PDUFA Date Approaches for Apellis Drug

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PDUFA Date Approaches for Apellis Drug

Apellis Pharmaceuticals’ potential blockbuster drug for the treatment of geographic atrophy secondary to age-related macular degeneration, intravitreal pegcetacoplan, is set to have its updated Prescription Drug User Fee Act goal date on February 26th. (The FDA was previously expected to deliver its verdict by November 26th, but in a move that stunned industry insiders and the stock market, Apellis submitted unsolicited, extended 24-month efficacy data on top of the 18-month and 1-year results in the original application package.)

“With the inclusion of this 24-month data, we have the potential to have the best product profile at launch for pegcetacoplan, with minimal impact to launch timing,” said Apellis CEO and Co-Founder Cedric Francois, MD, PhD, in a November press release. Read more.

Some Background

So, who is Apellis and what is pegcetacoplan? Apellis is a small biotech company that launched in 2009. Pegcetacoplan is an investigational, targeted C3 therapy designed to regulate excessive activation of the complement cascade, part of the body’s immune system, which can lead to the onset and progression of many serious diseases.

CAN YOU RELATE

More big news in the Davidson household! After extensive research to figure out which dog would fit in best with our growing family, we settled on a Goldendoodle, who came home to live with us last weekend. We named him Bronner, after our family’s favorite store, Bronner’s Christmas Wonderland, in Frankenmuth, Michigan.

Now, what you’re about to read may sound weird, but bear with me. I started thinking about the parallels between pets and practice settings. Some animals (cats) are generally the “set it and forget it” type. Make sure you give them food, water, and a little TLC here and there, and they’re a wonderful addition to your family without requiring too much of a time investment. This is much like the draw of corporate eye care. Show up during normal business hours, provide superb patient care, give the business/optical a little attention, and then go home to enjoy life while letting the corporate entity handle most of the details. Not a bad deal!

What about dogs, though? To me, dogs are the private/group practice modality in eye care. Some require constant attention, and much of your life revolves around them. Bronner seems to fit that bill thus far. He needs frequent walks and stimulation, unending games, and he chews on everything in the house—I imagine that’s what private practice ownership is like!

I see a lot of similarities between OD/MD group practice and my best friend’s Labrador Retrievers. The breed is friendly, active, and outgoing, and is well known for being a wonderful housemate loved by the whole family. Sounds like most of the docs in my clinic! Despite coming from multiple educational backgrounds, we all socialize and play well together.

What about those clinics with just a few different specialties? The vision therapy, myopia management, and medical practices? I’d say they’re Bulldogs—powerhouses in our profession!

OUTSIDE THE LANE

FDA Ends Animal Testing Requirement for Drugs

With the recent passage of the FDA Modernization Act 2.0, the FDA no longer requires all drugs to be tested on animals before human trials.

According to a statement from bill sponsor Senator Rand Paul, R-KY, the new law will help end the “needless suffering and death of animal test subjects” and “will get safer, more effective drugs to market more quickly by cutting red tape that is not supported by current science.”

What are the alternatives to animal testing? The three most well-known alternatives are organ-on-a-chip technology, cell cultures called organoids, and computer models. Read more.