Amid a parade of regulatory endorsements in Europe, Apellis Pharmaceuticals’ ophthalmology prospect pegcetacoplan has been left in the cold once again.
Europe’s Committee for Medicinal Products for Human Use (CHMP) has confirmed its negative stance on pegcetacoplan in geographic atrophy (GA), an advanced form of age-related macular degeneration that can lead to blindness, Apellis said Friday.
This marks the third rejection this year for pegcetacoplan in Europe, where there are currently no approved therapies for GA. The CHMP initially snubbed the drug in January and then again in June. Apellis has spent much of the year appealing the agency’s decisions.
Apellis is “deeply disappointed” by the latest rejection, the company’s co-founder and CEO, Cedric Francois, M.D., Ph.D., said in a statement. The negative opinion was handed down despite “broad support” for pegcetacoplan from the European retina community and “multiple dissenting votes” by CHMP members, the CEO said.
Pegcetacoplan was initially approved in the U.S. for the rare blood disorder paroxysmal nocturnal hemoglobinuria in 2022 under the brand name Syfovre. Syfovre subsequently won an FDA nod in GA in February of 2023.
In the U.S., Syfovre’s GA rollout has been tumultuous thanks to side effects concerns, Medicare hiccups and more. All told, Syfovre generated $154.6 million in 2024’s second quarter. As of August, the medicine remained the U.S. market leader in GA, Apellis said in its latest earnings release.
As for the CHMP’s rationale behind its rejection, the agency said that while Syfovre’s phase 3 OAKS and DERBY studies showed that the med slowed the growth of GA lesions, it failed to lead to clinically meaningful benefits for patients. Further, regular injections into the eye carry a significant risk of side effects and safety events, including developing other forms of AMD or inflammation that could worsen vision, the regulator pointed out.
European regulators are also reviewing Astellas Pharma’s Izervay for GA. The Japanese drugmaker’s medicine won U.S. approval in the eye condition last August.
Syfovre was the odd man out Friday as the CHMP announced multiple other approval recommendations—all of which were positive.
The drugs that got the thumbs up were ImmunoGen’s Elahere in ovarian, fallopian tube or primary peritoneal cancer; Henlius’ Hetronifly in extensive-stage small cell lung cancer; Pfizer’s Hympavzi to treat bleeding episodes in teens and adults with hemophilia A or B; Pfizer’s meningococcus vaccine Penbraya; Eckert & Ziegler Radiopharma’s radiopharmaceutical precursor Theralugand; the biosimilars for age-related macular degeneration Afqlir and Opuviz; and Teva’s generic version of pomalidomide for multiple myeloma treatment.
Ipsen also received good news from European regulators Friday as the company has secured conditional approval from the European Commission (EC) for its drug Iqirvo in the rare liver disease primary biliary cholangitis (PBC).
The EC based its decision upon a prior CHMP recommendation leveraging data from Ipsen’s late-stage ELATIVE study. In the trial, 51% of patients on 80 mg of Iqirvo achieved a biochemical response versus just 4% of patients in the control arm, Ipsen said in a release.
The positive update across the pond comes after Iqirvo snagged an FDA nod in the same condition back in June.
The timing may be beneficial for Ipsen, given that Intercept’s PBC rival Ocaliva is now facing an uncertain future in Europe. Several weeks ago, the EC moved to revoke Intercept’s conditional marketing nod in PBC, though the General Court of the European Union cut in and has temporarily suspended the EC’s decision.