Ranibizumab (Genentech)

The EMA's drug advisory panel backed a dozen medicines for approval at its July meeting, including Johnson & Johnson's IL-23 antagonist Icotyde (icotrokinra) for plaque psoriasis, Roche's ocular implant Susvimo (rabinizumab) for wet age-related macular degeneration (wAMD), and several cholesterol-lowering therapies.Among the more closely watched decisions, the Committee for Medicinal Products for Human Use (CHMP) recommended Icotyde for adults and adolescents aged 12 years and older with moderate-to-severe plaque psoriasis. If approved by the European Commission, Icotyde would be the first oral medicine targeting the Il-23 receptor available in the region, and expand a class that is dominated by injectables at the moment.The drug was approved in the US in March, with J&J recently saying its product is off to a strong commercial start. For related analysis, see Physician Views Results: J&J's Icotyde may have an edge on Takeda's TYK2 hopeful and Spotlight On: As Stelara fades, J&J's IL-23 tag team steps up.The CHMP recommendation was supported by four Phase III trials involving roughly 2500 patients. Across three studies, between 50% and 57% of patients achieved at least a 90% reduction in Psoriasis Area and Severity Index (PASI 90) scores after 16 weeks, versus between 1% and 4% of placebo recipients. Additionally, 65% to 71% of those given Icotyde achieved clear or almost clear skin, compared with 8% to 11% for placebo.Twice-yearly refillsMeanwhile, the Susvimo recommendation covers its use in adults with wAMD whose disease has stabilised following intravitreal anti-VEGF therapy. The treatment combines ranibizumab, which blocks VEGF, with a surgically implanted refillable ocular device designed to continuously release the drug.The implant is refilled every six months and would reduce the burden of monthly intravitreal injections that have been the standard for ranibizumab-treated patients since the drug was first approved in Europe nearly 20 years ago. If ultimately cleared by the European Commission, Susvimo would become the first implant of its kind approved in the EU.The recommendation was based on a Phase III study of 415 patients showing that Susvimo maintained visual and anatomical outcomes comparable to monthly ranibizumab injections. Common side effects included iritis, conjunctival bleb leak, cataract, vitreous floaters, conjunctival haemorrhage, conjunctival hyperaemia, eye pain and headache.Originally approved in the US for wAMD in 2021, Susvimo has since had its label expanded there to include diabetic macular oedema and diabetic retinopathy.Trio of anti-cholesterol drugsCHMP also backed approval of several cholesterol treatments for adults, including NewAmsterdam Pharma and Menarini's Ubeslo, a once-daily oral non-statin therapy containing low-dose CETP inhibitor obicetrapib, as well as Evlarco, a fixed-dose combination containing obicetrapib plus ezetimibe.Those positive opinions recommend the drugs for patients with primary hypercholesterolemia, both heterozygous familial and non-familial or mixed dyslipidemia based on Phase III data from the BROADWAY, BROOKLYN and TANDEM trials, which demonstrated significant LDL-C reductions reaching up to 40% with Ubeslo monotherapy and about 50% with Evlarco, when compared to placebo (see – KOL Views Q&A: NewAmsterdam's obicetrapib will elbow into LDL space pending two unknowns, says leading cardiologist).CHMP also adopted a positive opinion for LIB Therapeutics' Lyrokaul (lerodalcibep), an injection that patients can self-administer monthly. The third-generation PCSK9 inhibitor was approved by the FDA in December as Lerochol, based on data from the Phase III LIBerate programme showing sustained LDL-C reductions of 60% or more in patients with or at high risk of cardiovascular disease, and 50% or more in those with heterozygous familial hypercholesterolemia who have more severe LDL-C elevations.A first for cALDAmong medicines for rare diseases, CHMP recommended approval under exceptional circumstances for Minoryx Therapeutics and Neuraxpharm's Nezglyal (leriglitazone) to treat cerebral adrenoleukodystrophy (cALD) in patients aged two to 12 years with gadolinium-negative brain lesions.The opinion was based on the Phase II/III NEXUS study, where Nezglyal achieved the primary endpoint of clinical and radiological arrested disease at week 96 or at a visit prior to haematopoietic stem cell transplant, as well as additional real-world evidence from compassionate-use programmes.If regulators give the green light, expected in September, Nezglyal would become the first pharmacological treatment for cALD in the EU.Rare disease snubThe committee also issued negative opinions for three applications, including for Zevra Therapeutics' Meplyffa (arimoclomol citrate), which the company had been seeking approval for to treat Niemann-Pick disease type C in patients aged two years and older in combination with miglustat. "Due to the way the data had been handled and the results analysed, there were uncertainties about the reliability and robustness of the results. Moreover, no benefit was seen in terms of ambulation and cognition," the EMA said.The agency noted that data for a subgroup of patients who were also taking miglustat showed a treatment effect favouring Meplyffa, though this "was not considered robust since effectiveness had not been demonstrated in the overall population."Zevra, whose shares tumbled nearly 24% on Friday, said it is going to request a re-examination of the CHMP opinion.The oral heat shock protein amplifier was approved by the FDA in 2024 under the brand Miplyffa, after a bumpy regulatory journey. That decision was based on data from a 50-patient, placebo-controlled Phase II/III study where patients who received thrice-daily Miplyffa had slower disease progression than those on placebo, as measured by the 4-domain NPC Clinical Severity Scale (R4DNPCCSS).

Outlook Therapeutics' persistence has paid off. The drugmaker won an FDA approval Friday for its ophthalmic formulation of Roche's anti-VEGF mAb Avastin (bevacizumab) after a series of complete response letters (CRLs) that culminated in a formal dispute earlier this year. The US regulator greenlit Lytenava (bevacizumab-vikg), given as an intravitreal injection, to treat wet age-related macular degeneration (AMD), making it the first FDA-approved ophthalmic formulation of bevacizumab for the indication. The therapy is already approved in the EU and UK for wet AMD."For more than two decades, bevacizumab has served as one of the most widely utilised anti-VEGF therapies in retina care despite the absence of an FDA-approved ophthalmic formulation," said CEO Bob Jahr. "Rather than relying on repackaged formulations originally intended for intravenous use, physicians now have access to a purpose-built ophthalmic formulation of bevacizumab designed specifically for intravitreal administration that delivers the manufacturing consistency, product quality, and regulatory oversight patients deserve."The drugmaker expects Lytenava to be available to US patients by year-end.Regulatory journey Friday's approval caps off a four-year regulator saga for Outlook. The company first filed for approval of Lytenava in 2022, but withdrew its application after the FDA requested further information. It then resubmitted the BLA, only to receive its first CRL in 2023 that called for additional confirmatory clinical evidenceLytenava went on to rack up two more CRLs, as the US regulator remained unconvinced by the therapy's data package. The Phase III NORSE EIGHT study, which served as the backbone for several of Outlook's submissions, came up short against Roche's Lucentis (ranibizumab), with Lytenava demonstrating a mean +4.2 letter improvement in best-corrected visual acuity after eight weeks, compared to +6.3 letters for Lucentis in the intent-to-treat primary dataset.In April, Outlook moved to contest the third CRL by submitting a formal dispute resolution request to the FDA. The drugmaker argued that the clinical, functional, safety and pharmacodynamic data it generated for Lytenava support its approval for wet AMD — and in May, the FDA said it agreed, allowing Outlook to once again submit a BLA for the ophthalmic formulation. The US regulator accepted the resubmitted application mid-June. When reviewing Outlook's appeal, the FDA's Office of New Drugs said findings from the NORSE TWO and NORSE EIGHT trials, alongside natural history and mechanistic data, support Lytenava's efficacy to treat wet AMD.