Apellis Pharmaceuticals Inc (NASDAQ:APLS) shares surged in pre-market deals after the Nasdaq-listed global biopharma announced that the US regulator had accepted the company's amendment to its new drug application (NDA) for a therapy for geographic atrophy (GA) - an advanced form of age-related macular degeneration (AMD).
GA is a leading cause of blindness that impacts more than five million people worldwide, including one million in the US. There are currently no approved treatments.
The firm's Pegcetacoplan therapy has already been granted Fast Track designation by the US Food and Drug Administration (FDA) for the treatment of GA.
Earlier in November, Apellis, which has a market cap of US$5.5 billion, revealed it had decided to submit 24-month efficacy data from the Phase 3 DERBY and OAKS studies as part of the NDA review.
That data showed increasing and consistent effects with every-other-month and monthly pegcetacoplan treatment and a favorable safety profile in both studies.
Best product profile at launch
"With the inclusion of the 24-month data, we have the potential to have the best product profile at launch for pegcetacoplan, with minimal impact to launch timing," said Cedric Francois, the CEO and co-founder of Apellis.
"We appreciate the opportunity for the FDA to review these data and look forward to working with the agency to bring this first potential therapy to people living with GA as quickly as possible."
Pegcetacoplan is an investigational, targeted C3 therapy designed to regulate excessive activation of part of the body’s immune system, which can lead to the onset and progression of many serious diseases.
Apellis said it remained on track to submit an EU marketing authorization application, which will also include the 24-month results, to the European Medicines Agency by the end of 2022.
Shares in New York added over 13% to stand at US$49 each.
Contact the writer at giles@proactiveinvestors.com