Neuraxpharm, Apellis asking for fresh drug reviews after EU rejections
26 Jan 2024
Phase 3Drug ApprovalClinical ResultNDA
Neuraxpharm and Apellis each received negative opinions from an advisory panel to the European Medicines Agency (EMA) on Friday, prompting both drugmakers to ask for a re-examination of their respective marketing authorisation applications (MAAs).
Shares of Apellis, which was notified in December that its application for intravitreal pegcetacoplan to treat geographic atrophy (GA) secondary to age-related macular degeneration (AMD)AMD) was likely to be rejected, slipped about 3% on the news.
Neuraxpharm and partner Minoryx Therapeutics are seeking conditional approval of leriglitazone to treat cerebral adrenoleukodystrophy (cALD). The former in-licensed exclusive European rights to the PPAR gamma agonistPPAR gamma agonist from the latter in 2022.
Missing walk data
The EMA’s Committee for Medicinal Products for Human Use (CHMP) said it recommended against leriglitazone because of a lack of both efficacy data and a side effect mitigation strategy.
In its decision, CHMP cited data from the Phase II/III ADVANCE1 trial, which did not show a difference between leriglitazone and placebo in terms of the distance patients could walk in six minutes. The panel also had concerns about weight gain and oedema, but did not identify any measures in the MAA to manage or reduce these risks.
“We are obviously disappointed with the committee’s decision, but we strongly believe that leriglitazone provides clinically meaningful benefits for patients by reducing the development of progressive cALD and stabilising lesion progression,” Minoryx CEO Marc Martinell said in a statement.
According to the partners, data from the ADVANCE1 trial showed that leriglitazone reduces the progression of lesions and the development of progressive cALD.
Intravitreal injection risk
Apellis’ pegcetacoplan was approved by the FDA last year as Syfovre, but its uptake has been limited due to concerns over an increased risk for retinal vasculitis – a risk that was called out by CHMP in its negative opinion.
“Regular injections into the eye carry a significant risk of adverse events, including the development of other forms of AMD or inflammation in the eye, that could further worsen vision,” the committee noted.
While CHMP acknowledged that the targeted C3 therapy slowed growth of GA lesions, the drug did not lead to a clinically meaningful benefit for patients. “Therefore, the agency’s opinion was that the benefits of Syfovre did not outweigh its risks and it recommended refusing marketing authorisation,” CHMP concluded.
The EU filing was based on 24-month data from two Phase III studies used to back the US approval. Compared to sham treatment, monthly and every-other-month dosing with pegcetacoplan slowed GA lesion growth.
However, a third of doctors in a FirstWord poll last year said they had stopped prescribing the drug as they awaited more clarity on its safety profile. For more, see, KOL Views Q&A: Safety issues won’t necessarily sink Apellis’ Syfovre but door is ajar for Astellas' Izervay in GA.
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