ORPHAN DRUG DESIGNATION
There are many advantages in addressing a rare disease that qualifies for an orphan drug designation (ODD). One of the benefits is enhanced intellectual property protection via added market exclusivity corresponding to 10 years in Europe and Japan, and 7 years in the US. Other advantages include regulatory assistance, higher rates of approval, higher pricing scenarios, faster uptake and more potent market share, and smaller resource needs for marketing. While the smaller patient pool for rare diseases may create certain trial recruitment challenges, patients are often concentrated into known clinical centers of excellence and the ODD allows the sponsors to run smaller trials. Additional incentives are specific to concrete regions – for example, in the US, companies are offered certain government fee exemptions, a 25% tax credit on clinical trial costs, and FDA-related benefits including protocol assistance and the Fast Track procedure for evaluating product registration files.
Lipum’s proposed treatment and drug candidate can qualify for an ODD. Lipum is evaluating the possibility to address Juvenile Idiopathic Arthritis (JIA) as the first indication for the product.