Rare Disease Market Revs Up

Young scientist pipetting in life science laboratory.

(DGIwire) – When a disease affects fewer than 200,000 Americans, it is considered “rare.” According to PhRMA, 30 million Americans—10 percent of the population—have one of the 7,000 known rare diseases. Rare diseases have been a focus of drug development for decades, and since the 1980s, there have been financial, legal and legislative incentives—such as the Orphan Drug Act—for companies to enter the market.

As recently reported in PharmaVOICE, the rare disease market is growing faster than that for traditional drugs. According to Scalar Market Research, the global orphan drug market is expected to grow at a steady annual growth rate of 11.4 percent over the next six years. At this rate, the market will expand from nearly $112 billion in 2016 to $214 billion by 2022. In the past decade, PharmaVOICE reports, more than 230 new orphan drugs were approved by the U.S. Food and Drug Administration (FDA). In 2015 alone, nearly half—47 percent—of novel new drug approvals were for rare diseases.

“There are several factors at play as to why there is increased focus on the rare disease market,” says Gur Roshwalb, M.D., CEO of Akari Therapeutics. “These include legislation on orphan diseases that provides benefits to companies at the regulatory level, as well as the general advance in sciences.”

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.

Recently, the FDA allowed an Investigational New Drug Application (IND) for the clinical development of Coversin in patients with PNH. The FDA’s allowance of the IND permits Akari to expand its clinical program for the development of Coversin in PNH to the United States. Akari has one currently treated eculizumab-resistant PNH patient who has been on Coversin for approximately 11 months pursuant to an approved clinical protocol in the Netherlands, and it plans to open this ongoing Phase II trial of Coversin in eculizumab-resistant PNH in the United States.

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.

“An array of factors, especially the regulatory support, makes it much more achievable for a small company to advance a drug in the rare disease sector, and we are enthusiastic about what the future holds for the areas we are focusing on,” Roshwalb adds.

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