CHMP Rejects Neuraxpharm and Apellis Drugs, Companies to Appeal

The European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has declined marketing approvals for two drugs at its January meeting. Apellis' drug, Syfovre (pegcetacoplan), intended for treating geographic atrophy (GA) associated with age-related macular degeneration (AMD), was rejected. The CHMP stated that the treatment "did not lead to clinically meaningful benefits for patients." Despite its approval in the US last year for GA, the intravitreal injection formulation faced setbacks in the EU.

Similarly, Neuraxpharm and its partner Minoryx Therapeutics were denied conditional approval for Nezglyal (leriglitazone), aimed at treating cerebral adrenoleukodystrophy (cALD). The CHMP highlighted concerns over the drug's efficacy, noting it "was unable to achieve an improvement compared to placebo on the six-minute walk test (6MWT)," and raised issues regarding tolerability, specifically weight gain and swelling (oedema).

Both companies have expressed their intentions to appeal the CHMP's decisions. Apellis anticipates a re-examination after the panel's initial indication to reject the application in December. Neuraxpharm and Minoryx Therapeutics are also seeking a re-examination, firmly believing in the "positive benefit/risk balance" of leriglitazone for cALD patients.

In contrast, the CHMP issued positive opinions for other drug applications, including a generic version of Johnson & Johnson's schizophrenia drug Xeplion (paliperidone) by Neuraxpharm, under the brand name Niapelf, and Advanz Pharma's antibiotic Exblifep for treating urinary tract infections and pneumonia in adults.