Innovation in Rare Diseases


Rare diseases in the U.S. are defined as conditions which affect less than 200,000 people. Despite their name, 1 in 10 U.S. citizens suffers from one of the 8,000 rare diseases. Recently, with the support of the FDA, researcher­s and pharmaceut­ical companies have been able to bring innovative treatments to the market that have given hope and relief to patients who previously had limited options. “Rare disease innovation has been the success story of the last two or three decades,” says William H. Lewis, Chair & CEO of Insmed. Nowadays, more and more companies are doing research in this field. Acasti is a specialty pharma whose strength lies in reformulat­ing already marketed drugs. Its President and CEO, Jan D’alvise, claims “they will have two drugs ready to move into Phase 3 in 2023, and a third drug which is a topical spray used for a pain indication for a disease called postherpet­ic neuralgia (the single largest cause of suicide in people over the age of 70 just due to the severe pain it causes) going into Phase 2.” Another small biotech, PTC Therapeuti­cs, is already successful­ly commercial­izing a drug that targets Duchenne muscular dystrophy, “the first approved gene therapy in the world directly injected into the brain and the first approved oral treatment for spinal muscular atrophy developed from PTC’S Splicing platform,” explained CEO Stuart Peltz. The 25-year-old company attributed its success to innovation, adding that “in this day and age, you definitely do not want to make the best horse and buggy when we have flying cars.”

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