Apellis Pharmaceuticals said that its APL-2 drug has received approval from the Food and Drug Administration to be designated an “orphan drug” for two blood-related autoimmune diseases.
The designation qualifies the Crestwood, Ky.-based pharmaceutical company for development incentives, including tax credits.
Apellis said the FDA approved the orphan drug designation for APL-2 to treat warm autoimmune hemolytic anemia (wAIHA) and cold agglutinin disease (CAD). In both conditions, the body’s immune system mistakenly attacks and destroys its own red blood cells. Both can lead to anemia and other associated signs and symptoms, according to the National Institutes of Health. In severe cases, wAIHA can lead to heart failure.
APL-2 previously had received the designation for a kidney disorder. And for geographic atrophy, an age-related form of macular degeneration, a blinding disease, APL-2 has a fast track designation, which indicates an expedited review and is given to drugs that treat serious conditions and fill an unmet medical need.
Apellis shares rose 1.04 percent Wednesday, closing at $13.58. The S&P 500, Dow Jones industrial average and Nasdaq all fell slightly.
Apellis had said in December that new data from clinical studies showed that the drug provided “meaningful improvement” in the treatment of the autoimmune diseases.
CEO Dr. Cedric Francois at the time told Insider that the results reinforced company leaders’ hope that the drug would provide doctors with new weapons for life-altering conditions for which patients have few or no treatment options. Patients with wAIAH and CAD have no FDA-approved therapies, he said.
In a presentation at the Muhammad Ali Center on Wednesday, Francois credited the kindness and authenticity of Louisvillians as a kind of “magic sauce” that has helped propel Apellis toward success.
Francois told attendees of the Venture Connectors luncheon that he had received lots of help from Louisvillians in the last 18 years, joking that he had spent many a night with fellow entrepreneurs at watering holes such as Garage Bar.
After the presentation, he said that despite his love for Louisville, the tight labor market has prompted Apellis to open divisions in San Francisco and Boston for better and faster access to engineering and medical talent.
Especially for a company that needs to ramp up quickly, the talent pool in Kentucky is too small, Francois said, though he added that the problem is not isolated to the commonwealth.
“I consider it more of a national problem,” he said.
The company’s European division, which recently received a new leader, has not encountered those labor issues, Francois said, primarily because it focuses on production. The CEO said that Apellis certainly plans, at this point, to be in charge of commercialization of its drugs.
While APL-2 is in late-stage clinical trials, Francois said that it’s difficult to say whether Apellis can get the drug to commercialization. From the early days of the company, he said he has told investors that Apellis will do its best to get drugs to market, but he also warns them that the industry is complicated and unpredictable, primarily because it deals with human biology.