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Take a look at a selection of our recent media coverage:
26th July 2024
The interleukin (IL)-23 inhibitor risankizumab (brand name Skyrizi) has been granted marketing authorisation by the European Commission for ulcerative colitis.
The indication covers the treatment of adult patients with moderately to severely active ulcerative colitis who have had an inadequate response to, lost response to, or were intolerant to conventional therapy or a biologic therapy.
This marks the fourth approved indication for risankizumab after plaque psoriasis, psoriatic arthritis and Crohn’s disease.
Risankizumab selectively blocks IL-23 by binding to its p19 subunit. The recommended induction dose is 1,200 mg administered by intravenous infusion at Week 0, Week 4 and Week 8. Starting at Week 12 and every eight weeks thereafter, a recommended maintenance dose of either 180 mg or 360 mg administered by subcutaneous injection should be based on individual patient presentation.
‘Ulcerative colitis is a chronic, unpredictable and sometimes debilitating disease, and people living with the condition need sustained symptom relief,’ said Dr Edouard Louis, professor and head of gastroenterology at Liège University Hospital and dean of faculty at Liège University in Belgium. ‘This approval introduces a new treatment option to help patients with ulcerative colitis reach their long-term treatment goals.’
The EC approval of risankizumab is based on the results of two phase 3 clinical trials, INSPIRE and COMMAND, in which the primary endpoint was clinical remission. Secondary endpoints included mucosal healing and histologic endoscopic mucosal healing (HEMH).
In the INSPIRE induction trial, a significantly higher proportion of patients treated with risankizumab 1,200 mg IV achieved the primary endpoint of clinical remission (per Adapted Mayo Score) at Week 12 than patients receiving placebo (20% vs 6%; p<.00001).
Mucosal healing (defined as ES ≤1 without friability) was observed at Week 12 in 37% of patients treated with risankizumab 1,200 mg IV compared to 12% of those receiving placebo (p<.00001). In patients without previous biologic or JAK inhibitor failure, 48% of patients treated with risankizumab 1,200 mg IV achieved mucosal healing at Week 12 versus 14% of those receiving placebo.
Some 24% of patients treated with risankizumab 1,200 mg IV achieved HEMH (defined as Geboes score ≤3.1 and ES ≤1 without friability) at week 12 versus 8% of those receiving placebo (p<.00001).
In the COMMAND maintenance trial, a significantly higher proportion of patients who received risankizumab 180 mg or 360 mg SC achieved clinical remission at Week 52 than patients in the induction-only control group: 40% and 38%, respectively, versus 25% (p≤.01).
Some 51% of patients treated with risankizumab 180 mg and 48% of patients treated with 360 mg achieved mucosal healing at Week 52 versus 32% of patients in the induction-only control group (p<.001). In patients without previous biologic or JAK inhibitor failure, 60% of patients who received risankizumab 180 mg and 76% who received 360 mg achieved mucosal healing versus 36% of patients in the induction-only control group.
Some 43% of patients treated with risankizumab 180 mg and 42% receiving 360 mg achieved HEMH at Week 52 versus 23% in those treated with the induction dose only (p≤0.01).
Professor Louis, who was also a trial investigator, added: ‘Patients treated with Skyrizi in the INSPIRE and COMMAND clinical trials experienced significant improvements in clinical remission and mucosal healing. These are important findings as mucosal healing goes beyond symptom management to restoration of the intestinal lining and is associated with improved long-term outcomes.’
The safety profile of risankizumab in both trials was consistent with the safety profile observed in previous trials across other indications, with no new safety risks observed. The most common adverse events observed were Covid-19, anaemia, nasopharyngitis and arthralgia.
19th July 2024
The AKT inhibitor capivasertib (brand name Truqap) has been approved by the Medicines and Healthcare products Regulatory Agency (MHRA) for treating eligible patients with advanced breast cancer.
Capivasertib is indicated in combination with the hormone therapy fulvestrant for treating advanced hormone receptor (HR) positive, human epidermal growth factor receptor 2 (HER2) negative locally advanced breast cancer with specific biomarker alterations (PIK3CA, AKT1 or PTEN) following recurrence or progression on or after an endocrine-based regimen.
This follows the marketing authorisation of capivasertib by the European Commission (EC) in June 2024 for the same indication.
The first-in-class drug is taken orally at a starting dose of 400 mg twice a day for four days followed by three days of rest, then repeated.
The MHRA and EC approvals were based on the results of the phase 3, randomised, double-blind CAPItello-291 trial, which included 708 patients with locally advanced or metastatic HR-positive, HER2-negative breast cancer. Of these, 289 patients (40.8%) had AKT pathway alterations.
Patients were randomised to receive either capivasertib or a placebo, both in combination with fulvestrant. The dual primary endpoint was investigator-assessed progression-free survival assessed both in the overall population and among patients with AKT pathway–altered (PIK3CA, AKT1, or PTEN) tumours.
In the overall population, the median progression-free survival was 7.2 months in the capivasertib–fulvestrant group, as compared with 3.6 months in the placebo–fulvestrant group (HR 0.60; 95% CI, 0.51 to 0.71; P<0.001).
In the AKT pathway–altered population, the median progression-free survival was 7.3 months in the capivasertib–fulvestrant group, as compared with 3.1 months in the placebo–fulvestrant group (HR 0.50; 95% CI, 0.38 to 0.65; P<0.001).
Potential side effects of capivasertib included high blood sugar, diarrhoea, rash and other skin drug reactions, urinary tract infection, low level of haemoglobin in blood, loss of appetite, nausea, vomiting, mouth sores or ulcers with gum inflammation, itching, and tiredness.
The most frequent adverse events of grade 3 or higher in patients receiving capivasertib–fulvestrant were rash (12.1% vs in 0.3% of those receiving placebo–fulvestrant) and diarrhoea (9.3% vs 0.3%).
Adverse events leading to discontinuation were reported in 13.0% of the patients receiving capivasertib and in 2.3% of those receiving placebo.
Professor Nicholas Turner, professor of molecular oncology at The Institute of Cancer Research, London, and consultant medical oncologist at The Royal Marsden NHS Foundation Trust, who led the CAPItello-291 trial, said the MHRA approval is ‘wonderful development in the treatment of the most common type of advanced breast cancer’.
He added: ‘Around half of patients with this kind of breast cancer have mutations in one or more of the genes PIK3CA, AKT1 or PTEN, and for these patients, capivasertib provides an exciting, new targeted treatment which can keep their cancer from progressing for longer.
‘We hope NICE will recommend the use of capivasertib in combination with fulvestrant for patients on the NHS.’
Speaking about the EC marketing authorisation of this AKT inhibitor, Dr Mafalda Oliveira, senior clinical investigator of the Vall d’Hebron Institute of Oncology’s Breast Cancer Group in Barcelona, Spain, said: ‘Patients with advanced [HR]-positive breast cancer typically experience tumour progression or resistance with widely used endocrine-based treatment regimens, and there is an urgent need to provide them more time with their disease under control.
‘[This] approval is welcome news for approximately half of [HR]-positive breast cancer patients in Europe who have tumours with these biomarkers, and it is important for clinicians to test and identify eligible patients who may be able to benefit from this combination.’
4th July 2024
The monoclonal antibody dupilumab (brand name Dupixent) has been approved by the European Commission for eligible adults with uncontrolled chronic obstructive pulmonary disease (COPD) with raised blood eosinophils.
The first-ever targeted therapy to be approved for patients with COPD, dupilumab is now indicated as an add-on maintenance treatment for patients already on a combination of an inhaled corticosteroid (ICS), a long-acting beta-2 agonist (LABA) and a long-acting muscarinic antagonist (LAMA), or on a combination of a LABA and a LAMA if ICS is not appropriate.
Dupilumab targets interleukin-4 and interleukin-13 pathways – the key drivers of type 2 inflammation – and is the first biologic approved for COPD, as well as being the first new treatment for the disease approved in Europe for over a decade.
Sanofi and Regeneron estimate that around 220,000 patients with COPD in the EU could be eligible for treatment with dupilumab.
The approval is based on results from the phase 3 BOREAS and NOTUS trials, which evaluated the efficacy and safety of dupilumab in adults who had uncontrolled COPD with blood eosinophils ≥300 cells per μL.
The primary endpoint in both trials was the annualised rate of moderate or severe exacerbations.
Patients were on background maximal standard-of-care inhaled therapy, with nearly all on triple therapy, and were randomised to receive subcutaneous dupilumab (300 mg) or placebo every two weeks.
Dupilumab patients in BOREAS (n=468) experienced a 30% reduction in the annualised rate of moderate or severe COPD exacerbations over 52 weeks compared to placebo (BOREAS n=471).
In NOTUS (n=470) this was a 34% reduction compared to placebo (NOTUS n=465).
In terms of the secondary and other endpoints, there were improvements in lung function (prebronchodilator FEV1) from baseline by 160 mL and 139 mL at 12 weeks compared to 77 mL and 57 mL in the placebo group. These were observed as early as Week 2 and 4 and were sustained at Week 52 in both trials.
The researchers also noted improvements in health-related quality of life, which was statistically significant in BOREAS and nominally significant in NOTUS, as assessed by the St. George’s Respiratory Questionnaire.
Safety results in both studies were found to largely be consistent with the known safety profile of dupilumab in its approved indications.
Speaking of dupilumab’s potential to ‘redefine the treatment landscape’ of COPD, George D. Yancopoulos, board co-chair, president and chief scientific officer at Regeneron, said: ‘The approval of Dupixent for COPD is a long-awaited turning point for those who struggle to breathe even through the simplest of tasks, while also facing the risk of hospitalisation, irreversible health decline and feelings of hopelessness.’
Paul Hudson, chief executive officer at Sanofi, added: ‘Patients with uncontrolled COPD have been waiting for a new treatment approach for many years, so we are thrilled to bring to market the first biologic to target an underlying cause of this devastating disease to reduce COPD exacerbations and improve lung function.’
This latest approval means dupilumab is indicated for certain patients with six conditions in the EU: moderate-to-severe atopic dermatitis, severe asthma, chronic rhinosinusitis with nasal polyposis, moderate-to-severe prurigo nodularis, eosinophilic oesophagitis, and now COPD.
25th April 2024
The monoclonal antibody tislelizumab has been approved by the European Commission (EC) for use in patients with treatment-naive and relapsed non-small cell lung cancer (NSCLC), its manufacturer BeiGene has announced.
This follows its positive opinion from the European Medicines Agency’s Committee for Medicinal Products for Human Use in March 2024.
The approval of tislelizumab (under the brand name Tizveni, but also known as Tevimbra) is across three indications, including first- and second-line use, which are:
Tislelizumab, which is a PD-1 inhibitor, is also approved in the European Union for the treatment of locally advanced or metastatic oesophageal squamous cell carcinoma (OSCC) after prior chemotherapy.
The NSCLC indications were approved under the brand name Tizveni. However, BeiGene plans to combine the NSCLC indications with the second-line OSCC indication under the brand name Tevimbra, which will launch in the first EU countries later in 2024.
Commenting on the approval, Dr Luis Paz-Ares, head of the medical oncology service at University Hospital October 12 in Madrid, Spain, said: ‘Non-small cell lung cancer remains one of the most common and deadly cancers in Europe, with 50% of patients diagnosed already progressed to advanced stages, making it difficult to treat.
‘Across three phase 3 studies, tislelizumab has been shown to improve outcomes for patients with certain types of NSCLC, providing a new option for those facing the disease.’
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: RATIONALE 307 and RATIONALE 304, which both looked at first-line treatment in combination with chemotherapy for various types of NSCLC, and RATIONALE 303, which looked at tislelizumab as second- and third-line treatment compared with docetaxel.
23rd April 2024
A type II variation of ciltacabtagene autoleucel (cilta-cel; brand name Carvykti) has been approved by the European Commission (EC) 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, have demonstrated disease progression on the last therapy, and are refractory to lenalidomide.
Cilta-cel is a chimeric antigen receptor (CAR) T-cell therapy directed against B-cell maturation antigen (BCMA) and is the first BCMA CAR-T therapy approved in Europe for the treatment of eligible patients as early as first relapse.
The approval follows a positive opinion from the European Medicines Agency’s Committee for Medicinal Products for Human Use in March 2024.
Professor Jesús San Miguel, director of clinical & translational medicine at the University of Navarra in Pamplona, Spain, said: ‘Patients with lenalidomide-refractory multiple myeloma tend to experience early resistance to standard treatments and their disease worsens exponentially with each additional line of therapy.
‘A single infusion of cilta-cel has been shown to significantly lower the risk of progression or death compared to current treatment options, as early as after first relapse.’
The EC approval was based on the results of a phase 3 CARTITUDE-4 clinical trial evaluating the efficacy and safety of cilta-cel in patients with relapsed and lenalidomide-refractory multiple myeloma.
Data from the study were previously published in the New England Journal of Medicine.
The primary endpoint was progression-free survival (PFS), 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.
Patients were randomised to receive either a sequence of apheresis, bridging therapy, lymphodepletion and cilta-cel (n=208) or standard of care, which included daratumumab, pomalidomide and dexamethasone (DPd) or pomalidomide, bortezomib and dexamethasone (PVd) (n=211).
At a median follow-up of 15.9 months, a single infusion of cilta-cel 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).
The median duration of PFS was not reached in the cilta-cel arm and was 11.8 months in the standard care arm (95% CI, 10-14).
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).
22nd April 2024
Bimekizumab (brand name Bimzelx) has been granted marketing authorisation by the European Commission (EC) for active moderate-to-severe hidradenitis suppurativa (HS), its manufacturer UCB has announced.
Now indicated for adults with an inadequate response to conventional systemic HS therapy, the humanised monoclonal IgG1 antibody is designed to selectively inhibit the two cytokines interleukin 17A (IL-17A) and interleukin 17F (IL-17F).
This approval follows a positive opinion from the European Medicines Agency’s Committee for Medicinal Products for Human Use in March 2024 and makes bimekizumab the first biologic for moderate-to-severe HS to target IL-17F in addition to IL-17A.
It also marks the fourth marketing authorisation for the drug, adding to the existing indications in moderate-to-severe plaque psoriasis, active psoriatic arthritis and active axial spondyloarthritis.
Professor Christos Zouboulis, director of the departments of dermatology, venereology, allergology and immunology at Städtisches Klinikum Dessau, Brandenburg Medical School, Germany, and president of the European Hidradenitis Suppurativa Foundation, said: ‘The European Commission’s approval of bimekizumab marks a significant milestone for the EU hidradenitis suppurativa community, particularly considering the limited treatment options currently available.
‘As a community, we strive to improve the management of hidradenitis suppurativa. Bimekizumab offers a promising new therapeutic option for moderate to severe disease, supported by Phase 3 evidence that demonstrated clinically meaningful and sustained responses over 48 weeks.’
Indeed, the EC approval was based on results from the BE HEARD I and BE HEARD II studies, which evaluated the efficacy and safety of bimekizumab in the treatment of moderate-to-severe HS.
The multicentre, randomised, double-blind, placebo-controlled phase 3 trials had a combined enrolment of 1,014 adult patients with a diagnosis of moderate to severe HS.
The primary endpoint in both studies was HiSCR50 at Week 16, with HiSCR75 at Week 16 as a key secondary endpoint. These are defined as at least either a 50 or 75% reduction from baseline in the total abscess and inflammatory nodule count, with no increase from baseline in abscess or draining tunnel count.
A significantly higher proportion of patients treated with bimekizumab versus placebo achieved a 50% or greater improvement in HS signs and symptoms at Week 16, as measured by HiSCR50.
Bimekizumab treatment also resulted in clinically meaningful improvements in the ranked key secondary endpoint, HiSCR75 versus placebo at Week 16.
Responses were maintained to Week 48, and the safety profile of bimekizumab was consistent with safety data seen in previous trials with no new observed safety signals.
27th February 2024
Etrasimod (brand name Velsipity) has been granted marketing authorisation by the European Commission (EC) for use in eligible patients with ulcerative colitis (UC), its manufacturer Pfizer has announced.
It is approved to treat patients aged 16 years and older with moderately to severely active UC who have had an inadequate response, lost response, or were intolerant to either conventional therapy or a biological agent.
This means etrasimod has received the first global approval of an oral advanced UC therapy for use in older adolescents.
The marketing authorisation is valid in all 27 EU member states as well as Iceland, Liechtenstein and Norway.
The approval of etrasimod follows a positive opinion from the European Medicines Agency’s Committee for Medicinal Products for Human Use in December 2023.
Commenting on the approval, Dr Séverine Vermeire, professor of medicine at KU Leuven in Belgium, said: ‘For the 2.6 million people in Europe living with UC, the unpredictable physical, mental, and emotional impacts of the condition can be debilitating. They may cycle through several different conventional treatment options to find relief for their symptoms.
‘The approval of Velsipity helps bridge the gap for those with moderately to severely active UC who need an effective advanced treatment but may be apprehensive about using injectable therapies like biologics.’
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.
Etrasimod can help eligible patients who are struggling to achieve remission with conventional therapies and has a favourable benefit-risk profile, Pfizer said.
The EC marketing authorisation of etrasimod was based on results from the phase 3 ELEVATE UC 52 and ELEVATE UC 12 trials, for which Dr Vermeire was an investigator.
These trials evaluated the safety and efficacy of etrasimod 2 mg once daily on clinical remission in UC 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.
18th January 2024
Subcutaneous atezolizumab (brand name Tecentriq SC) has been granted marketing authorisation by the European Commission (EC) for multiple cancer types, its manufacturer Roche has announced.
It is the first PD-(L)1 cancer immunotherapy for subcutaneous (SC) injection available in the European Union and offers the potential for a faster and more convenient alternative to intravenous (IV) infusion of atezolizumab.
The SC injection is administered under the skin and takes approximately seven minutes to complete, with most injections taking between four and eight minutes. This is 75% quicker than the 30-60 minute duration of the IV infusion.
The EC approval covers all indications in which the IV form of atezolizumab has previously been approved, including certain types of lung, liver, bladder and breast cancers.
In August 2023, the NHS in England became the first health system in the world to roll out SC atezolizumab after its approval by the Medicines and Healthcare products Regulatory Agency (MHRA).
The EC and MHRA approvals were based on pivotal data from the Phase IB/III IMscin001 study, which showed comparable levels of atezolizumab in the blood, when administered subcutaneously, and a safety and efficacy profile consistent with the IV formulation.
Some 90% of healthcare professionals who were surveyed as part of the study agreed that the SC formulation is easy to administer and 75% said it could save time for healthcare teams compared with the IV formulation.
While the IMscin001 trial was conducted within the hospital setting, SC atezolizumab may be administered by a healthcare professional outside of the hospital in a community care setting or at a patient’s home, depending on national regulations and health systems.
Commenting on the approval, Dr Enriqueta Felip, head of the Thoracic Cancer Unit of Vall d’Hebron Hospital in Barcelona, Spain, said: ‘Ensuring the best possible quality of life is crucial for people living with cancer.
‘The availability of a subcutaneous cancer immunotherapy option that can minimise the time receiving treatment and even allow for treatment outside of a hospital will undoubtedly make a significant difference to patients and their loved ones.’
Levi Garraway, Roche’s chief medical officer and head of global product development, added: ‘Giving Tecentriq subcutaneously provides more flexibility to patients, while also helping to free up resources in constrained healthcare systems.’
Roche is working with national health systems in Europe to ensure patients can access SC atezolizumab as quickly as possible, and the company is also in discussion with several providers in Europe to include the drug in cancer homecare initiatives where possible.
9th January 2024
The oral poly ADP-ribose polymerase (PARP) inhibitor talazoparib (brand name Talzenna) has been approved by the European Commission (EC) in combination with the androgen receptor blocker enzalutamide (brand name Xtandi) in eligible patients with prostate cancer, its manufacturer Pfizer has announced.
Indicated for the treatment of adult patients with metastatic castration-resistant prostate cancer (mCRPC) in whom chemotherapy is not clinically indicated. It is now the first and only PARP inhibitor licensed in the European Union for use with enzalutamide for patients with mCRPC, with or without gene mutations.
Preclinical studies have demonstrated that talazoparib blocks PARP enzyme activity and traps PARP at the site of DNA damage, leading to decreased cancer cell growth and cancer cell death.
Robert Jones, professor of clinical cancer research at the University of Glasgow, Scotland, said: ‘New treatment options are needed to increase the proportion of patients with metastatic castration-resistant prostate cancer who can benefit from current anticancer medicines that keep the disease under control for longer.
‘The European Commission’s approval of talazoparib in combination with enzalutamide offers a meaningful advancement for the treatment of patients with metastatic castration-resistant prostate cancer, the most advanced and aggressive stage of the disease.’
Erik Briers, vice chairman of Europa UOMO – a European advocacy movement for people with prostate cancer – added: ‘After years of fighting prostate cancer, it can be devastating for a patient to learn that their cancer has stopped responding to testosterone-lowering treatments. At this stage of the disease, the prognosis is generally poor.
‘Patients urgently need new treatment options and Talzenna in combination with Xtandi can bring new hope to these patients.’
The EC approval is based on data from the Phase 3 TALAPRO-2 trial, a multicentre, randomised, double-blind, placebo-controlled study, which compared the efficacy and safety of talazoparib plus enzalutamide versus enzalutamide alone in patients with asymptomatic or mildly symptomatic mCRPC receiving ongoing androgen deprivation therapy.
Patients were prospectively assessed for homologous recombination repair (HRR) gene alterations in tumour tissue and randomly assigned (1:1) to talazoparib 0.5 mg or placebo, plus enzalutamide 160 mg, administered orally once daily.
Randomisation was stratified by HRR gene alteration status (deficient vs non-deficient or unknown) and previous treatment with life-prolonging therapy (docetaxel or abiraterone, or both: yes vs no) in the castration-sensitive setting.
The results from TALAPRO-2 Cohort 1, published in The Lancet, showed that treatment with talazoparib plus enzalutamide reduced the risk of disease progression or death by 37% versus placebo plus enzalutamide (Hazard Ratio [HR]: 0.63; 95% Confidence Interval [CI], 0.51–0.78; P< 0.0001), meeting the study’s primary endpoint of improving radiographic progression-free survival (rPFS).
A trend in overall survival (OS), a key secondary endpoint, favouring talazoparib plus enzalutamide was also observed, though these data are immature.
The safety of talazoparib plus enzalutamide in the TALAPRO-2 trial was generally consistent with the known safety profile of each medicine.
The researchers concluded that talazoparib plus enzalutamide resulted in clinically meaningful and statistically significant improvement in rPFS versus standard of care enzalutamide as first-line treatment for patients with mCRPC.
Talazoparib is also approved in over 70 countries, including in the EU, as a once-daily monotherapy for the treatment of adult patients with deleterious or suspected deleterious germline breast cancer susceptibility gene (BRCA)-mutated (gBRCAm) human epidermal growth factor receptor 2 (HER2)-negative locally advanced or metastatic breast cancer.
4th January 2024
Rezafungin (brand name Rezzayo) has been approved by the European Commission for the treatment of invasive candidiasis in adults, its manufacturer Mundipharma has announced.
An antimycotic, rezafungin selectively inhibits the fungal enzyme 1,3-β-D-glucan synthase, inhibiting formation of 1,3-β-D-glucan, an essential component of the fungal cell wall. This, in turn, results in rapid and concentration-dependent fungicidal activity in Candida species.
The first new treatment option in over 15 years for patients with invasive candidiasis, rezafungin has been granted orphan drug designation and will be available as a 200 mg powder for concentrate for solution for infusion.
Professor Oliver Cornely, head of the European Excellence Centre for Medical Mycology at the University Hospital Cologne, Germany, said: ‘There has been a significant global unmet need for treating people with invasive candidiasis. [This] announcement by the European Commission marks an important moment that could enable the healthcare professional community to manage invasive candidiasis patients in a different way using a new treatment option.’
Despite a number of available treatments, the mortality rate for patients with invasive candidiasis is as high as 40% or more.
The approval follows the positive opinion of the Committee for Medicinal Products for Human Use in October 2023 and is based on results from the pivotal ReSTORE Phase III clinical trial, for which Professor Cornely was a data review committee member.
This multicentre, double-blind, double-dummy, randomised trial compared the efficacy and safety of intravenous rezafungin compared with intravenous caspofungin – the current standard of care – in patients with candidaemia and invasive candidiasis.
The primary endpoint relevant for the EU approval was global cure (consisting of clinical cure, radiological cure and mycological eradication) at day 14 with a target non-inferiority margin of 20%, assessed in the modified intention-to-treat population.
Of the 222 patients screened for inclusion, 199 were deemed eligible and 100 were randomly assigned to the rezafungin group and 99 to the caspofungin group.
Some 59% of patients in the rezafungin group and 61% of patients in the caspofungin group had a global cure at day 14 (weighted treatment difference −1·1% [95% CI −14·9 to 12·7].
The trial therefore demonstrated statistical non-inferiority for rezafungin, dosed once weekly, when compared to caspofungin dosed once daily.
The most common treatment-emergent adverse events that occurred in at least 5% of patients in either group were pyrexia, hypokalaemia, pneumonia, septic shock and anaemia.
The findings are supported by the positive results of the STRIVE Phase II clinical trial and an extensive non-clinical development program.
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