Insights+: EMA Marketing Authorization of New Drugs in August 2023

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Insights+: EMA Marketing Authorization of New Drugs in August 2023


  • The EMA approved 5 New Chemical Entity (NCE) and 6 Biologic Drugs in August 2023, leading to treatments for patients and advances in the healthcare industry
  • In August 2023, the major highlight drugs were Aquipta (atogepant) approved for the preventive treatment of migraine in adults & Opdivo (nivolumab) as an adjuvant treatment for completely resected stage IIB or IIC melanoma
  • PharmaShots has compiled a list of a total of 11 new drugs approved by the EMA in August 2023

1. Taiho’s Lonsurf (trifluridine/tipiracil) + Bevacizumab Receives EC’s Approval as 3L Treatment of Refractory Metastatic Colorectal Cancer


Active ingredient: trifluridine and tipiracil                       Approved: Aug 01, 2023

Company: Taiho Pharmaceutical                                      Disease: Colorectal Cancer

  • The EC has approved Lonsurf + bevacizumab for adult patients with mCRC who received 2 prior anti-cancer treatments incl. fluoropyrimidine-, oxaliplatin & irinotecan-based chemotherapies, anti-VEGF agents, and/or anti-EGFR agents. The trial was conducted by Servier & Taiho Oncology
  • The approval was based on the P-III trial (SUNLIGHT) evaluating trifluridine/tipiracil + bevacizumab vs trifluridine/tipiracil alone in a ratio (1:1) in 492 patients. The primary objective was to evaluate trifluridine/tipiracil + bevacizumab in terms of OS & 2EPs were PFS, ORR, DCR & QoL, safety & tolerability
  • The marketing authorization is valid in 27 EU countries, Iceland, Northern Ireland, Liechtenstein & Norway. Lonsurf, an oral nucleoside antitumor agent discovered & developed by Taiho Pharmaceutical

2. AbbVie’s Aquipta (atogepant) Receives EC’s Approval for the Preventive Treatment of Migraine in Adults


Active ingredient: atogepant                                               Approved: Aug 17, 2023

Company: AbbVie                                                                 Disease: Migraine

  • The EC has approved Aquipta (CGRP receptor antagonist) for the prophylaxis of migraine in adults who have ≥4 migraine days per month. The approval was based on the 2 P-III studies (PROGRESS) & (ADVANCE) evaluating Aquipta (60mg, qd) vs PBO in 778 & 910 patients
  • Both studies met their 1EPs i.e., reduction in mean monthly migraine days vs PBO, improvements were observed in all 2EPs with Aquipta (60mg, qd). In the (PROGRESS) & (ADVANCE) study, a reduction in the changes from baseline in MMDs of 6.8 vs 5.1 days & 4.1 vs 2.5 days; 40% vs 27% & 59% vs 29% achieved a 50% reduction in MMDs, respectively
  • The therapy was approved in the US for chronic & episodic migraines and in Canada for episodic migraines under the brand name Qulipta

3. Janssen’s Tecvayli (teclistamab) Receives Type II Variation Application Approval from EC for Relapsed and Refractory Multiple Myeloma


Active ingredient: teclistamab                                               Approved: Aug 18, 2023

Company: Janssen                                                                 Disease: Multiple Myeloma

  • The EC has granted conditional marketing authorization to Talvey as a monotx. for adult patients with RRMM who received 3 prior therapies
  • The authorization was based on the P-I/II study (MonumenTAL-1) evaluating talquetamab, which showed ORR across 0.8mg/kg, q2w, and 0.4mg/kg, qw doses. 71.7% and 74.1% treated at 0.8mg/kg, q2w, and 0.4mg/kg, qw dose achieved a response with a median follow-up of 12.7 and 18.8mos, VGPR or better (60.8% and 59.5%) and CR (38.7% and 33.6%), respectively
  • Responses were durable with m-DoR (not reached and 9.5mos.) in both doses; 76.3% and 51.5% maintained a response for 9mos., adverse reactions leading to treatment discontinuation due to ICANS (1.1%) & weight loss (0.9%). The results were presented at ASCO & EHA 2023

4. Iveric Bio Reports EMA Acceptance of MAA for Avacincaptad Pegol to Treat Geographic Atrophy


Active ingredient: Avacincaptad Pegol                                  Approved: Aug 18, 2023

Company: Iveric Bio                                                                 Disease: Geographic Atrophy

  • The EMA has accepted the MAA for regulatory review of avacincaptad pegol, a complement C5 inhibitor for the treatment of GA secondary to AMD. The MAA was based on the results from the P-III (GATHER1 & 2) trials evaluating safety and efficacy of ACP (qm, 2mg, IVT) in 286 & 448 patients
  • The primary analysis showed a significant reduction in the rate of GA growth in patients treated with ACP over sham in each registrational trial over a 12mos. period while safety was evaluated in ~700 patients across both trials
  • Avacincaptad pegol, a complement C5 protein inhibitor was approved in the US as Izervay for the treatment of GA secondary to AMD

5. Regeneron Reports EMA Acceptance of MAA for Odronextamab to Treat Relapsed/Refractory Follicular Lymphoma and Diffuse Large B-cell Lymphoma

REGN 1979

Active ingredient: Odronextamab                                        Approved: Aug 18, 2023

Company: Regeneron                                                            Disease: Follicular Lymphoma and DLBCL

  • The EMA has accepted the MAA for review of odronextamab (CD20xCD3 bispecific Ab) for adult patients with r/r FL or r/r DLBCL who have progressed after 2 prior systemic therapies. The MAA was based on the P-I study (ELM-1) and P-II trial (ELM-2) evaluating odronextamab in FL and DLBCL
  • Both studies' results presented at ASH for r/r FL showed ORR (82%) in patients with grades 1 to 3a disease, CR (75%), median duration of complete response (mDOCR) was 20.5mos., m-PFS (20mos.), m-OS (not reached)
  • Among CAR-T naïve & post-CAR-T patients showed ORR (49% & 48%), CR (31% & 32%), mDOCR was 18mos. & not reached, respectively for r/r DLBCL. The company continues to advance odronextamab in the P-III development program for earlier lines of therapy & additional B-NHLs

6. BMS’ Opdivo (nivolumab) Receives EC’s Approval as an Adjuvant Treatment for Completely Resected Stage IIB or IIC Melanoma


Active ingredient: nivolumab                                                   Approved: Aug 23, 2023

Company: BMS                                                                          Disease: Melanoma

  • The EC has approved Opdivo as monotx. for the adjuvant treatment of adults & adolescents aged ≥12yrs. with stage IIB or IIC melanoma who have undergone complete resection
  • The approval was based on the results from the P-III trial (CheckMate -76K) evaluating Opdivo (480mg, q4w for ~12mos.) vs PBO which showed a 58% reduction in risk of recurrence or death with a minimum follow-up of 7.8mos., 12mos. RFS rates were 89% vs 79%, RFS benefit was observed across predefined subgroups incl. T category and disease stage
  • The safety profile was consistent with prior reported studies. Opdivo (PD-1 immune checkpoint inhibitor) is currently approved in 65+ countries, incl. the US, the EU, Japan, and China

7. Pfizer’s Abrysvo Receives EC’s Marketing Authorization to Protect Infants through Maternal Immunization and Older Adults from RSV


Active ingredient: RSVpreF                                                       Approved: Aug 25, 2023

Company: Pfizer                                                                         Disease: Respiratory Syncytial Virus

  • The EC has granted marketing authorization for Abrysvo, the RSVpreF vaccine for older adults & for immunization of pregnant individuals to help protect their infants immediately from birth through 6mos.
  • The authorization was based on the P-III study (RENOIR) in adults aged ≥60yrs. & (MATISSE)  trial in infants born to healthy individuals vaccinated during pregnancy evaluating the efficacy, immunogenicity & safety of Abrysvo
  • In the (RENOIR) study, the RSVpreF vaccine prevented RSV-associated LRTD and RSV-associated acute respiratory illness in adults aged ≥60yrs. while the vaccine administered during pregnancy in (MATISSE) trial was effective in infants with no safety concerns. The authorization is valid in all 27 EU member states, Iceland, Liechtenstein & Norway

8. Roche's Evrysdi Receives the EC’s Marketing Authorization Extension for Newborns with Spinal Muscular Atrophy


Active ingredient: risdiplam                                                 Approved: Aug 29, 2023

Company: Roche                                                                   Disease: Spinal Muscular Atrophy

  • The EC has expanded the marketing authorization for Evrysdi to include infants with SMA Type 1, Type 2, or Type 3 or with one to four SMN2 copies from birth to ≤2mos.
  • The approval was based on the interim analysis from the (RAINBOWFISH) study. All 6 infants incl. in the analysis were able to sit after 1yr. of treatment with Evrysdi i.e., 100% (6/6) while 67% (4/6) could stand, 50% (3/6) walked independently & all infants were alive at 12mos. without permanent ventilation
  • The safety profile of Evrysdi in pre-symptomatic babies was consistent with the safety profile in prior trials. Evrysdi is currently being studied in the P-II/III trial (MANATEE) in combination with an anti-myostatin molecule targeting muscle growth for SMA

9. Merck’s Keytruda (pembrolizumab) Receives EC’s Approval in Combination with Trastuzumab as 1L Treatment for HER2-Positive Advanced Gastric Cancer


Active ingredient: pembrolizumab                                      Approved: Aug 29, 2023

Company: Merck                                                                   Disease: Gastric Cancer

  • The EC has approved Merck’s Keytruda (anti-PD-1 therapy) in combination with trastuzumab, fluoropyrimidine- and Pt-containing CT as 1L treatment of LA unresectable or metastatic HER2+ gastric or GEJ adenocarcinoma in adults whose tumors express PD-L1
  • The approval was based on results from the P-III trial (KEYNOTE-811) evaluating Keytruda (200mg, q3w) + trastuzumab and CT vs trastuzumab and CT alone in 732 patients which showed significant improvement in PFS, & ORR, ≥80% of patients had tumors that were PD-L1+
  • The trend toward improvement in OS in the ITT population but these results did not meet statistical significance per the pre-specified statistical analysis plan. The results will be presented at ESMO 2023

10. Pfizer and BioNTech Receive the EMA’s CHMP Positive Opinion Recommending Marketing Authorization for Omicron XBB.1.5-adapted COVID-19 Vaccine

Comirnaty Omicron XBB.1.5

Active ingredient: raxtozinameran                                       Approved: Aug 30, 2023

Company: Pfizer and BioNTech                                           Disease: COVID-19

  • The EMA’s CHMP has recommended marketing authorization for Omicron XBB.1.5-adapted monovalent COVID-19 vaccine (Comirnaty Omicron XBB.1.5) as a single dose for individuals aged ≥5yrs.
  • The recommendation was based on the previous clinical, non-clinical & real-world evidence of COVID-19 vaccines. The recommendation is also based on pre-clinical data showing that the COVID-19 vaccine generates an improved response against multiple XBB-related sublineages
  • The committee also recommended the updated vaccine for children aged 6mos. to 4yrs. given as part of all of the primary three-dose vaccination series or as a single dose for individuals with a complete COVID-19 primary vaccination course. In the pre-clinical data, the updated vaccine induces serum Abs that neutralize the WHO-designated variant of interest EG.5.1 (Eris)

11. InflaRx Report EMA’s Validation of MAA for Vilobelimab to Treat Critically Ill COVID-19 Patients


Active ingredient: Vilobelimab                                               Approved: Aug 30, 2023

Company: InflaRx                                                                    Disease: COVID-19

  • The EMA has validated the MAA for Vilobelimab to treat critically ill COVID-19 patients. The application is now under regulatory review by CHMP under the centralized procedure which valid to all 27 member states of the EU
  • The submission was based on the P-III trial (PANAMO) trial evaluating vilobelimab in invasively mechanically ventilated COVID-19 patients in intensive care units which showed that vilobelimab improved survival with a relative reduction in 28-day all-cause mortality of 23.9%
  • The results were published in The Lancet Respiratory Medicine. The company continues to discuss with the US FDA related to the submission of a BLA for full approval of Gohibic (vilobelimab)

Note: Omicron XBB.1.5-adapted COVID-19 Vaccine received EMA’s CHMP positive opinion; EC’s Marketing Authorization for Abrysvo & Evrysdi; EMA’s Validation of MAA for Vilobelimab; Type II variation application approval from EC for Tecvayli and EMA Acceptance of MAA for Avacincaptad Pegol & Odronextamab

Related Post: Insights+: EMA Marketing Authorization of New Drugs in July 2023

Senior Editor

Neha is a Senior Editor at PharmaShots. She is passionate and very enthusiastic about recent updates and developments in the life sciences and pharma industry. She covers Biopharma, MedTech, and Digital health segments along with different reports at PharmaShots. She can be contacted at connect@pharmashots.com.

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