Tumour-induced osteomalacia (TIO) is a rare disorder characterized by weakened bones due to the excess secretion of a hormone called fibroblast growth factor 23 (FGF23) by a benign tumour. This disorder affects both children and adults, and symptoms include bone pain, muscle weakness, and fractures. The TIO market is small but growing, with a limited number of treatment options available.
One of the key players in the TIO market is Ultragenyx Pharmaceutical Inc., which has developed Crysvita (burosumab), a monoclonal antibody that targets FGF23 and is approved for the treatment of TIO in both adults and children. Crysvita is the first and only FDA-approved treatment for TIO, and it has shown promising results in clinical trials, improving bone mineral density and reducing pain and fractures in patients with TIO.
Other companies developing treatments for TIO include Ipsen Biopharmaceuticals, which is developing a somatostatin analogue called pasireotide, and Novartis, which is developing a small molecule inhibitor of FGF23 called KRN23. These treatments are currently in clinical trials, and if successful, they could provide additional treatment options for patients with TIO.
In addition to the development of new treatments, there is also a growing awareness of TIO among healthcare providers and patients. This is due in part to the efforts of patient advocacy groups such as the TIO Patient Registry and the TIO Support Group, which work to raise awareness of the disorder and provide support and resources for patients and their families.
However, there are also challenges facing the TIO market, including the high cost of treatment and the limited number of patients with TIO. Crysvita, for example, has a list price of approximately USD 400,000 per year, which may limit its accessibility to some patients. In addition, the rarity of the disorder means that there is a limited patient population, which can make it challenging for companies to recoup their research and development costs.
In conclusion, the TIO market is a small but growing segment of the healthcare industry, driven by factors such as the increasing prevalence of TIO, the growing awareness of the disorder, and the development of new treatment options. While there are challenges facing the market, the potential benefits of these treatments in improving the lives of patients with TIO make it an important area of innovation in the healthcare industry.