Orphan Drugs: What Are They?


By Staff Reports

(DGIwire) – Anyone who spends time browsing through healthcare news stories is likely to come across the term “orphan drug.” But what is an orphan drug? According to the U.S. Food and Drug Administration (FDA), it grants Orphan Drug Designation status to products that treat rare diseases, providing incentives to sponsors developing drugs or biologics. The FDA defines rare diseases as those affecting fewer than 200,000 people in the U.S. at the time of designation.

Orphan Drug Designation provides the sponsor, or organization developing the drug, certain benefits and incentives, including a period of marketing exclusivity if regulatory approval of the drug is ultimately received for the designated indication, potential tax credits for certain activities, eligibility for orphan drug grants and the waiver of certain administrative fees. The European Union has its own version of the orphan drug designation program.

“The orphan drug program has been a boon to those drug developers who are interested in helping patient populations that have been historically underserved by the market,” says Gur Roshwalb, MD, the CEO of Akari Therapeutics, a clinical-stage biopharmaceutical company. “We are among those companies that have been the beneficiaries of this program in both the U.S. and the EU.”

In May 2016, Akari Therapeutics was granted an Orphan Drug Designation by the FDA for its lead clinical product, Coversin, for the treatment of Guillain-Barré Syndrome (GBS). According to the National Institutes of Health, GBS is a disorder in which the body’s immune system attacks part of the peripheral nervous system. In September 2016, the company was granted another Orphan Drug Designation by the FDA for Coversin for the treatment of paroxysmal nocturnal hemoglobinuria (PNH), an ultra-rare, life-threatening and debilitating disease of the blood.

Also in May 2016, the company received a positive opinion for Orphan Drug Designation for Coversin in the EU for Guillain-Barré Syndrome—and two months later, received another positive opinion for Coversin in the EU for PNH. These opinions were issued by the European Medicines Agency Committee for Orphan Medicinal Products. The opinions are then submitted to the European Commission for decision.

Coversin is designed to inhibit the action of the protein C5 and ecosanoid LTB4, molecules that play key roles in a component of the immune system called the complement system and in the inflammatory system. Ordinarily, the complement system helps disable and clear out foreign invaders and unwanted cells, but when C5’s variants are produced in unregulated numbers, the result can trigger life-threatening inflammatory and autoimmune conditions, such as GBS and PNH. Further, LTB4 attracts white blood cells (neutrophils) to the area of inflammation, increasing the inflammatory reaction. Coversin has shown promise as a combination C5 and LTB4 inhibitor.

“We look forward to potentially helping those groups of patients who may benefit from Coversin and the orphan program is an important component to bringing these therapeutics through clinical trials,” adds Roshwalb.

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