Fera Requests FDA Orphan Drug Designation for Naproxcinod for SCD

Fera Pharmaceuticals submitted an application to the U.S. Food and Drug Administration (FDA) requesting orphan drug status for naproxcinod, a potential treatment for for sickle cell disease (SCD).

Orphan drug designation is given to experimental treatments for rare diseases (those that affect fewer than 200,000 people in the U.S.) to support their development. The designation also comes with certain benefits, including financial incentives for clinical development and commercialization, special fee exemptions, as well as seven-year market exclusivity in the U.S. if granted regulatory approval.

Naproxcinod, originally developed by Nicox, is a non-steroidal anti-inflammatory treatment candidate specifically designed to release nitric oxide (NO), a gas that induces the relaxation and widening of blood vessels.

The experimental medication also is intended to block the activity of cyclooxygenase (COX) — an enzyme involved in the production of pro-inflammatory molecules, including prostaglandins.

With its dual mechanism of action, naproxcinod is expected to lower inflammation caused by the destruction of sickled red blood cells and improve blood flow.

Based on promising data from previous pharmacodynamics studies — studies that assess the effects a medication has on the body — in animal models of SCD, Fera decided to focus the development of naproxcinod on the treatment of vaso-occlusive crises (VOCs). VOCs are the most common complication of SCD caused by the rupture of blood vessels throughout the body.

The company is planning additional studies and other activities in preparation for a clinical trial to evaluate naproxcinod’s effectiveness in SCD patients. The study’s launch is pending on the FDA’s decision to grant orphan drug designation to naproxcinod, Nicox stated in a press release.

Naproxcinod already has been tested as a treatment for osteoarthritis in three completed Phase 3 trials (NCT00504127, NCT00541489, NCT00542555) that enrolled more than 2,700 participants.

In December 2015, Nicox and Fera entered into a collaboration agreement, in which Fera received exclusive rights to develop and commercialize naproxcinod in the U.S.

Under the terms of the agreement, Fera has sole responsibility for the clinical development, manufacturing, regulatory and marketing activities related to naproxcinod in the country. 

In turn, Nicox became eligible to receive $40 million if the annual sales of naproxcinod reach $1 billion for any indication in the U.S. In addition, the company is eligible to receive royalty payments of 7% on the product’s net sales in the country.