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Press Releases

Take a look at a selection of our recent media coverage:

Empagliflozin receives EU approval for chronic kidney disease

27th July 2023

Empagliflozin has become the first SGLT2 inhibitor to be approved by the European Medicines Agency (EMA) for the treatment of adult patients with chronic kidney disease (CKD), Boehringer Ingelheim and Eli Lilly and Company have announced.

This EMA approval represents a major advance in the standard of care for the estimated 47 million patients living with the disease in Europe. CKD, characterised by a reduction in the glomerular filtration rate, is often progressive, and the presence of albuminuria is a key risk factor for the subsequent development of kidney failure. Hence, any treatment that slows disease progression offers a clinical benefit to patients.

In June 2023, the Committee for Medicinal Products for Human Use adopted a positive opinion to recommend a change to the terms of the marketing authorisation for the medicinal product Jardiance (empagliflozin). This was based on the findings of the EMPA-KIDNEY trial, which were published in the New England Journal of Medicine earlier in the year.

With existing indications in type 2 diabetes and heart failure, empagliflozin could help manage the risks of cardio-renal-metabolic conditions.

Leonard Glass, senior vice president, diabetes and obesity global medical affairs at Lilly, said: ’CKD is closely linked to other cardio-renal-metabolic conditions such as type 2 diabetes and heart failure – thus an integrated approach is vital for optimised treatment of these interconnected conditions. We look forward to continuing conversations with other regulatory bodies worldwide so that empagliflozin can be made available for as many people living with these conditions as quickly as possible.’

Commenting on the approval, Daniel Gallego, president of European Kidney Patients’ Federation, added: ’We celebrate this significant milestone in the field of chronic kidney disease. CKD is a silent killer and prevention and early detection are crucial in the general population.

’This new treatment option has the potential to further improve the management of cardiorenal metabolic syndrome and renal disease, offering renewed hope and improved quality of life for countless individuals living with CKD worldwide.’

Empagliflozin and the EMPA-KIDNEY trial

The EMPA-KIDNEY trial enrolled patients with chronic kidney disease who had an estimated glomerular filtration rate (eGFR) of at least 20 but less than 45 ml per minute per 1.73 m2 of body-surface area. Alternatively, enrolled participants could have an eGFR of at least 45 but less than 90 with a urinary albumin-to-creatinine ratio of at least 200.

EMPA-KIDNEY showed that during a median of 2.0 years of follow-up, the use of empagliflozin was associated with a 28% lower risk of progression of kidney disease or death from cardiovascular causes, compared to the placebo (Hazard ratio, HR = 0.72, 95% CI 0.64 – 0.82, p < 0.001).

These findings were consistent among patients with or without diabetes and across subgroups defined according to eGFR ranges. In addition, the rate of hospitalisation from any cause was lower in the empagliflozin group (HR = 0.86, 95% CI 0.78 – 0.95, p = 0.003).

Kaftrio approval for cystic fibrosis extended by EMA from 6 years of age

17th January 2022

Kaftrio, which represents a triple therapy for patients with cystic fibrosis, can now be used in children from 6 years of age

Kaftrio has been granted an extension to its marketing authorisation by the European Medicines Agency (EMA) for children as young as age 6 who have cystic fibrosis (CF) and at least one F508del mutation in combination with ivacaftor.

Kaftrio is a combination treatment containing 75mg ivacaftor, 50mg tezacaftor and 100mg elexacaftor and is already indicated in a combination regimen with ivacaftor 150mg tablets for the treatment of CF in patients aged 12 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

CF is a rare, monogenic disease (i.e., caused by variation in a single gene) which is thought to affect at least 100 000 people worldwide. The condition is best described as a progressive, multi-organ disease that affects the lungs, liver, pancreas, GI tract, sinuses, sweat glands, reproductive tract and a reduced life expectancy. However, according to Cystic Fibrosis, the life expectancy of children born today is likely to surpass 50 years for the first time.

CF is caused by mutations in the genes responsible for encoding of the cystic fibrosis transmembrane conductance regulator (CFTR) protein. This defect results in a reduced chloride secretion and increased sodium absorption through epithelial sodium channels and removal of water from secretions, which therefore become abnormally viscous. Although more than 2000 gene variants of the disease have been discovered, the most predominant is the F508del mutation.

The three drugs present in Kaftrio work in combination. For example, ivacaftor functions as a potentiator of the CFTR protein for common gating mutations, allowing for an increase in chloride ion flow. Tezacaftor ensures correct folding and presentation of the mature CFTR protein to the cell surface, improving CFTR function for the F508del mutation.

Finally, elexacaftor is also helps to ensure correct folding of the CFTR protein but acts at an alternate binding site to tezacaftor on the CFTR protein. Overall, this triple combination increases the function of the F508del mutated CFTR protein at the cell surface resulting in increased chloride ion transport and ultimately help hydrate and clear mucus from the airways.

Clinical data already support the use of Kaftrio in children aged 12 years and over though a more recent study demonstrated the combination therapy was also effective in children from 6 years of age and enabled the EMA to extend its licensed usage.

In addition to approval from the EMA, the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has also confirmed the same license extension for Kaftrio.

RoActemra (tocilizumab) approved by EMA for use in severe COVID-19

9th December 2021

RoActemra is EMA-approved for severe COVID-19 in adults receiving systemic corticosteroids and supplemental oxygen or mechanical ventilation

RoActemra (tocilizumab) has received approval from the EMA for the treatment of adults with severe COVID-19 and who are in receipt of systemic treatment with corticosteroids and requiring supplemental oxygen or mechanical ventilation.

Until the COVID-19 approval, RoActemra was already approved for use in the management of inflammatory conditions including rheumatoid arthritis, systemic juvenile idiopathic arthritis, juvenile idiopathic polyarthritis, giant cell arteritis and cytokine release syndrome (CRS).

Tocilizumab is a monoclonal antibody that works by inhibiting the binding of interleukin-6 (IL-6) to its receptor and in doing so, Inhibits IL-6 signal transduction of inflammatory mediators in rheumatic diseases. However, emerging evidence has indicated a role for IL-6 signalling in patients hospitalised with severe COVID-19, especially those who are critically ill.

Clinical data

The approval for RoActemra was based on data from three phase III trials. In COVACTA, patients hospitalised with severe COVID-19 pneumonia were randomised, 2:1 to either a single intravenous infusion of tocilizumab (at a dose of 8mg per kilogram of body weight) or placebo. The primary outcome was clinical status at day 28 on an ordinal scale which ranged from 1 (discharged or ready for discharge) to 7 (death). In the published results of the trial, tocilizumab did not result in a significantly better clinical status or lower mortality than placebo at 28 days. 

In EMPACTA, hospitalised patients with COVID-19 pneumonia, not receiving mechanical ventilation, were randomised to receive standard care plus one or two doses of either tocilizumab (8 mg per kilogram of body weight intravenously) or placebo. The primary outcome was mechanical ventilation or death by day 28 and the results showed that although tocilizumab reduced the likelihood of progression to the composite outcome of mechanical ventilation or death, it did not improve survival.

In the REMDACTA study, patients were again randomised (2:1) to tocilizumab plus remdesivir or placebo plus remdesivir. The primary outcome was the time from randomisation to hospital discharge or “ready for discharge” (category 1 on a 7-category ordinal scale of clinical status) to day 28. As with the other trials, when published, the authors concluded that ‘tocilizumab plus remdesivir did not shorten time to hospital discharge or “ready for discharge” to day 28 compared with placebo plus remdesivir.’

However, despite these somewhat negative findings, a systemic review assessing the efficacy of IL-6 antagonists in patients hospitalised for COVID-19, in nearly 11,000 patients, concluded that ‘administration of IL-6 antagonists, compared with usual care or placebo, was associated with lower 28-day all-cause mortality.’

Details of the revised indicated use in COVID-19 can be found in the summary of product characteristics.

Byannli approved by EMA for twice-yearly dosing in schizophrenia

24th November 2021

Byannli (paliperidone palmitate) is the first long-acting injectable treatment approved by the EMA for a twice-yearly dosing regime.

Byannli (paliperidone palmitate) is the first long-acting injectable agent to be approved by the European Medicine Agency (EMA) for the maintenance treatment of schizophrenia in adult patients, deemed clinically stable and already prescribed injectable paliperidone palmitate given either monthly or 3‑monthly.

Schizophrenia is a chronic and severe mental illness that affects around 20 million people worldwide. The illness is characterised by distorted thinking, perception, emotions, language, sense of self and behaviour. Among sufferers, common experiences will include hallucinations, such as seeing or hearing things which are invisible or inaudible to others together with fixed and false beliefs, i.e., delusions. Typically, patients experience an initial period in which some deterioration in personal functioning and emergence of negative symptoms occurs, followed by an acute phase with both positive and negative symptoms that can interfere with daily functioning.

Treatment is aimed at reducing the acute phase symptoms and trying to return patients to their normal state. However, for many individuals, non-adherence to treatment is a major barrier to successful disease control with identified drivers of non-adherence including lack of insight, medication beliefs and substance abuse. These factors combine to increase the risk of relapse and in fact, treatment relapse with a gap of between 11 and 30 days has been associated with a nearly three-fold increased risk of hospitalisation. Consequently, most patients with schizophrenia require maintenance treatment.

Several anti-psychotic agents are available, one of which is Byannli (paliperidone palmitate), which is available as both an oral an injectable form and has usually been administered either monthly or three-monthly. A clear advantage of long-acting over oral therapy, particularly among schizophrenic patients, is that blood levels of the drug remain stable over time, which reduces the risk of a relapse.

Clinical efficacy

The EMA approval was based on the results of the Route 6 study with paliperidone palmitate in 702 adults (ages 18–70) with schizophrenia. The purpose of the study was to show that the efficacy of 6-monthly, PP6M (700 or 1000mg) of Byannli were non-inferior to a three-monthly (PP3M) regime preventing relapse in participants with schizophrenia who were previously stabilised on corresponding doses of the drug given either monthly (100 or 150mg) or three-monthly (350 or 525mg). In the trial all stabilised adult patients (n=702) were randomised in a 2:1 ratio to receive a six-monthly (n = 478) or three-monthly (n = 224) dose of the drug. The primary outcome was the time to first relapse which was defined as psychiatric hospitalisation, increase in positive and negative syndrome scale (PANSS) total score, increase in individual PANSS item scores, violent behaviour resulting in self-injury or suicidal/homicidal ideation.

The results showed non-inferiority of PP6M compared with PP3M on the primary endpoint of time to first relapse at the end of the 12-month period. Results showed that 92.5% of patients treated with PP6M and 95.1% treated with PP3M were relapse-free at 12 months. In addition, the safety profile observed for PP6M was consistent with previous studies with no new safety signals emerging.

The EMA approval follows a similar approval by the FDA in September 2021.

Source. Janssen press release November 2021

Risankizumab approved by EMA for active psoriatic arthritis

22nd November 2021

Risankizumab can now be used by patients with active psoriatic arthritis after an inadequate response or intolerance to one or more DMARDs

The European Medicines Agency (EMA) has approved the use of risankizumab (brand name Skyrizi) for the treatment of patients with active psoriatic arthritis (PsA) who have failed to adequately response to one or more disease modifying anti-rheumatic drugs (DMARDs) or who are intolerant to DMARDs.

Psoriatic arthritis is an inflammatory arthritis affecting the joints and connective tissue which is associated with psoriasis and which is present on the skin or nails. While the prevalence of psoriasis in the general population is low, at around 3%, at least 20% of psoriasis patients have PsA which is a progressive disease that ranges from mild synovitis to severe erosive arthropathy. Sufferers of PsA experience joint inflammation which causes swelling and pain and which has a negative impact on their quality of life.

The treatment of PsA starts with non-steroidal anti-inflammatories and as the disease progresses, escalates to oral corticosteroids, DMARDs and finally biologic agents. There are currently several biologics approved for the management of PsA including ixekizumab and guselkumab. Skyrizi was previously approved by the EMA in 2019 for the treatment of plaque psoriasis but that has been extended to include PsA.

Clinical efficacy

The EMA approval of Skyrizi (risankizumab) was based the findings of two Phase III clinical trials, KEEPsAKE-1 and KEEPsAKE-2. Both trials were placebo-controlled trials in patients with moderate to severe PsA although KEEPsAKE-1 included patients with an inadequate respond to one or more DMARDs whereas KEEPsAKE-2 recruited those with an inadequate response to other biologicals. In both trials patients received skyrizi at subcutaneous dose of 150 mg and the primary outcome for both studies was the ‘percentage of Participants Achieving at least 20% Improvement in American College of Rheumatology (ACR20)‘.

The ACR20 is a composite measure defined as both improvement of 20% in the number of tender and number of swollen joints, and a 20% improvement in three of the following five criteria: patient global assessment, physician global assessment, functional ability measure [most often Health Assessment Questionnaire (HAQ)], visual analog pain scale, and erythrocyte sedimentation rate or C-reactive protein (CRP).

In both trials, the ACR20 response was set as the primary endpoint and assessed after 24 weeks. Secondary endpoints included the Health assessment questionnaire disability index (HAD-DI), which represents a measure of physical function and the proportion of patients achieving minimal disease activity (MDA).

In KEEPsAKE-1 (KS1) and KEEPsAKE-2 (KS2), 57.3% and 51.3% of patients respectively, given risankizumab achieved the primary endpoint compared to 33.5% and 26.5% receiving placebo (p<0.001).

Similarly, improvements in HAQ-DI of -0.31 (KSI) and -0.22 (KS2) compared with -0.11 and -0.05, in the respective placebo groups was seen at week 24 (p<0.001). Finally, 25% (KS1) and 25.6% (KS2) of Skyrizi patients achieved MDA, compared to 10.2% (KS1) and 11.4% (KS2) of those on placebo (p<0.001).

In terms of safety, serious adverse events occurred in 2.5% (KS1) and 4% (KS2) of patients given Skyrizi although this was comparable to the placebo rate (3.7% and 5.5%, KSI and KS2).

According to the EMA approval, Skyrizi can be used either alone or in combination with methotrexate.

Source. Abbvie press release. 17th November 2021