FDA Approves Increlex for Short Stature
Yesterday, the U.S. FDA approved Tercica’s Increlex (recombinant hIGF-1) as an orphan-status treatment for short stature in children with severe primary IGF-1 deficiency. The press release and Tercica’s website can provide details on the drug and why it is important to Tercica, to the U.S. community of pediatric endocrinologists and to their short-stature patients who will benefit from the drug. I simply wanted to express my own views of the historic importance of this event, which was appropriately understated by the company during their investor conference today. This is the first new treatment for short stature in over 20 years, since the approval of recombinant human growth hormone. More importantly from the historical perspective, this particular new treatment is native rhIGF-1, one of the first bioengineered human proteins ever created (six years after recombinant human insulin was synthesized). It has taken a long time for its appearance in the pharmacy for many reasons. Genentech spent quite a long time debating the clinical and commercial value of the product, even as they conducted and sponsored clinical trials in a wide variety of clinical conditions. While they were debating, long-term treatment data accrued from children with severe short stature due to a primary deficiency of IGF-1. It was these data that provided the basis for yesterday’s landmark approval. Increlex undoubtedly will make a major difference to the lives of many children with severe primary IGF-1 deficiency, who currently do not respond to other therapies (about 6,000 in the U.S.). What is particularly exciting for Tercica and its investors is the potential for Increlex to be used to treat a broader population of IGF-1 deficient children who currently must use very high doses (at very high cost) of growth hormone. This potential is currently being studied by Tercica and its clinical investogators. In addition to Tercica’s internal programs, we can expect that Increlex will be studied and used “off-label” in a number of other conditions with unmet needs including: short stature that is poorly responsive to GH, severe insulin-resistant diabetes, acute and chronic renal failure, amyotrohic lateral sclerosis and other neuromuscular diseases, osseous defects and fractures and severe injury and other states associated with hypercatabolism. This is indeed an historical and important occasion. (Disclaimer: I, and entities I control, own stock in Tercica).
