Orphan Drug Designation to Foster Rising Consumption of Drug

Orphan drugs are developed specifically for the treatment of rare medical conditions that are known as orphan diseases. Approvals of the US Food and Drug Administration (FDA) play a crucial role in the implementation of drugs for treatment. The new and first-of-its-kind protein aggregation inhibitor LMTX developed by TauRx has reached Phase 3 clinical development for neurodegenerative disease treatment. Backed by the company’s three decades of research, this drug was granted an approval by FDA getting an Orphan Drug Designation (ODD) for treating frontotemporal dementia.

Protein aggregation is widely considered to be a driver in dementia development, owing to which, protein aggregation inhibitors are used for undoing the process. This approach helps in potentially reducing the speed at which underlying disease pathology spreads, consequently helping preserve the function, cognition and quality of patient’s life. An active substance in LMTX is reflected to be active against TDP-43 protein aggregation apart from primarily focusing on tau protein.

Addressing Rare Neurodegenerative Conditions

Professor Claude Wischik, Excutive Chairman of TauRx along with Professor of Old Age Psychiatry at the University of Aberdeen stated that the FDA Orphan Drug Designation of the drug acts as a favorable step for clinical researchers, care teams, and patients, reinforcing their persistent research for disease-modifying treatment for the in-adequately addressed category of rare neurodegenerative conditions. LMTX has also been granted the Orphans Designation by the European Medicines Agency (EMA) in 2010.

FDA approvals of the ODD status are provided only to those medicines that are developed for diagnosis, treatment, or prevention of rare disorders. ODD is recognized to offer incentives for sponsors for the development of products intended for rare diseases.

Research Unveils Promising Future for Orphan Drugs

Similarly, other orphan drugs are undergoing key developments that are boosting the capabilities of the clinical and medical science for the diagnosis, treatment, and prevention of rare orphan diseases. A clinical-stage biopharmaceutical company, Entera Bio recently announced the completion of the part 1 of Phase 2 clinical trials of Natpara, an injectable orphan drug developed for treating the rare endocrine disease, hypoparathyroidism. A recent market study presented by Future Market Insights reveals the developments in treatment of hypoparathyroidism. The senior team of analysts state that favorable government schemes related to orphan drug, tax incentives for qualified clinical trials, along with regulatory approvals are some major factors that could translate in promising earnings for companies. Such support combined by the process of gaining regulatory approvals is expected to paint a favorable picture with respect to treatment of rare diseases.

Key insights and opportunities in the hypoparathyroidism treatment market could be availed here.

Published by Sabyasachi Ghosh

Sabyasachi Ghosh is an experienced market research analyst and consultant, with over six years of experience in end-to-end project management. He has worked at numerous leadership positions and has a vast experience of compiling high-quality market research reports. Sabyasachi is an authoritative voice in the market research sector, and has been cited in top industry publications. He is a travel junkie, and loves to travel far and wide with his friends.

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