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Orphan drugs are pharmaceutical products developed to treat rare medical conditions or diseases that affect a relatively small number of people. These conditions are often referred to as “orphan diseases.” Due to their rarity and the limited potential for profit, traditional drug development approaches might not be financially viable for these diseases.
To incentivize the development of treatments for orphan diseases, many countries have established special regulatory and financial frameworks. These frameworks provide benefits such as extended market exclusivity, reduced regulatory fees, and tax incentives to pharmaceutical companies that develop orphan drugs. This encourages research and development efforts in this neglected area of medicine.
The goal of orphan drugs is to provide effective therapies for patients with rare diseases who would otherwise have limited or no treatment options. By promoting the development of orphan drugs, these initiatives aim to improve the lives of individuals affected by rare diseases and address unmet medical needs in these populations.