EMA Marketing Authorization of New Drugs in September 2026 

Shots:  The European Commission (EC) approved multiple medicines in September 2026 across cardiovascular disease, immunology, ophthalmology, rare diseases, and oncology, with approvals involving companies including NewAmsterdam, Johnson & Johnson, Roche, and Neuraxpharm.   The European Commission (EC) approved Ubeslo and Evlarco for primary hypercholesterolemia, Icotyde for plaque psoriasis, Susvimo for neovascular age-related macular degeneration, and Nezglyal for cerebral adrenoleukodystrophy in September 2026.  The CHMP issued positive opinions for several medicines, including Frehemgo for hemophilia A, Klygefa for generalized myasthenia gravis, Lifyorli for platinum-resistant ovarian cancer, Sepalna for ovarian cancer maintenance, and Zeydovio for short bowel syndrome.  NewAmsterdam and Menarini Report the EC Approval for Ubeslo and Evlarco for Primary Hypercholesterolemia                  Company: NewAmsterdam and Menarini                  Product: Ubeslo and Evlarco                 Active Ingredient: Obicetrapib and Obicetrapib + Ezetimibe FDC                 Disease: Primary Hypercholesterolemia                 Date: Sep 21, 2026                   Shots:   The EC has approved Ubeslo (obicetrapib 10mg) & Evlarco (10mg obicetrapib + 10mg ezetimibe FDC) for pts with primary hypercholesterolemia, both HeFH & non-familial or mixed dyslipidemia; approval will be valid across 30 EEA states   Approval was supported by extensive clinical data assessing obicetrapib, incl. the P-III (BROADWAY, BROOKLYN & TANDEM) trials, which showed up to 40% LDL-C reductions with Ubeslo vs PBO & ~50% with Evlarco vs PBO  NewAmsterdam & Menarini continue are advancing obicetrapib through P-III trials, incl. the cardiovascular outcomes PREVAIL study, as well as REMBRANDT, & RUBENS trial  Editor’s Take: Beyond the Headlines   The EC approvals of Ubeslo and Evlarco give NewAmsterdam and Menarini a broader commercial platform for LDL-C reduction across European markets. Evlarco’s fixed-dose combination offers a simplified option with greater reported LDL-C lowering, while the ongoing PREVAIL outcomes trial will be critical to establishing obicetrapib’s role in reducing cardiovascular risk beyond cholesterol management.   Johnson & Johnson Reports the EC Approval of Icotyde (Icotrokinra) to Treat Plaque Psoriasis (PsO)                  Company: Johnson & Johnson                 Product: Icotyde                 Active Ingredient: Icotrokinra                 Disease: Plaque Psoriasis                  Date: Sep 21, 2026                   Shots:   The EC has approved Icotyde for the treatment of pts (≥12yrs., ≥40kg) with mod. to sev. plaque PsO who are candidates for systemic therapy  Approval was backed by P-III (ICONIC-TOTAL, ICONIC LEAD, ICONIC-ADVANCE 1 & 2) trials, where Icotyde met all 1EP across all 4 trials (n=2,500). In the head-to-head studies, ~70% pts achieved IGA 0/1 & 55% reached PASI 90 at Wk. 16  Icotyde (JNJ-2113) is also being assessed in ICONIC-PsA 1 & 2 (vs PBO) for active psoriatic arthritis, ICONIC-UC for mod. to sev. active ulcerative colitis & ICONIC-CD for mod. to sev. active Crohn’s disease    Editor’s Take: Beyond the Headlines  The EC approval of Icotyde strengthens Johnson & Johnson’s immunology portfolio with an oral IL-23 receptor-targeting therapy for moderate-to-severe plaque psoriasis, offering an alternative to injectable biologics. Phase III efficacy results may support competition on skin clearance and convenience, while ongoing trials in psoriatic arthritis and inflammatory bowel disease could expand its future market opportunity.   Roche Reports EC Approval of Susvimo to Treat  nAMD                  Company: Roche                 Product: Susvimo                 Active Ingredient: Ranibizumab                 Disease: Neovascular Age-Related Macular Degeneration                  Date: Sep 21, 2026                   Shots:   The EC has approved Susvimo (ranibizumab 100mg/mL solution for injection) for the treatment of neovascular age-related macular degeneration (nAMD), based on the 3 studies, P-III (Archway), P-II (LADDER) & open-label LTE study (Portal)  Archway showed Susvimo (refilled in Q6M) maintained vision improvements equivalent to ranibizumab (QM, IVT), with Portal showing maintenance of vision up to 7yrs. Also, ~95% pts treated with Susvimo required no supplemental or additional anti-VEGF treatment   In 2025, Roche received a CE Mark for its Port Delivery Platform, known as Contivue, which incl. the eye implant for Susvimo & 4 ancillary devices to initially fill, insert, refill, & remove the implant, while Roche is evaluating additional molecules for the platform   Editor’s Take: Beyond the Headlines  The EC approval of Susvimo strengthens Roche’s position in nAMD by offering sustained ranibizumab delivery with six-month refill intervals, potentially reducing treatment burden compared with monthly injections. Long-term vision maintenance and low supplemental-treatment needs could support adoption, while the Contivue platform creates opportunities to extend Roche’s sustained-delivery technology to additional therapies.  Neuraxpharm and Minoryx Report the EC Approval of Nezglyal for Cerebral Adrenoleukodystrophy (cALD)                  Company: Neuraxpharm and Minoryx                  Product: Nezglyal                 Active Ingredient: Leriglitazone                 Disease: Cerebral Adrenoleukodystrophy                 Date: Sep 25, 2026                   Shots:   EC has approved Nezglyal (leriglitazone) under exceptional circumstances for male cALD pts (2-12yrs.) with Gadolinium (Gd)-negative brain lesions, based on P-II/III (NEXUS) trial data & RWE data from compassionate use programs   The first EU launch is expected in Germany by year-end, with additional launches anticipated following national reimbursement negotiations, while Neuraxpharm evaluates access pathways for eligible pts  Nezglyal is being further assessed in the P-III (CALYX) trial in adult male cALD pts with Gd-enhancing lesions, & the ongoing P-IIa (TREE) trial in paediatric pts with Rett syndrome; read-outs are expected in early 2028 & by the end of 2026, respectively  Editor’s Take: Beyond the Headlines  Nezglyal’s EC approval establishes an initial European foothold for Neuraxpharm and Minoryx in pediatric cALD, addressing an underserved patient population with limited treatment options. Germany’s planned launch could set the pace for broader uptake, while national reimbursement negotiations will shape access. Adult cALD and Rett syndrome trials may expand its commercial potential. The CHMP Recommends Novo’s Frehemgo (Denecimig) for Haemophilia A                  Company: Novo                 Product: Frehemgo                 Active Ingredient: Denecimig                 Disease: Haemophilia A                 Date: Sep 17, 2026                   Shots:  The CHMP has recommended Frehemgo (QM, Q2W or QW) to treat haemophilia A, with or without inhibitors, in adults & children, based on the FRONTIER program; launch is expected in the first EU countries in Q4’26 & broadly across the EU starting early 2027  FRONTIER 2 trial showed Frehemgo significantly reduced annualized bleeding rates (ABR) vs prior factor prophylaxis & on-demand treatment, with or without inhibitors, while FRONTIER 3 showed consistent efficacy in children (<12yrs.)  Across the program, mean ABRs were generally <1 in P-III populations, with many pts experiencing zero treated bleeds, while FRONTIER 5 identified no new safety signals after switching from emicizumab & showed a clear preference for the Frehemgo device  Editor’s Take: Beyond the Headlines  Nezglyal’s EC approval gives Neuraxpharm and Minoryx a foothold in cALD, targeting children with Gd-negative brain lesions, a population with limited treatment options. The exceptional-circumstances authorization and planned German launch create an early access opportunity, while reimbursement negotiations will shape uptake. Adult cALD and Rett syndrome trials could broaden its future market.  AstraZeneca Reports the CHMP Positive Opinion for Klygefa to Treat Generalised Myasthenia Gravis                  Company: AstraZeneca                 Product: Klygefa                 Active Ingredient: Gefurulimab                 Disease: AChR Antibody-Positive Generalised Myasthenia Gravis                  Date: Sep 17, 2026                   Shots:  The CHMP has recommended Klygefa (gefurulimab; SC, QW) as an add-on to standard therapy for the treatment of generalised myasthenia gravis (gMG) in adults who are AChR antibody-positive; filings are under review in the US, China & other countries  Opinion was supported by the P-III (PREVAIL) trial assessing Klygefa vs PBO in 260 gMG adults for 26wks. across North America, EU, & APAC region; eligible pts could then enter an ongoing OLE study   Trial met its 1EP, showing improvement in MG-ADL total score at week 26, with benefit seen as early as Wk. 1 & was sustained through Wk. 26; data were published in JAMA Neurology   Editor’s Take: Beyond the Headlines  Frehemgo’s positive CHMP recommendation positions Novo to compete in haemophilia A with a subcutaneous prophylactic option offering weekly to every-four-week dosing, including patients with inhibitors. Low bleeding rates and patient preference for its device could support uptake against emicizumab, while real-world outcomes, safety and pricing will shape market adoption.  Corcept Therapeutics Reports the CHMP Positive Opinion of Lifyorli (Relacorilant) to Treat Platinum-Resistant Ovarian Cancer                  Company: Corcept Therapeutics                  Product: Lifyorli                 Active Ingredient: Relacorilant                 Disease: Platinum-Resistant Ovarian Cancer                 Date: Sep 17, 2026                   Shots:  The CHMP has recommended Lifyorli (relacorilant) + nab-paclitaxel for the treatment of patients with platinum-resistant ovarian cancer; EC decision is expected in Q4’26  Approval was backed by the P-II trial & the P-III (ROSELLA) study assessing Lifyorli + nab-paclitaxel vs nab-paclitaxel alone in above mentioned pts (n=381) across the US, EU, South Korea, Brazil, Argentina, Canada & Australia  The P-III trial showed improved PFS & OS in pts treated with Lifyorli + nab-paclitaxel, along with a favorable safety profile  Editor’s Take: Beyond the Headlines  Lifyorli’s positive CHMP opinion could strengthen Corcept’s position in platinum-resistant ovarian cancer, where treatment options remain limited after platinum-based therapy fails. Improved progression-free and overall survival with nab-paclitaxel may differentiate the combination from chemotherapy alone, while the upcoming EC decision, safety monitoring and commercial uptake will determine its competitive impact.   CHMP Recommends IMPACT and Pharmanovia’ Sepalna  as Maintenance Therapy for 1L Advanced Epithelial Ovarian, Fallopian Tube, and Primary Peritoneal Cancer                  Company: IMPACT Therapeutics and Pharmanovia                 Product: Sepalna                  Active Ingredient: Senaparib                 Disease: Advanced Epithelial Ovarian, Fallopian Tube, and Primary Peritoneal Cancer                  Date: Sep 17, 2026                   Shots:  The CHMP has recommended Sepalna (senaparib) as the maintenance treatment of advanced epithelial ovarian, fallopian tube and primary peritoneal cancer; if approved, authorization would be valid in all 30 EEA states    Opinion was based on the P-III (FLAMES) trial assessing Sepalna vs PBO in pts with advanced (FIGO Stage III-IV) epithelial high-grade Ovarian Cancer (OC), fallopian tube, and primary peritoneal cancer who are in response (complete or partial) following 1L Pt-based CT  Senaparib reduced the risk of progression or death by 57% vs PBO in advanced ovarian cancer, regardless of BRCA mutation status, with mPFS  not reached vs 13.6mos.; data were published in Nature Medicine  Editor’s Take: Beyond the Headlines  Sepalna’s positive CHMP opinion could expand the PARP inhibitor market for first-line ovarian cancer maintenance by offering a potential option irrespective of BRCA mutation status. The 57% reduction in progression or death risk supports its competitive potential, but final EC approval, long-term safety, and differentiation from established PARP inhibitors such as AstraZeneca’s Lynparza will shape adoption.  Zealand Pharma Reports CHMP Positive Opinion for Zeydovio  (Glepaglutide) for Short Bowel Syndrome (SBS)                  Company: Zealand Pharma                 Product: Zeydovio                  Active Ingredient: Glepaglutide                 Disease: Short Bowel Syndrome                  Date: Sep 17, 2026                   Shots:  The CHMP has recommended Zeydovio (glepaglutide) for the treatment of adults with SBS. Zealand is pursuing partnerships for the future global commercialization of Zeydovio for SBS  Opinion was based on the P-III (EASE-1) trial of Zeydovio (10mg, QW or Q2W) vs PBO in 106 SBS pts with intestinal failure (IF), dependent on parenteral support (PS) for ≥3 days/wk., & was further backed by interim data from EASE-2 & EASE-3 trials, LTE trials of ~2yrs. & mechanistic EASE-4 trial   Zeydovio (Q2W) reduced PS requirements vs PBO in SBS pts with IF, with two-thirds achieving ≥20% reduction in weekly PS volume & 1 in 7 weaning off support entirely after 24wks.; EASE-2 & 3 long-term efficacy & safety data are expected in 2027  Editor’s Take: Beyond the Headlines  Zeydovio’s positive CHMP opinion strengthens Zealand Pharma’s position in short bowel syndrome by offering a potential alternative to Takeda’s Gattex/Revestive, with weekly or every-two-week dosing and reduced parenteral support needs. Long-term efficacy, complete weaning rates, reimbursement and a global commercialization partner will be key to its competitive uptake.  Notes:-    The following drugs have received CHMP positive opinion; however, no PR was available:   Povofortay (Povorcitinib)  VaxRabeo [Rabies vaccine (inactivated) for human use]  Gevalka (Ensartinib)  The post EMA Marketing Authorization of New Drugs in September 2026  appeared first on PharmaShots.