Monday, July 15, 2024

Geographic Atrophy Treatments Face Setbacks as EU Drug Regulator Rejects Apellis’ Syfovre Again

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For the second time in six months, the European Medicines Agency’s (EMA) European Committee for Medicinal Products for Human Use (CHMP) has rejected Apellis’ Syfovre for the treatment of geographic atrophy (GA) caused by age-related macular degeneration. Despite multiple dissenting votes, the CHMP maintained that while Syfovre slowed the growth of geographic atrophy lesions, it did not lead to clinically meaningful benefits for patients. Additionally, safety concerns were raised regarding the regular injections into the eye, which 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.

In January of this year, the CHMP issued its first negative opinion on Syfovre. Despite its approval by the U.S. Food and Drug Administration (FDA) in February 2023 and generating $275 million in sales last year, Apellis has faced challenges with its launch, admitting to these difficulties while holding an 85% market share in geographic atrophy. In Europe, Apellis competes with Astellas’ Izervay, which has filed for approval. Following the CHMP’s recent decision, Apellis plans to seek a reexamination, with a final decision expected in the fourth quarter of this year.

Mizuho Securities analyst Graig Suvannavejh noted a 5% drop in Apellis’ share price on the news, lowering the probability of Syfovre’s EU approval from 20% to 10%. He pointed out that there have yet to be data convincingly demonstrating Syfovre’s use leading to clear, clinically meaningful functional benefits. Furthermore, Suvannavejh remarked that from a drug approval perspective, the European Medicines Agency is typically more conservative than the FDA.

Geographic Atrophy Drug Syfovre and Others Face Rejection by CHMP Amid Mixed Regulatory Decisions

Syfovre was not the only drug to face rejection by the CHMP multiple times. The committee has also recommended not renewing the marketing authorization for PTC Therapeutics’ Translarna as a treatment for Duchenne muscular dystrophy. This follows the European Commission’s May decision to not adopt the CHMP’s negative opinion from January, with the CHMP having reviewed and declined to renew the drug’s conditional approval twice already.

Despite evidence of significant efficacy provided by the STRIDE trial, the agency maintained its stance. PTC plans to request a reexamination of this third opinion, expecting the European Commission to make a final decision thereafter. Based on the timeline of these procedures, PTC expects Translarna to remain on the market through the end of 2024, even if the negative opinion holds.

The CHMP also refused to recommend marketing authorization for AB Science’s masitinib, a treatment for the nervous system disorder amyotrophic lateral sclerosis (ALS). The France-based company announced it would request a reexamination. Additionally, the CHMP recommended revoking the marketing authorization for Advanz’s Ocaliva for the treatment of patients with primary biliary cholangitis.

Despite these rejections, the CHMP issued positive recommendations for several therapies. Among them, Merck’s Winrevair for pulmonary arterial hypertension (PAH) received a positive opinion. Winrevair, which gained FDA approval in March, is the first disease-modifying treatment for the condition and is expected to achieve peak sales between $3 billion and $4 billion, according to J.P. Morgan analyst Chris Schott. The CHMP also recommended ARS Pharmaceuticals’ marketing application for the adrenaline nasal spray Eurneffy, set to become the first needle-free adrenaline option in Europe for emergency Type 1 allergic reactions. ARS expects EU authorization in the third quarter and to launch Eurneffy in the fourth quarter, with an FDA decision anticipated by October 2 of this year.

Geographic Atrophy

CHMP Recommends Positive Approvals for Johnson & Johnson’s Balversa, Moderna’s RSV Vaccine

Johnson & Johnson’s Balversa also received a positive recommendation from the CHMP for treating patients with unresectable or metastatic urothelial carcinoma, a type of bladder cancer. The FDA approved Balversa for this indication earlier this year. Moderna’s respiratory syncytial virus (RSV) vaccine mRasvia gained a recommendation for adults aged 60 and older. The vaccine, 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.

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Regeneron’s CD20xCD3 bispecific T-cell engager Ordspono (odronestamab) was also recommended by the CHMP for treating two types of blood cancers: follicular lymphoma and diffuse large B-cell lymphoma. This 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. Roche’s Piasky (crovalimab) received a recommendation for marketing authorization just days after the FDA endorsed it for patients aged 13 and older with paroxysmal nocturnal hemoglobinuria (PNH), a rare genetic disorder.

Additionally, Eli Lilly’s Tauvid (flortaucipir) was recommended by the CHMP as the first drug used to image a characteristic of Alzheimer’s disease in the brain called tau pathology, following FDA approval. Celltrion’s Steqeyma, a biosimilar of Johnson & Johnson’s Stelara, was also recommended for patients with Crohn’s disease, plaque psoriasis, and psoriatic arthritis. The CHMP’s decisions this week reflect a mix of rejections and approvals, highlighting the rigorous scrutiny and high standards maintained by the European drug regulatory framework.


Resource: European Medicines Agency, June 28, 2024

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