Regulatory Strategy

Our Regulatory Strategy and Orphan Drugs

We have a diversified portfolio of products and product candidates, which were developed or are being developed using our proprietary ALT® platform. Our regulatory strategy is focused on seeking the most efficient pathway for obtaining drug approval while obtaining the best available protections for our product candidates. As each of our current proprietary product candidates consists of either active ingredients already approved by the Food and Drug Administration (FDA) for similar indications or active ingredients for which clinical proof of safety or efficacy is available in published literature, our current development strategy is primarily focused on availing ourselves of the benefits of Section 505(b)(2) of the FDA for regulatory approval for our pipeline product candidates.

As part of our commitment to seek the best available protections for our product candidates, we have also sought, and been granted orphan drug designation for both SC411, which targets pediatric sickle cell disease and SC403, which targets malabsorption as a result of short bowel syndrome. As a result of these orphan drug designations, upon approval of an NDA for any of these orphan drugs, we will be granted seven years of market exclusivity.