IGFD is a growth hormone-resistant state characterized by abnormally low blood IGF-1 levels in the presence of normal or elevated growth hormone.
Without adequate IGF-1, children cannot achieve height within the normal range for the long-term treatment of growth failure in children with severe primary IGF-1 deficiency (Primary IGFD) or with growth hormone (GH) gene deletion who have developed neutralizing antibodies to growth hormone. The FDA also designated as an orphan drug for severe Primary IGFD.
Children with severe Primary IGFD have height and serum IGF-1 levels that are more than three standard deviations below normal. They are not growth hormone deficient, and, because they are resistant to the effects of growth hormone, they cannot be expected to respond adequately to growth hormone therapy. In both children and adults, severe Primary IGFD can lead to a range of other metabolic disorders including lipid abnormalities, decreased bone density, obesity and insulin resistance.
Nearly 6,000 children in the United States have the specific condition for which the F.D.A. approved the drug, although some doctors expect Increlex to be more broadly prescribed to children with less severe growth abnormalities, it is estimated.
The drug's price has not yet been set, but it is expected to be similar to that for growth hormone, which is roughly $20,000 a year.
Tercica licensed Increlex from Genentech, which did the original development work in the 1980's and 90's and will receive an undisclosed royalty on all sales.
Currently, Tercica is enrolling patients in clinical trials for the treatment of less severe growth disease, which would expand the potential market to about 35,000 children. Because the F.D.A. permits so-called off-label prescriptions, though, some pediatric endocrinologists are likely to begin recommending the drug immediately for patients who fail to respond to growth hormone.
Insmed, a biotechnology company developing a competing drug, had filed a petition with the F.D.A. challenging Increlex's safety.
BY OUR PHARMA CORRESPONDENT
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