EU's drug regulator again rejects Apellis' eye med and PTC's Translarna in busy week of up-and-down decisions

28 Jun 2024
www.fiercepharma.com
Drug ApprovalVaccineAccelerated Approval
Drug reviewers at the European Medicines Agency have given a thumbs up for Merck's PAH drug Winrevair and a second thumbs down to Apellis' geographic atrophy treatment Syfovre.
For the second time inEuropean Medicines Agencymittee for Medicinal ProductMerck HuPAH Use (CHMP) has given a thumbs down to ApellApellisovgeographic atrophynt of patieSyfovreh geographic atrophy (GA) caused by age-related macular degeneration.
In a release, Apellis noted that there were “multiple dissenting votes” by CHMP members to the opinion. The company added that it planSyfovreek a reexamination of the opinion, wgeographic atrophy (GA)pected in tage-related macular degeneration
“Although the studies showed that Syfovre slowed the growth of geographic atrophy lesions, this did not lead to clinically meaningful benefits for patients,” the CHMP said. “It was noted that benefits of a treatment should impact patients’ everyday functioning, and this was not demonstrated in the studies.”
“In terms of safety,” the CHMP conSyfovre “regular injections igeographic atrophy lesionsicant risk of adverse events, including the development of other forms of AMD or inflammation in the eye, that could further worsen vision.”
In January of this year, the CHMP adopted its first negative opinion on Syfovre. The drug was approved by the FDA in February of 2023 and generated $275 million inAMDles inflammationspite early reports of inflammation for some users.
In May, the company said that it holds an 85% share of the market in GA,Syfovremitted to challenges with its lFDAch. In the indication, Apellis is competing with Astellas’ Izervay, which has filed for apprinflammationpe.
Mizuho Securities analyst Graig Suvannavejh noted that there was a 5% drop in Apellis' share price on Friday morning.ApellisAstellas
Mizuho Securitieso be data convincingly demonstrating Syfovre use leading to cApellislinically meaningful functional benefit," Suvannavejh wrote in a note to clients. "Moreover, from a drug approval perspective, we view the European Medicines Agency as typically more conservative than the FDA. As such, we lower our probability of success for Syfovre's EU approval from 20% to 10%."
Syfovre was not the only drug that was beaten back by the CHMP more than once. The European drug reviewers have once again recommended not renewing the marketing authorization for PTC Therapeutics’ Translarna as a treatment for Duchenne muscular dystrophy.
Syfovregative opinion follows the European Commission’s May decision to not adopt the CHMP’s negative opinion from January. By that time, The CHMP had already reviewed and declinedPTC TherapeuticsPTC renew the drugTranslarnaonal nod twice.Duchenne muscular dystrophy
According to PTC, the scientific aEuropean Commissionned for the latest review concluded that the evidence of significant efficacy provided by the STRIDE trial "should not be ignored." And yet, the agency still maintained its previous stance.
Now, PTC planPTCo request a reexamination of the CHMP’s third opinion. After that, the European Commission will make a decision. Based on the timeline of these procedures, PTC said it expects Translarna to remain on the market through the end of 2024 even if the negative opinion holds.
Elsewhere, the CHMP also refused to recommend a marketing authorization for AB Science’European Commissiontment for the nervous system disorder amyotrophic lateral sclerosiPTCALS). The France-based company said it will ask for a reexamination.
The CHMP also recommended that the marketing authorization for Advanz's OcalAB Scienceokemasitinib treatment of patientsnervous system disorderhamyotrophic lateral sclerosis (ALS)
Meanwhile, the CHMP this week issued positive recommendations for several therapies, including Merck’s blockbuster-to-be Winrevair for pulmonary arterial hypertension (PAH). Winrevair, which gained a high-profile approval from the FDA in March, is the first disease-modifying treatment for the condition.
Winrevair, which was acquired by Merck in an $11.5 billion buyout of Acceleron in 2021, has beeMerckged by J.P Morgan analyst Chris Schpulmonary arterial hypertension (PAH)biWinrevair $4 billion.FDA
Winrevairalso recommended ARS PhaMerckuticals’ marketing application Accelerondrenaline nasal spray EurneffJ.P Morganproved. It would become the first needle-free adrenaline option in Europe for the treatment of emergency Type 1 allergic reactions (anaphylaxis), which include those to food and insect bites.
ARS expects to receive autARS Pharmaceuticals EU in the third quarter and to launch Eurneffy in the fourth quarter. The company also hopes for approval of the prodadrenalinehe FDA, with a decision date set for Ocemergency Type 1 allergic reactions (anaphylaxis)
The CHMP recommended Johnson & Johnson’s Balversa for the treatment of patients with unresectable or metastatic urothelial carcinoma, a type of bladder cancer. The FDA signed oFDAon Balversa in the indication in January of this year, turning a 2019 accelerated approval into a full nod.
Moderna’s respiratoryJohnson & Johnson(RSBalversane mRasvia has made the grade with tunresectable or metastatic urothelial carcinomaunresectable or metastatic urothelial carcinomats ages 60 abladder cancershot, FDAch was approved by the FDA four weeks ago, is the first mRNA vaccine targeting a pathogen other than the coronavirus to receive a positive opinion from the CHMP.
Modernaon’s CD20xCD3 bispecific T-cell engager Ordspono (odronestamab) netted a positive opinion from the CHMP to treat two types of blood cancers—follicular lymphoma and diffuse large B-celFDAymphoma. The thumbs up comes three months after the FDA sent two complete response letters to Regeneron for the same indications, questioning the timelines of the company’s confirmatory trials.
Regeneronrecommended Roche’s Piasky (crovalimab) for marketing authorization, just four days after the FDA endorsed it for patients ablood cancersefollicular lymphomaocturdiffuse large B-cell lymphomaare, genetic life-threatening disorder of theFDAmune system that causes premature breakRegeneronhe red blood cells.
The CHMP also has issRoche positive ocrovalimabEli Lilly’s Tauvid (flortaucipir), the first drug used tFDAmage a characteristic of Alzheimer’s disease in paroxysmal nocturnal hemoglobinuria (PNH)igned off on Tauvid four weeks ago.
Also making the grade from the CHMP was CelltriElisLillyeymTauvidchflortaucipirilar of J&J’s Stelara. It has been recommended for pAlzheimer’s diseases disease, plaque psotausis and psoriatiFDArthritis.Tauvid
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