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Drug approvals in Europe| EMA drug approvals | 2023 |European Commission News| iPharmaCenter


European Commission (EC) has granted conditional marketing authorization for AbbVie's Tepkinly (epcoritamab) as a standalone treatment for adult patients facing relapsed or refractory (R/R) diffuse large B-cell lymphoma (DLBCL) after undergoing two or more lines of systemic therapy. This marks a significant milestone as Tepkinly becomes the first and only subcutaneous bispecific antibody to receive approval for treating this patient group within the European Union (EU) and in Liechtenstein, Norway, and Iceland.


DLBCL is the most prevalent form of B-cell non-Hodgkin's lymphoma globally. Patients grappling with this condition often have access to chemoimmunotherapy regimens for disease management. However, there is a pressing need for additional treatment options, particularly for those individuals whose disease has either relapsed or developed resistance to previous therapeutic interventions.


The conditional marketing approval for Tepkinly is based on data derived from the pivotal EPCORE NHL-1 Phase 1/2 clinical trial, which encompassed various cohorts and centres and was conducted in an open-label, single-arm manner. This trial aimed to assess the preliminary efficacy and safety of Tepkinly in patients with R/R large B-cell lymphoma (LBCL), including the DLBCL subtype.


Within the DLBCL patient group (comprising 139 individuals) participating in the study, those treated with Tepkinly exhibited an impressive overall response rate of 62 percent, with a complete response rate of 39 percent. The median duration of response was 15.5 months, with a range spanning from 9.7 months to not yet determined.

Crucially, the safety profile of Tepkinly across the entire LBCL patient cohort (which included DLBCL patients, totalling 167) was manageable. The most frequently observed adverse reactions in more than 20 percent of patients included cytokine release syndrome, fatigue, neutropenia, injection site reaction, musculoskeletal pain, abdominal pain, pyrexia, nausea, and diarrhoea.


Tepkinly represents a collaborative effort between AbbVie and Genmab as part of their ongoing oncology partnership. Commercial responsibilities for Tepkinly will be shared by the two companies in the United States and Japan, with AbbVie taking the lead in the global commercialization efforts. AbbVie intends to pursue regulatory submissions for epcoritamab across various international markets in the coming year, underscoring its commitment to expanding access to this innovative treatment option.


Merck received approval from the European Commission (EC) for Keytruda (pembrolizumab) plus trastuzumab, fluoropyrimidine- and platinum-containing chemotherapy for locally advanced unresectable or metastatic human epidermal growth factor receptor 2 (HER2)-positive gastric or gastroesophageal junction (GEJ) adenocarcinoma.



The EC's decision comes after a favorable recommendation from the Committee for Medicinal Products for Human Use in July 2023 and is founded on data from the Phase 3 KEYNOTE-811 trial. In this trial, the combination of Keytruda, trastuzumab, and chemotherapy exhibited significant benefits regarding progression-free survival (PFS) and objective response rate (ORR) versus trastuzumab and chemotherapy alone for this patient group. Notably, over 80% of the patients in the study had PD-L1-positive tumours.


The KEYNOTE-811 trial employed dual primary endpoints, including PFS according to RECIST v1.1 as assessed by blinded independent central review and OS. Secondary endpoints encompass ORR, duration of response, and safety. The trial enrolled an estimated 732 patients randomly assigned to either receive Keytruda (200 mg every three weeks) combined with trastuzumab and chemotherapy (chosen by the investigator) or placebo alongside trastuzumab and chemotherapy.


While there was a positive trend observed in overall survival (OS) among patients who received the Keytruda combination compared to those who received a placebo in combination with trastuzumab and chemotherapy, these results did not reach statistical significance as per the pre-defined statistical analysis plan. Merck announced that the ongoing analysis of OS of this trial will continue.


Bristol Myers Squibb has received approval from the European Commission (EC) for Opdivo (nivolumab) in combination with chemotherapy as a neoadjuvant treatment for resectable non-small cell lung cancer (NSCLC) in patients at high risk of recurrence, with tumor cell PD-L1 expression ≥1%.


The approval is based on Phase 3 CheckMate -816 trial, which demonstrated that neoadjuvant treatment with Opdivo and chemotherapy improved event-free survival and pathologic complete response compared to chemotherapy alone. This approval makes Opdivo with chemotherapy the first immunotherapy-based neoadjuvant treatment option approved for NSCLC patients in the European Union.



This decision expands the use of Opdivo-based regimens in earlier stages of cancer in the European Union, following approvals for melanoma, esophageal/gastroesophageal junction cancer, and urothelial carcinoma. Bristol Myers Squibb announced the EC's approval of Opdivo in combination with platinum-based chemotherapy for the neoadjuvant treatment of resectable NSCLC at high risk of recurrence and with tumor cell PD-L1 expression ≥1%.


Opdivo with chemotherapy has also been approved for neoadjuvant treatment in patients with resectable NSCLC regardless of PD-L1 expression levels in several countries, including the United States, Japan, and China. Additional regulatory applications for Opdivo in earlier stages of cancer are under review by global health authorities.


Event-Free Survival (EFS) - The median EFS for patients receiving Opdivo in combination with chemotherapy before surgery was 31.6 months, versus to 20.8 months for patients receiving chemotherapy alone. This represents a 37% reduction in the risk of an event or death.


Pathologic Complete Response (pCR) - Among patients treated with Opdivo in combination with chemotherapy prior to surgery, 24% achieved pCR, meaning no evidence of cancer cells in the resected tissue. In comparison, only 2.2% of patients treated with chemotherapy alone achieved pCR.


Overall Survival (OS) - Opdivo with chemotherapy reduced the risk of death by 43% versus chemotherapy.


Safety - In the pooled dataset of patients receiving Opdivo with chemotherapy across different tumor types (n=1268), the most common side effects were nausea (51%), peripheral neuropathy (39%), fatigue (39%), diarrhea (33%), decreased appetite (33%), constipation (31%).

Bristol Myers Squibb, a pharmaceutical company, has recently received approval from the European Commission (EC) for its innovative medication called Camzyos (mavacamten). This approval allows Camzyos to treat symptomatic obstructive hypertrophic cardiomyopathy (HCM) in adult patients in all European Union member states.


Camzyos is a groundbreaking medication as it is the first and only allosteric and reversible inhibitor selective for cardiac myosin to be approved in the European Union. By explicitly targeting cardiac myosin, Camzyos addresses the underlying pathophysiology of HCM, making it a significant advancement in cardiovascular medicine.


The approval of Camzyos by the European Commission was based on the compelling efficacy and safety results from two crucial Phase 3 trials: EXPLORER-HCM and VALOR-HCM. These trials demonstrated substantial benefits for patients treated with Camzyos compared to those who received a placebo.


Symptomatic obstructive HCM is a prevalent and often-inherited heart disease that can have a chronic, debilitating, and progressive impact on individuals. Furthermore, they are at risk of severe complications, including heart failure, arrhythmias, stroke, and, in rare cases, sudden cardiac death.


The EXPLORER-HCM Phase 3 trial enrolled 251 adult patients with symptomatic obstructive HCM. The study participants had specific criteria, including measurable left ventricular ejection fraction (LVEF) and significant left ventricular outflow tract (LVOT) gradient. The primary endpoint of this trial was a composite functional endpoint, assessed at 30 weeks, which evaluated improvements in mixed venous oxygen tension, NYHA class, and other parameters. At Week 30, 37% of patients achieved composite endpoint versus 17% in patients on placebo.


Similarly, the VALOR-HCM trial investigated Camzyos in patients with symptomatic obstructive HCM who met the criteria for septal reduction therapy. The study demonstrated Camzyos' efficacy in reducing the need for invasive procedures or maintaining eligibility for such procedures. The primary endpoint measured the patient's decision to undergo septal reduction therapy (SRT) before or at Week 16 or their continued eligibility for SRT based on specific criteria such as a left ventricular outflow tract (LVOT) gradient of ≥50 mmHg and NYHA class III-IV, or class II with exertional syncope or near syncope. After 16 weeks of treatment, 82% of patients either became ineligible for the surgical procedure or chose not to proceed with SRT. In contrast, only ten patients (17.9%) who received Camzyos decided to proceed with SRT or remained eligible for SRT at Week 16, compared to 43 patients (76.8%) in the placebo group.


Regarding safety, the pooled data from both trials showed that the most commonly reported adverse reactions associated with Camzyos included dizziness, dyspnea, systolic dysfunction, and syncope. It is worth noting that reversible reductions in left ventricular ejection fraction (LVEF) were observed in a small percentage of patients but resolved upon discontinuing Camzyos treatment.


The approval of Camzyos by the European Commission provides a new therapeutic avenue for individuals suffering from symptomatic obstructive HCM, offering hope for improved outcomes and enhanced quality of life.

UCB, a global biopharmaceutical company, has received new approvals from the European Commission (EC) for their drug Bimzelx (bimekizumab) in the treatment of psoriatic arthritis (PsA) and axial spondyloarthritis (axSpA). Bimekizumab is the first and only inhibitor of IL-17A and IL-17F to be approved in the European Union for these conditions. This marks the first authorization for bimekizumab in PsA and axSpA globally, following its previous EMA approval for moderate to severe plaque psoriasis in August 2021.


The approval for PsA is supported by data from two Phase 3 studies called BE OPTIMAL and BE COMPLETE. Bimekizumab significantly improved joint and skin symptoms compared to placebo in biologic-naïve patients and those with inadequate response to TNF inhibitors. The primary endpoint of achieving ACR50 response at Week 16 was met, and sustained clinical responses were observed up to Week 52, as measured by ACR50, PASI90, PASI100, and Minimal Disease Activity (MDA) criteria.


Joint Symptoms, ACR50: In bDMARD naive and cDMARD treated patients, at week 16, 45% and 44% achieved primary endpoints versus 13% and 6% in patients on placebo.


For axSpA, the EC approval is based on findings from the Phase 3 studies BE MOBILE 1 and BE MOBILE 2. Bimekizumab met the primary endpoint of the Assessment of SpondyloArthritis International Society (ASAS) 40 response at Week 16 compared to the placebo. The drug showed improvements in signs, symptoms, and disease activity across the spectrum of the disease. ASAS40 responses were consistent in TNF-naïve and TNF-inadequate responder patients, and sustained clinical responses were observed up to Week 52.

Assessment of SpondyloArthritis international Society (ASAS) response: 47.7% of patients with nr-axSpA and 44.8% of patients with AS met the primary endpoint versus 21.4% and 22.5% of patients on placebo.


Jefferies estimated Bimzelx could generate peak revenues of $3.6 billion.

Novartis has received approval from the European Commission (EC) for Cosentyx (secukinumab) as a treatment for moderate to severe hidradenitis suppurativa (HS) in adults who have not responded adequately to conventional systemic HS therapy.

European Commission approved HS therapy after almost a decade post the approval of Humira.

The decision by the EC was based on data from Phase III clinical trials, including the SUNSHINE (NCT03713619) and SUNRISE (NCT03713632) trials. The efficacy, safety, and tolerability of two different dosing regimens of Cosentyx were evaluated in adults with moderate to severe HS over a short-term (16 weeks) and long-term (up to 52 weeks) period.

The trials demonstrated that, with the treatment with Cosentyx, response rates continued to improve over one year, with more than 55% of patients achieving a Hidradenitis Suppurativa Clinical Response (HiSCR) at Week 52. These findings highlight the clinically meaningful benefits of Cosentyx in alleviating the most debilitating symptoms of HS.

HS is a chronic inflammatory skin disease characterised by painful and potentially disfiguring abscesses. It affects approximately 200,000 people in Europe with moderate to severe stages of the condition.


Bristol Myers Squibb (BMS) has received approval from the European Commission for Breyanzi (lisocabtagene maraleucel), a chimeric antigen receptor (CAR) T-cell therapy, to treat adults with relapsed or refractory large B-cell lymphoma who were previously treated with one therapy.


The approved indications include the following:

  • Diffuse large B-cell lymphoma (DLBCL).

  • High-grade B-cell lymphoma (HGBCL).

  • Primary mediastinal large B-cell lymphoma (PMBCL).

  • Follicular lymphoma grade 3B (FL3B).


The approval was based on the Phase 3 TRANSFORM trial, in which Breyanzi was compared with standard therapy (intense salvage immunochemotherapy followed by high-dose chemotherapy and HSCT). The event-free survival (EFS) was 10.4 months in Breyanzi versus 2.3 months in the interim analysis. At 17.5 months during preliminary analysis, the results were consistent with the interim analysis; median EFS was not reached in patients who were treated wtih Breyanzi versus 2.4 months in patients on chemotherapy. Nearly 74% of patients achieved complete response versus 43.5% of patients who were treated with standard therapy. In patients on Breyanzi, median PFS was not reached versus 6.2 months in patients on standard treatment.


The safety profile was consistent with the established profile of Breyanzi with low-grade cytokine release syndrome (CRS) and neurologic events.

Janssen announced the first approval of Akeegar (niraparib and abiraterone acetate), a dual-action tablet approved for treating adults with metastatic castration-resistant prostate cancer (mCRPC). It is indicated for patients who are not eligible for chemotherapy.


The combination has two drugs with two different action mechanisms that act on the slow cancer progression. Niraparib is a PARP inhibitor that prevents cancer cells from repairing their DNA. At the same time, abiraterone acetate is a hormone therapy that lowers testosterone levels.



The approval of Akeegar was based on data from the Phase 3 MAGNITUDE, which showed that the combination tablet significantly prolonged radiographic progression-free survival compared to abiraterone acetate alone in patients with mCRPC.


The approval was based on the placebo-controlled Phase 3 MAGNITUDE trial; the primary endpoint is radiographic progression-free survival (rPFS). The risk of disease progression was reduced by 47%; the rPFS was 19.5 months versus 10.9 months in patients on placebo plus AAP. There was a trend in the improvement of overall survival.


The combination's safety profile was consistent with each drug's safety.


Bristol Myers Squibb's Sotyktu (deucravacitinib), an oral drug for moderate to severe plaque psoriasis, has received approval from the European Commission for adult patients.

With the availability of Sotyktu, patients with moderate to severe plaque psoriasis in Europe now have an additional treatment option that offers convenience and efficacy in a once-daily oral medication.


The approval was based on the Phase 3 POETYK PSO-1 and POETYK PSO-2 clinical trials, demonstrating the efficacy of Sotkyu versus Otezla, a twice-daily tablet at 16 and 24 weeks. The primary endpoints in both trials were sPGA 0/1 at week 16 and PASI 75 at week 16.


Sotkyu is a TYK2 inhibitor developed for other indications, including psoriatic arthritis, systemic lupus erythematosus, Crohn's disease, lupus nephritis, and ulcerative colitis.


Bristol Myers Squibb's Reblozyl (luspatercept) has received approval from the European Commission for treating anemia in adult patients with non-transfusion-dependent beta-thalassemia.

This approval is based on Phase 2 BEYOND study results, in which Reblozyl was tested for efficacy in 145 patients with NTD beta-thalassemia. The study's primary endpoint was ≥1.0 g/dL mean Hb increase from baseline, in which 77.1% of patients met the primary endpoint versus 0% in patients on placebo. A mean Hb increase of ≥1.5 g/dL from baseline between Weeks 37-48 was included as a key secondary endpoint; 49% of patients achieved met the endpoint versus 0% of patients on placebo.


Reblozyl binds to TGF-beta superfamily ligands, diminishing Smad2/3 signaling and helping patients produce healthy red blood cells. The US FDA previously approved it for treating anemia in patients with beta-thalassemia who require regular red blood cell transfusions and anemia failing an erythropoiesis-stimulating agent.

Reblozyl is developed through a collaboration between BMS and Merck.


 

Bayer announced that European Commission approved Nubeqa (darolutamide) for treating adult patients with metastatic hormone-sensitive prostate cancer (mHSPC). Previously, it was approved for non-metastatic castration-resistant prostate cancer (nmCRPC) who were at risk of developing metastatic disease.


The approval was based on the Phase III ARASENS trial. The combination of Nubeqa, ADT, and docetaxel significantly reduced the risk of death by 32.5% versus ADT plus docetaxel combination. The adverse events were similar in both treatment arms.


Bayer and Orion Corporation developed Nubeqa.

 


AstraZeneca announced that Enhertu (trastuzumab deruxtecan) was approved in the European Union (EU) as monotherapy in patients with unresectable or metastatic HER2-low breast cancer. It is indicated in patients who received prior chemotherapy.

The approval was based on the DESTINY-Breast 04 trial, in which Enhertu reduced the risk of disease progression or death by 50%. The overall survival was increased by six months versus chemotherapy. The progression-free survival was 9.9 months in patients on Enhertu versus 5.1 months in patients on chemotherapy. The risk of death was reduced by 36%, with median overall survival of 23.4 months versus 16.8 months in patients on chemotherapy.

In March 2019, Daiichi Sankyo and AstraZeneca entered a collaboration to develop and commercialise Enhertu.


 

Roche announced that the European Commission approved Xofluza (baloxavir marboxil) for treating children aged one year and older, uncomplicated influenza, and post-exposure prophylaxis.

The approval was based on two Phase 3 trials, miniSTONE-2 and BLOCKSTONE. In the miniSTONE-2 trial, Xofluza reduced the length of time of influenza release versus oseltamivir (24.2 hours versus 75.8 hours).


In the BLOCKSTONE study, Xofluza showed a significant prophylactic effect versus placebo (1.9% in patients treated with Xofluza versus 13.6% in patients on placebo).


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