Expert Available: What Are the Challenges of Treating Orphan Diseases?

In January 2016, the U.S. Food and Drug Administration (FDA) announced it had approved 21 new orphan drugs to treat rare diseases in 2015, nearly half (47 percent) of all novel new drugs approved for the year. This was the second consecutive year in which the FDA approved a record-setting number of orphan drugs for rare diseases. This highlights an important trend: after decades of neglecting rare disease research, biopharma companies are increasingly identifying this category as an attractive development pathway. This is great news for the tens of millions of patients suffering from one of approximately 7,000 rare diseases recognized worldwide. There are numerous factors contributing to this trend but regulatory incentives—such as accelerated approval and waived fees—are a primary driver.

Gur Roshwalb, MD, the CEO of Akari Therapeutics, is developing treatments for a wide range of severe and orphan autoimmune and inflammatory diseases. Akari’s lead product, Coversin, is a small molecule that inhibits molecular activity responsible for rare diseases such as paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS) and Guillain Barré syndrome (GBS). Akari has received orphan drug designations for Coversin from the FDA for treatment of GBS and PNH; and a positive opinion for Orphan Drug Designation for Coversin in the EU for GBS and PNH.

“Orphan drug designation has transformed our approach to orphan diseases,” says Dr. Roshwalb. “In contrast to the era before this designation was established, it is easier to obtain the support necessary to sustain clinical studies in the pursuit of new therapies. This means greater attention can be paid to the specific challenges of assessing the safety and efficacy of these therapies.”

More About Dr. Gur Roshwalb

Dr. Roshwalb joined Akari as CEO in March 2013. From April 2008 to February 2013, he was a Vice President at Venrock, where he was an investment professional on the healthcare team investing in both private and public companies. From May 2004 to March 2008, he was a vice president and equity analyst at Piper Jaffray, publishing research on specialty pharmaceutical companies. Prior to Piper, Dr. Roshwalb was in private practice in New York and Board Certified in Internal Medicine. He received his MD from Albert Einstein College of Medicine and his MBA from the NYU Stern School of Business. Dr. Roshwalb trained in internal medicine at the Mount Sinai Medical Center, where he also served as Chief Resident in 1997-1998.


Orphan disease, Food and Drug Administration, FDA, Gur Roshwalb, Akari Therapeutics, autoimmune, inflammatory, Coversin, paroxysmal nocturnal hemoglobinuria, PNH, atypical hemolytic uremic syndrome, aHUS, Guillain Barré syndrome, GBS


Interested in conducting an expanded interview?


Laura Radocaj

(212) 825-3210

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