FDA Grants Orphan Drug Status to Naproxcinod for Pain Crises

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by Vanda Pinto PhD |

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Naproxcinod, an investigational therapy being developed by Fera Pharmaceuticals as a treatment for sickle cell disease (SCD), has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA).

Orphan drug status provides regulatory support and financial benefits to experimental treatments for diseases that affect fewer than 200,000 people in the U.S to accelerate their clinical development. Benefits include special fee exemptions, tax credits for clinical studies, and seven years of market exclusivity in the U.S. upon approval.

Fera first filed an application requesting naproxcinod be granted orphan drug status to treat SCD in 2020.

“We are extremely pleased that the FDA granted Orphan Drug status for naproxcinod as it now allows us to continue our development for sickle cell disease with the benefits that come along with this designation,” Frank DellaFera, CEO of Fera, said in a press release.

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Red Blood Cell Biomarkers May Predict SCD Severity, Crisis Risk

SCD is a rare condition in which abnormally shaped red blood cells stiffen and become sticky, blocking small blood vessels. As a result, blood flow to different areas in the body can become restricted, leading to tissue and organ damage.

Sickle cells also rupture and die prematurely, causing an increase in inflammation and a reduction in nitric oxide (NO), a gas that relaxes and widens blood vessels. Symptoms of SCD include pain, anemia, and recurrent infections.

Naproxcinod is an anti-inflammatory treatment that works by releasing NO and at the same time suppressing the activity of the enzyme cyclooxygenase, which is responsible for producing pro-inflammatory molecules, such as prostaglandins. The treatment is designed to reduce pain and inflammation, and improve blood flow.

It was first developed by Nicox which had previously tested its safety and efficacy for treating osteoarthritis in several Phase 3 trials (NCT00504127, NCT00541489, NCT00542555). The three trials combined enrolled more than 2,700 participants.

Nicox and Fera entered into a collaboration agreement in 2015 that granted Fera exclusive rights to develop and commercialize naproxcinod in the U.S.

Fera has conducted preliminary studies in SCD animal models to test the effects of naproxcinod. Promising findings prompted the company to further develop the treatment for vaso-occlusive crises (VOCs), a frequent complication of SCD.

“We congratulate Fera on achieving Orphan Drug Designation for naproxcinod, which is a very important step in being able to develop this molecule as a potential treatment for sickle cell disease,” Michele Garufi, CEO and chairman of Nicox, said.

“Fera has already carried out pre-clinical development work on naproxcinod in models of sickle cell disease, and the extensive clinical package already developed by Nicox positions the molecule — the first that was taken into clinical development by our Company — for an accelerated development,” Garufi added.