CHMP turns down Neuraxpharm, Apellis drugs
The EMA’s human medicines committee refused marketing approvals for two drugs from Neuraxpharm and Apellis at its January meeting, but both companies have said they will appeal the decision.
Apellis’ application to market its complement C3 inhibitor Syfovre (pegcetacoplan) to treat geographic atrophy (GA) – a sight-robbing condition that accompanies age-related macular degeneration (AMD) and is a leading cause of blindness – was rejected on the grounds that treatment “did not lead to clinically meaningful benefits for patients.”
Syfovre was approved for GA in the US last year and is an intravitreal injection formulation of the drug, which is also approved in a subcutaneous form as Empaveli/Aspaveli for rare blood disorder paroxysmal nocturnal haemoglobinuria (PNH).
Neuraxpharm and partner Minoryx Therapeutics, meanwhile, were unable to convince the CHMP that they should get conditional approval for PPAR gamma agonist Nezglyal (leriglitazone) as a treatment for cerebral adrenoleukodystrophy (cALD), an inherited disease that leads to the destruction of nerve cells.
The panel said that the drug was unable to achieve an improvement compared to placebo on the six-minute walk test (6MWT). It also had concerns about the tolerability of the drug with regard to weight gain and swelling (oedema) and said the manufacturer had not put forward any plans to mitigate the risks.
Leriglitazone was developed by Minoryx, which filed the marketing application, and Neuraxpharm licensed exclusive EU rights to the drug in a deal signed in 2022. In a statement, the two companies said they are seeking a re-examination of the application, saying they “strongly believe that leriglitazone has a positive benefit/risk balance in patients with cALD.”
Apellis said it was expecting the CHMP’s decision after the panel indicated it was minded to reject the application in December and added that it would seek “immediate re-examination” of the decision.
Other drugs backed for EU approval
There was better news for other drug applications at the meeting, including consolation recommendations for a Neuraxpharm generic.
The CHMP issued a positive opinion for Neuraxpharm’s copycat version of Johnson & Johnson’s schizophrenia drug Xeplion (paliperidone), under the Niapelf brand name, as well as for Advanz Pharma’s antibiotic product Exblifep (cefepime dihydrochloride monohydrate/enmetazobactam) for urinary tract infections and pneumonia in adults and Evive Biotechnology’s Ryzneuta (efbemalenograstim alfa) for neutropenia associated with chemotherapy.
Among other decisions, the CHMP also gave a green light to the use of Pfizer and Astellas’ Nectin-4 targeting antibody-drug conjugate (ADC) Padcev (enfortumab vedotin) as a combination therapy with MSD’s checkpoint inhibitor Keytruda (pembrolizumab) as a first-line treatment for locally advanced or metastatic urothelial cancer based on the results of the EV-302 trial.
