EU Drug Regulator Rejects Apellis' Eye Med and PTC's Translarna Again

Drug reviewers at the European Medicines Agency have issued a positive review for Merck's Winrevair, a treatment for pulmonary arterial hypertension (PAH), while once again rejecting Apellis' geographic atrophy (GA) treatment, Syfovre.

The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has, for the second time in six months, denied approval for Apellis’ Syfovre for GA caused by age-related macular degeneration. Apellis mentioned in a release that several CHMP members dissented from the negative opinion. The company plans to seek a reexamination with a final decision anticipated in the fourth quarter of the year.

The CHMP's negative stance was backed by findings that, although Syfovre slowed the growth of GA lesions, it did not offer significant clinical benefits in patients' daily lives. Additionally, concerns were raised about the safety of regular eye injections, which carry risks of adverse events like other forms of AMD or eye inflammation that could worsen vision.

Previously, in January, the CHMP had also issued a negative opinion on Syfovre. Despite its FDA approval in February 2023 and generating $275 million in sales last year, Apellis faced early reports of inflammation from some users. In May, Apellis reported an 85% market share in GA but acknowledged challenges with its launch. It faces competition in Europe from Astellas' Izervay, which has applied for approval.

Mizuho Securities analyst Graig Suvannavejh indicated a 5% drop in Apellis' share price following the news. He noted the absence of clear, clinically meaningful benefits from Syfovre and viewed the European Medicines Agency as more conservative compared to the FDA. Suvannavejh adjusted the probability of Syfovre's EU approval from 20% to 10%.

Syfovre wasn't the only drug facing rejection. The CHMP also denied renewing PTC Therapeutics' Translarna marketing authorization for Duchenne muscular dystrophy. This follows the European Commission's decision in May to not adopt the CHMP’s negative opinion from January. PTC plans to request a reexamination of this third opinion, with the European Commission making the final decision. Even if the negative opinion holds, PTC expects Translarna to remain available through the end of 2024.

Additionally, the CHMP refused marketing authorization for AB Science’s masitinib for amyotrophic lateral sclerosis (ALS). The company plans to request a reexamination. Advanz's Ocaliva also had its marketing authorization revoked for treating primary biliary cholangitis.

On a positive note, the CHMP recommended several therapies. Merck’s Winrevair received a thumbs up for PAH. Winrevair, acquired through Merck's $11.5 billion purchase of Acceleron in 2021, is expected to achieve peak sales between $3 billion and $4 billion, according to J.P Morgan analyst Chris Schott.

Additionally, ARS Pharmaceuticals' adrenaline nasal spray Eurneffy was recommended for approval. If authorized, it would be the first needle-free adrenaline treatment in Europe for emergency Type 1 allergic reactions. ARS expects EU authorization in the third quarter and plans to launch Eurneffy in the fourth quarter, with an FDA decision expected on October 2.

The CHMP also recommended Johnson & Johnson’s Balversa for unresectable or metastatic urothelial carcinoma, Moderna’s mRasvia vaccine for respiratory syncytial virus (RSV) for adults 60 and older, and Regeneron’s Ordspono for treating two types of blood cancers. Roche’s Piasky received a positive opinion for treating paroxysmal nocturnal hemoglobinuria (PNH), and Eli Lilly’s Tauvid was recommended for imaging tau pathology in Alzheimer’s disease. Celltrion’s Steqeyma, a biosimilar of J&J’s Stelara, was endorsed for Crohn’s disease, plaque psoriasis, and psoriatic arthritis.