![]() Learn more about orphan designations and their incentives in this blog article. Securing an orphan designation carries with it determinate advantages endeavoured to incentivizing the development of new medicines for rare diseases, since orphan designation applications can be submitted early in the development of a drug and clinical data are not required. Cooperation between sponsors and regulators on the development of orphan drug ATMPs could support successful outcomes.īecause a return on the investment into the development of the medicine for rare diseases may not be obtained, the development of medicines designated for small patient populations may have restricted commercial value. Consequently, a rise in requests for services offered by the European Medicines Agency could be expected. Indeed, gene therapy medicinal products seem to be of greatest interest possibly due to the potential they have for curing certain rare diseases, in particular those caused by single gene defects. ![]() ATMPs can represent a more specific and causal/targeted treatment for many rare diseases for which the specific underlying cause is known, e.g., a gene defect therefore, this trend is expected to continue. Since the establishment of the Advanced Therapies Regulation in 2008 in the European Union, ATMPs are emerging as a growing and increasing class of innovative medicinal products which potentially present an alternative approach to traditional small molecule medicinal products or biologicals (learn more about ATMPs and their classification in this blog article). ![]() Orphan Designation of ATMPs for Rare Diseases: MPS II Case Study Many advanced therapy medicinal products (ATMPs) in development in the EU are for rare diseases and conditions. ![]()
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