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Kv1.3 Therapeutics is pursuing the use of its novel therapeutics in rare or “orphan” diseases that are often overlooked by large pharmaceutical companies.  Rare diseases are defined as a disease with less than 200,000 patients in the US or in Europe a disease impacting less than 5 in 10,000 patients.  Patient need for new drug options is very high for rare diseases.  While rare diseases were largely overlooked by the industry for many decades, over the past years, the scientific community and industry has been drawn focus on developing drugs for these previously “orphan” diseases. Today, there can be advantages to pursuing a rare disease registration strategy including tax credits on research expenses, extended market exclusivity periods, and regulatory technical assistance on the submission. But most importantly, orphan drug development timelines and costs to marketing approval can be dramatically reduced. This would allow us to provide patients with a new treatment alternative more rapidly than with a traditional drug development timeline.

Small patient populations do not mean small opportunities

There are an estimated 7,000 rare diseases worldwide affecting approximately 30M patients in the US and 350M people worldwide.  Rare diseases represent a significant medical need in the US and around world.  While each individual disease may be rare, in total rare diseases touch hundreds of millions of families around the world.  Drugs being developed to treat these rare diseases represent major opportunities both for the companies developing them and for the patients that need these drugs.

We are initially focusing our clinical activity on myositis, however the application of dalazatide may extend to other rare diseases as well as more common autoimmune diseases such as rheumatoid arthritis, and many other immune disorders.