Regulus Therapeutics Inc (NASDAQ: RGLS) is pleased after the U.S. Food and Drug Administration (FDA) made its determination and approved its orphan drug designation (ODD). It grants the substance to RGLS8429 after agreeing on its suitability to treat Autosomal Dominant Polycystic Kidney Disease (ADPKD).
The CEO of Regulus Therapeutics, Jay Hagan, discloses how matters look currently among patients. He says it is sad to watch how many patients struggle with limited treatment options and hopes the company could step in to offer working solutions.
Jay notes that almost half of the patients struggle with a condition called renal disease when they clock 60 years of age. He reveals that the state is dire because it is expensive to treat. Patients need to undergo transplantation or dialysis to be on the safe side.
The leader classifies ADPKD as a condition of high unmet need, which has expanded to become a nightmare for almost 160,000 Americans. Nevertheless, he looks forward to a future where they achieve a lot with their advancement of RGLS8429 through the clinic.
He hopes their efforts will serve patients with working treatments in the future. The needy patients continue to increase, and he hopes they will be time to help them.
The FDA’s Office of Orphan Products Development undertakes many activities, most of which ensure that patients benefit from working solutions and products. One of its responsibilities is to approve the orphan designation status to biologics and drugs that qualify.
The drugs and biologics that pass the test indicate safety and effectiveness. Proper treatment involves the diagnosis and the rest of the professional tasks. There are different ways to look at the various conditions they struggle with within their lifetime. Some of the conditions affect fewer than 200,000 people in the U.S. It is the rare diseases category.
The body awards the Orphan designation status purposefully. It gives the incentive to the manufacturers so that they can do more in the development of drugs that help patients struggling with rare diseases. The developers can gain a lot more. For example, they may win financial incentives as part of offering support to develop products to serve diverse needs.