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26th March 2024
The antibiotic aztreonam-avibactam (brand name Emblaveo) has received a positive opinion to treat multidrug-resistant infections from the European Medicines Agency (EMA)’s Committee for Medicinal Products for Human Use (CHMP), its manufacturer Pfizer has announced.
Aztreonam-avibactam is recommended for use in the treatment of adult patients with complicated intra-abdominal infections (cIAI), hospital-acquired pneumonia (HAP) including ventilator-associated pneumonia (VAP), and complicated urinary tract infections (cUTI), including pyelonephritis.
The antibiotic combination, which is administered via an infusion into a vein, will also be indicated for the treatment of infections due to aerobic Gram-negative organisms in adult patients with limited treatment options.
Aztreonam, a monobactam β-lactam, is already authorised for use in the EU on its own (brand name Cayston) and avibactam, a recent broad-spectrum β-lactamase inhibitor, is authorised for use in combination with the antibiotic ceftazidime (brand name Zavicefta).
The combination of aztreonam with avibactam restores aztreonam’s activity against bacteria that co-produce metallo-β-lactamases (MBLs) and other β-lactamases.
If approved, this new fixed-dose combination would be the first β-lactam/β-lactamase inhibitor combination for treating serious bacterial infections caused by multidrug-resistant Gram-negative bacteria, including MBL-producing bacteria, approved for use in the EU.
The drug combination was evaluated under EMA’s accelerated assessment mechanism due to it being considered of major public health interest.
The positive CHMP opinion is based on the safety and efficacy data already available for each active substance and the results of two phase 3 randomised studies REVISIT and ASSEMBLE.
While the studies were not designed to demonstrate efficacy, they do provide safety and complementary data for the combination suggesting it is a well-tolerated and effective treatment option against multidrug-resistant Gram-negative bacteria, the EMA said.
The most frequent side effects in patients treated with aztreonam-avibactam were a decrease in the number of red blood cells, elevated levels of liver transaminase and diarrhoea. This is in line with the documented safety information available for each individual substance.
Earlier in 2024, the new antibiotic combination cefepime-enmetazobactam (brand name Exblifep) received a positive CHMP opinion for the treatment of certain adult patients with complicated urinary tract infections.
13th March 2024
A type II variation of ciltacabtagene autoleucel (cilta-cel; brand name Carvykti) has received a positive opinion from the European Medicines Agency (EMA)’s Committee for Medicinal Products for Human Use (CHMP) for use in the treatment of eligible patients with multiple myeloma, its manufacturer Janssen has announced.
Cilta-cel has been recommended for adult patients with relapsed and refractory (R/R) multiple myeloma, who have received at least one prior therapy including an immunomodulatory agent and a proteasome inhibitor, demonstrated disease progression on the last therapy, and are refractory to lenalidomide.
Cilta-cel is a B-cell maturation antigen-directed chimeric antigen (CAR) T-cell therapy and is the first CAR T-cell therapy to receive a positive CHMP opinion for this patient population as early as second line.
It is currently approved under conditional marketing authorisation in the EU for the treatment of adults with R/R multiple myeloma, after three prior lines of therapy.
Edmond Chan, EMEA lead for haematology at Janssen-Cilag, said: ‘Early resistance to standard treatments is becoming more common in patients with lenalidomide-refractory multiple myeloma, highlighting a need for new options earlier in the course of treatment.’
He added that the CHMP recommendation ‘recognises the potential of cilta-cel to significantly improve outcomes for eligible patients with relapsed and refractory multiple myeloma, as early as after first relapse.’
The CHMP recommendation was based on the results of a phase 3 CARTITUDE-4 clinical trial comparing the CAR T-cell therapy directed against b-cell maturation antigen versus the standard care treatment, which was the physician’s choice of pomalidomide, bortezomib and dexamethasone or daratumumab, pomalidomide and dexamethasone.
The primary endpoint was progression-free survival, defined as the time from randomisation to the first documentation of disease progression or death.
In total, 419 patients with relapsed and lenalidomide-refractory multiple myeloma who had received one to three prior lines of therapy underwent randomisation, with 208 (mean age 61.5 years, 55.8% male) receiving cilta-cel.
Cilta-cel treatment gave rise to a significantly lower risk of disease progression or death compared to standard care (hazard ratio, HR = 0.26, 95% CI 0.18 – 0.38, p < 0.001).
After 12 months, PFS was 75.9% (95% CI 69.4 – 81.1) in the cilta-cel group and 48.6% (95% CI 41.5 – 55.3) in the standard care group. In addition, a higher proportion of patients in the cilta-cel group had an overall response (84.6% vs. 67.3%), a complete response or better (73.1% vs. 21.8%) and an absence of minimal residual disease (60.6% vs. 15.6%).
In terms of safety, Grade 3 or 4 adverse events occurred in a similar proportion of participants in the two groups (96.6% vs 94.2%, cilta-cel vs standard care).
1st March 2024
The monoclonal antibody tislelizumab (brand name Tevimbra) has received a positive opinion from the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) for the treatment of non-small cell lung cancer (NSCLC) across three indications, its manufacturer BeiGene has announced.
Tislelizumab has been recommended by the CHMP for use in combination with carboplatin and either paclitaxel or nab-paclitaxel for the first-line treatment of adult patients with squamous NSCLC who have locally advanced NSCLC and are not candidates for surgical resection or platinum-based chemoradiation, or metastatic NSCLC.
The second indication is for use in combination with pemetrexed and platinum-containing chemotherapy for the first-line treatment of adult patients with non-squamous NSCLC whose tumours have PD-L1 expression on ≥50% of tumour cells with no EGFR or ALK positive mutations and who have locally advanced NSCLC and are not candidates for surgical resection or platinum-based chemoradiation, or metastatic NSCLC.
Tislelizumab is also recommended as a monotherapy for the treatment of adult patients with locally advanced or metastatic NSCLC after prior platinum-based therapy. Patients with EGFR mutant or ALK positive NSCLC should also have received targeted therapies before receiving tislelizumab, the CHMP said.
The positive opinion for these indications is based on the results of three Phase 3 trials which demonstrated the benefit of tislelizumab as a first- and second-line treatment for patients with NSCLC.
Dr Mark Lanasa, chief medical officer, solid tumors at BeiGene, said: ‘Through three Phase 3 clinical trials enrolling nearly 1,500 patients across the world including in the European Union, tislelizumab has been shown to be an effective therapy for patients with treatment-naïve and treatment-resistant NSCLC.’
He added that the positive CHMP opinion ‘brings us one step closer to providing an important treatment option to patients in Europe with lung cancer, which is among the most common cancers and a leading cause of cancer death in the region.’
The RATIONALE 307 trial looked at first-line tislelizumab in combination with chemotherapy for patients with advanced squamous NSCLC.
It met its primary endpoints with the median progression free survival at 7.7 months for tislelizumab in combination with paclitaxel and carboplatin (hazard ratio, HR: 0.45 [95% CI: 0.326-0.619]; P< 0.001) and 9.6 months for tislelizumab in combination with nab-paclitaxel and carboplatin (HR: 0.43 [95% CI: 0.308-0.60]; P< 0.001).
This was compared to 5.5 months for paclitaxel and carboplatin alone, at a median study follow-up of 8.6 months.
The most common grade ≥3 treatment emergent adverse events were decreased neutrophil levels, neutropenia and leukopenia.
RATIONALE 304 also considered tislelizumab in combination with chemotherapy as first-line treatment and focused on patients with locally advanced or metastatic non-squamous NSCLC.
The study met its primary endpoint, with first-line tislelizumab in combination with chemotherapy resulting in statistically significant improvement in progression free survival compared to chemotherapy (HR: 0.65 [95% CI: 0.47-0.91]; P=0.0054) along with higher response rates and longer response duration.
The most common grade ≥3 treatment emergent adverse events were associated with chemotherapy and included neutropenia and leukopenia.
Looking at second- and third-line treatment, the RATIONALE 303 trial looked at tislelizumab versus docetaxel in patients with advanced NSCLC who progressed on prior platinum-based chemotherapy.
This trial met its primary endpoint, with second- or third-line tislelizumab resulting in statistically significant and clinically meaningful improvement in overall survival compared with docetaxel in the intent-to-treat population (HR: 0.66 [95% CI: 0.56-0.79]; P<0.0001), regardless of PD-L1 expression.
At the final analysis, overall survival in the PD-L1 positive population was also significantly improved in favour of tislelizumab (median 19.3 vs 11.5 months, respectively; HR: 0.53 [95% CI: 0.41-0.70]; P<0.0001).
The most commonly reported grade ≥3 treatment emergent adverse events were pneumonia, anaemia and dyspnoea.
30th January 2024
The new antibiotic combination cefepime/enmetazobactam (brand name Exblifep) has received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) for the treatment of complicated urinary tract infections (cUTI), its manufacturer Advanz Pharma has announced.
The intravenous formulation is a fixed-dose combination of enmetazobactam – a novel extended-spectrum-lactamase inhibitor belonging to the penicillanic acid sulfone class – with the 4th generation cephalosporin cefepime.
This enhances the efficacy of cefepime against resistant bacteria, including extended spectrum beta-lactamase (ESBL)-producing pathogens.
Cefepime/enmetazobactam has been given the green light in the EU for the treatment of adult patients with cUTIs, including pyelonephritis; hospital-acquired pneumonia (HAP), including ventilator associated pneumonia (VAP); and the treatment of patients with bacteraemia that occurs in association with, or is suspected to be associated with, any of the infections listed.
The drug was designed as a new antibiotic combination to combat the growing antimicrobial resistance in Gram-negative bacteria, Advanz Pharma said.
The CHMP recommendation was based on the results of the phase 3 randomised, double-blind, multi-centre ALLIUM trial, which was undertaken at 112 sites in 19 countries.
The trial considered how the efficacy of cefepime/enmetazobactam compared with piperacillin/tazobactam for the treatment of cUTIs or acute pyelonephritis (AP) and the primary efficacy outcome was clinical cure and microbiological eradication.
Eligible patients were randomised to receive either cefepime, 2 g/enmetazobactam, 0.5 g (n = 520), or piperacillin, 4 g/tazobactam, 0.5 g (n = 521), by two-hour infusion every eight hours for seven days (up to 14 days in patients with a positive blood culture at baseline).
Cefepime/enmetazobactam demonstrated statistically significant superior overall treatment success (clinical cure combined with microbiological eradication) at a test-of-care visit compared with piperacillin/tazobactam in cUTI, including AP, caused by Gram-negative pathogens (79.1% vs 58.9%).
Statistically significantly superior results were also observed among patients with infections caused by ESBL-producing pathogens (73.7% vs 51.5%, respectively).
Cefepime/enmetazobactam demonstrated a tolerable safety profile, comparable to piperacillin/tazobactam.
Treatment-related serious adverse events were reported in 0.2% of patients treated with cefepime/enmetazobactam versus 0.6% of patients treated with piperacillin/tazobactam.
The European Medicines Agency noted the most common side effects as pain and inflammation at the infusion site, diarrhoea, skin rash and headache.
According to Advanz Pharma, cefepime/enmetazobactam has shown promising in vitro activity against the more resistant beta-lactamase mutations OXA-48 and AmpC, which are increasing in Europe and for which there are few therapeutic alternatives.
18th December 2023
Etrasimod (brand name Velsipity) has received a positive opinion from the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) for use in eligible patients with ulcerative colitis (UC), its manufacturer Pfizer has announced.
The oral, one-daily selective sphingosine-1-phosphate (S1P) receptor modulator selectively activates S1P receptor subtypes 1, 4 and 5, with no detectable activity on S1P.
It has been given the green light in the EU for the treatment of patients aged 16 years and older with moderately to severely active ulcerative colitis who have had an inadequate response, lost response, or were intolerant to either conventional therapy, or a biological agent.
If etrasimod were to subsequently be approved by the European Commission, it would represent the first global approval of an oral advanced ulcerative colitis therapy for use in older adolescents.
Velsipity is currently approved in the US, to treat adults with moderately to severely active ulcerative colitis.
Michael Corbo, chief development officer, inflammation and immunology, Pfizer Global Product Development, said: ‘Ulcerative colitis is a chronic condition that affects over 2.6 million people in Europe, and can have a debilitating effect on patients’ lives. If approved, Velsipity could offer patients with moderately to severely active ulcerative colitis the opportunity to achieve steroid-free remission.
‘This positive recommendation is a significant step forward in Pfizer’s efforts to bring this convenient once-daily oral treatment to appropriate patients in the EU affected by ulcerative colitis who require an advanced treatment option with a favourable benefit-risk profile.’
The CHMP positive opinion was based on results from the phase 3 ELEVATE UC 52 and ELEVATE UC 12 trials.
They evaluated the safety and efficacy of etrasimod 2 mg once daily on clinical remission in ulcerative colitis patients who had previously failed or were intolerant to at least one conventional, biologic or Janus kinase inhibitor therapy.
The coprimary endpoints in ELEVATE UC 52 were the proportion of patients who achieved clinical remission at week 12 (induction period) and week 52 (maintenance period).
The researchers found a significantly greater proportion of patients in the etrasimod group achieved clinical remission compared with patients in the placebo group at completion of the 12-week induction period (74 [27%] of 274 patients vs 10 [7%] of 135 patients) and at week 52 (88 [32%] of 274 patients vs 9 [7%] of 135 patients).
The primary endpoint for ELEVATE UC 12 was the proportion of patients in clinical remission at the end of the 12-week induction period.
Some 55 (25%) of 222 patients in the etrasimod group had clinical remission compared with 17 (15%) of 112 patients in the placebo group at the end of the 12-week induction period.
Both studies also achieved all key secondary efficacy endpoints, with a favourable safety profile consistent with previous studies of etrasimod.
The most common adverse reactions were lymphopenia (11%) and headache (7%).
The drug also demonstrated improvement in the total inflammatory bowel disease questionnaire score, which measures health-related quality of life.
20th November 2023
The Janus kinase (JAK) inhibitor momelotinib has received a positive opinion from the European Medicines Agency‘s Committee for Medicinal Products for Human Use (CHMP) for use in certain myelofibrosis patients, its manufacturer GSK has announced.
Momelotinib has been recommended for the treatment of disease-related splenomegaly or symptoms in adult patients with moderate-to-severe anaemia who have primary myelofibrosis, post polycythaemia vera myelofibrosis or post-essential thrombocythaemia myelofibrosis and who are JAK inhibitor naive or have been treated with ruxolitinib.
If approved, momelotinib (proposed brand name Omjjara) would be the only medicine in the European Union specifically indicated for both newly diagnosed and previously treated myelofibrosis patients with moderate-to-severe anaemia that addresses splenomegaly and symptoms.
Nina Mojas, senior vice president, oncology global product strategy at GSK, said: ‘Momelotinib has a differentiated mechanism of action that may address the significant medical needs of myelofibrosis patients, especially those with moderate to severe anaemia.
‘The vast majority of myelofibrosis patients will develop anaemia, causing them to require transfusions and leading a notable proportion to discontinue treatment.
‘This positive CHMP opinion is a significant step in bringing momelotinib to patients in the EU with this difficult-to-treat blood cancer.‘
The positive opinion was based on the results of the pivotal MOMENTUM trial, which evaluated the safety and efficacy of momelotinib versus danazol for the treatment and reduction of key manifestations of myelofibrosis in an anaemic, symptomatic, JAK inhibitor-experienced population.
All primary and key secondary endpoints were met, and momelotinib demonstrated a statistically significant response with respect to constitutional symptoms, splenic response and transfusion independence compared to danazol at Week 24.
Momelotinib was also associated with favourable safety at Week 48 in a follow-up analysis.
The CHMP recommendation was also supported by data from a subpopulation of adult patients with moderate-to severe-anaemia (haemoglobin <10 g/dL) from the SIMPLIFY-1 phase 3 trial.
This evaluated the efficacy and safety of momelotinib versus ruxolitinib in myelofibrosis patients who had not received a prior JAK-inhibitor therapy.
It found that in JAKi-naive patients with myelofibrosis, 24 weeks of momelotinib treatment was non-inferior to ruxolitinib for spleen response but not for symptom response. Momelotinib treatment was also associated with a reduced transfusion requirement.
In these clinical trials, the most common adverse reactions were diarrhoea, thrombocytopaenia, nausea, headache, dizziness, fatigue, asthenia, abdominal pain and cough.
A decision on EU marketing authorisation is expected for momelotinib by early 2024.
Momelotinib was approved by the US Food and Drug Administration in September 2023 under the brand name Ojjaara. It is currently not approved in any other market.
20th October 2023
Rucaparib has been given a positive opinion as a first-line maintenance treatment for patients with advanced ovarian cancer by the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP), its manufacturer Pharma& has announced.
Suitable for all women with advanced ovarian cancer, regardless of BRCA mutation status, who have responded to first-line platinum-based chemotherapy, rucaparib (brand name Rubraca) is a poly-ADP ribose polymerase (PARP) inhibitor.
The targeted cancer drug is currently approved as a monotherapy for the maintenance treatment of adult patients with platinum-sensitive relapsed high-grade epithelial ovarian, fallopian tube, or primary peritoneal cancer who have a complete or partial response to platinum-based chemotherapy.
The recent recommended approval from the CHMP is a Type II variation on the current license and is based on the randomised, double-blind, placebo-controlled, phase 3 ATHENA-MONO trial results.
The trial demonstrated that rucaparib significantly improved investigator-assessed progression-free survival compared with placebo in women, regardless of their BRCA mutation status in each of the populations studied.
The safety profile observed in the ATHENA-MONO trial was consistent with both the current US and European labels for rucaparib.
Dr Rebecca Kristeleit, consultant medical oncologist and adjunct reader at Guy’s and St. Thomas’ NHS Foundation Trust and King’s College London, and European Network of Gynaecological Oncological Trial (ENGOT) lead of the ATHENA trial, said: ‘In the ATHENA-MONO trial, rucaparib prolonged progression-free survival, irrespective of molecular characteristics, and its potential approval by the European Medicines Agency as a first-line maintenance treatment is an important step forward in this difficult-to-treat population.
‘Women with advanced ovarian cancer need and deserve new treatment options to improve outcomes, and [this] recommendation is hopeful news for eligible patients in Europe.’
The European Commission will now review the positive opinion and Pharma& anticipates an approval decision in the coming months.
Elmar Zagler, founder and managing director, Pharma&, said: ‘Accessing effective medicines is the primary goal for both healthcare providers and patients, and it can be devastating when these medicines are no longer available.
‘Over the last five years, Pharma& has established itself as an agile, fully integrated global company that aspires to breathe new life into proven medicines like rucaparib.’
Earlier this year, rucaparib was found to improve progression-free survival in metastatic, castration-resistant prostate cancer with BRAC alteration compared to usual care.
30th January 2023
The manufacturer Regeneron described how data from a study in The Lancet, showed that Dupixent treatment plus topical corticosteroids (hydrocortisone 1%) was effective in children under 6 years of age.
The Lancet trial enrolled and randomised 162 children aged 6 months to 6 years, to the drug or placebo. In the trial, participants were included if they had an Investigator’s Global Assessment (IGA) score of 3 – 4, which represents moderate to severe disease.
The drug was given at a dose based on the bodyweight. For instance, if > 5 kg but < 15 mg, a dose of 200 mg was given once every 4 weeks and for children weighing > 15 kg but < 30 kg, a dose of 300 mg was given. The primary endpoint was set as the proportion of participants with an IGA score of 0 or 1 (i.e., clear or almost clear) skin after 16 weeks.
At week 16, there was a 24% difference in IGA scores between Dupixent and placebo and which was statistically significant (p < 0.0001). The study also observed that the drug’s safety profile was generally in line with what might be expected.
The press release describes how in practice, 85 to 90% of children will develop atopic eczema before the age of 5 years and in Europe alone, it is estimated that up to 80,000 children aged between 6 months and 5 years have uncontrolled severe atopic dermatitis and might therefore benefit from a trial of systemic therapy.
According to the EMA, Dupixent will now be indicated for the treatment of severe atopic dermatitis in children 6 months to 11 years and who are candidates for systemic therapy.
The drug was approved for the same paediatric population by the FDA in June 2022.
5th January 2023
According to AstraZeneca, the Committee for Medicinal Products for Human Use (CHMP), has adopted a positive opinion recommending a change to the marketing authorisation for Enhertu.
The drug can now be used as monotherapy for the treatment of adult patients with unresectable or metastatic HER2‑low breast cancer who have received prior chemotherapy in the metastatic setting or developed disease recurrence during or within six months of completing adjuvant chemotherapy.
The World Health Organization estimates that in 2020, there were 2.3 million women diagnosed with breast cancer and which led to 685 000 deaths. In Europe alone in 2020, there were 531,086 cases of breast cancer that resulted in 141,765 deaths.
The human epidermal growth factor receptor 2 (HER-2) is a receptor tyrosine-protein kinase normally involved in the proliferation and division of breast cells and HER2-positive breast cancers are an aggressive type of breast cancer that tend to grow faster and are more likely to spread.
It is known that around 50% of all primary breast cancers show a low-level expression of HER2 (HER2-low), defined as immunohistochemically 1+ or 2+ and lack of HER2 gene amplification measured by in situ hybridisation. Moreover, this low HER2 expression is a promising new target for antibody-drug conjugates and Enhertu (which contains trastuzumab deruxtecan) is one such specifically engineered HER2-directed antibody drug conjugate.
Enhertu clinical efficacy
CHMP based its decision of data from the DESTINY-BREAST04 trial which compared previously treated patients with HER2-low (immunohistochemistry (IHC) 1+ or IHC 2+/in-situ hybridisation (ISH)-negative) unresectable and/or metastatic breast cancer with hormone receptor (HR) positive or HR-negative disease against standard of care physician’s choice of chemotherapy.
The study found that Enhertu demonstrated a 49% reduction in the risk of disease progression or death versus physician’s choice of chemotherapy in patients with HER2-low metastatic breast cancer with HR-positive disease (PFS hazard ratio, HR = 0.51, 95% CI 0.40 – 0.64, p < 0.001). In addition, the median overall survival in the hormone receptor–positive cohort was 23.9 months in the Enhertu group and 17.5 months in the physician’s choice group (HR for death = 0.64, 95% CI, 0.48 to 0.86, p = 0.003).
The safety profile of Enhertu was consistent with previous clinical trials with the most common Grade 3 or higher treatment-emergent adverse events were neutropenia (13.7%), anaemia (8.1%), fatigue (7.5%), leukopenia (6.5%), thrombocytopenia (5.1%) and nausea (4.6%).
A summary of the CHMP provides details on the full indications for Enhertu.
22nd July 2022
Teclistamab is an off-the-shelf, T-cell redirecting bispecific antibody. It targets both B-cell maturation antigen, a marker found on multiple myeloma cells, and CD3, on T-cells.
It is recommended as monotherapy for adult patients with relapsed and refractory multiple myeloma (RRMM), who have received at least three prior therapies, including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 antibody and have demonstrated disease progression on the last therapy.
In December 2021, the EMA granted accelerated assessment for teclistamab. Accelerated assessment reduces the timeframe for the CHMP to review a marketing authorisation application and is granted when a medicinal product is of major interest for public health and therapeutic innovation.
The CHMP recommendation is based on positive results from the multicohort, open-label, Phase I/II MajesTEC-1 study (NCT03145181 and NCT04557098), evaluating the safety and efficacy of teclistamab in adults with RRMM.
The latest findings from the study were presented at the American Society of Clinical Oncology (ASCO) 2022 Annual Meeting and published in The New England Journal of Medicine.
Teclistamab resulted in deep and durable responses in patients with triple-class exposed multiple myeloma (n=165). With a median follow-up of approximately 14 months (14.1), the overall response rate was 63% (95% CI: 55.2–70.4), with 39.4% having a complete response (CR) or better. Almost half (46%) of patients who achieved a CR or better were minimal residual disease-negative.
Adverse events (AEs) were consistent with this patient population and toxicities consistent with T-cell redirection were mostly Grade 1/2. The most common AEs were cytokine release syndrome and neutropenia. Infections were frequent (76.4%; 44.8% Grade 3 or 4). The overall incidence of neurotoxic events was low and five patients had immune effector cell-associated neurotoxicity syndrome. There were five treatment-related deaths, and dose reductions and discontinuations due to AEs were infrequent.
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