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Take a look at a selection of our recent media coverage:
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.
15th December 2023
Dostarlimab (brand name Jemperli) has been granted marketing authorisation by the European Commission (EC) in combination with carboplatin-paclitaxel (chemotherapy) for certain types of endometrial cancer, its manufacturer GSK has announced.
It is the first and only frontline immuno-oncology treatment in the European Union (EU) for adult patients with mismatch repair deficient (dMMR)/microsatellite instability-high (MSI-H) primary advanced or recurrent endometrial cancer and who are candidates for systemic therapy.
The authorisation also converts the EC’s previous conditional approval of dostarlimab to a full approval as a monotherapy for treating adult patients with dMMR/MSI-H recurrent or advanced endometrial cancer that has progressed on or following prior treatment with a platinum-containing regimen.
Dostarlimab is a programmed death receptor-1 (PD-1)-blocking antibody that binds to the PD-1 receptor and blocks its interaction with the PD-1 ligands PD-L1 and PD-L2.
Dr Mansoor Raza Mirza, chief oncologist, Copenhagen University Hospital, Denmark, said: ’Today’s European Commission approval is welcomed news as I believe it will define a new standard of care for certain patients with advanced or recurrent endometrial cancer in the EU.’
The EC authorisation of dostarlimab is based on interim analysis results from the two-part global, randomised, double-blind, multicentre phase 3 trial RUBY.
The dual-primary endpoints in Part 1 were investigator-assessed progression-free survival (PFS) based on the Response Evaluation Criteria in Solid Tumours v1.1 and OS.
The trial met its primary endpoint of investigator-assessed PFS, demonstrating a statistically significant and clinically meaningful benefit in patients treated with dostarlimab plus carboplatin and paclitaxel in the dMMR/MSI-H population.
A 72% reduction in the risk of disease progression or death was observed relative to chemotherapy alone (HR: 0.28 [95% CI: 0.16-0.50]).
In a prespecified, exploratory analysis of OS in the dMMR/MSI-H population, the addition of dostarlimab to chemotherapy resulted in a 70% reduction in the risk of death relative to chemotherapy alone (HR: 0.30 [95% CI: 0.13-0.70]).
The safety and tolerability profile for dostarlimab plus carboplatin and paclitaxel was generally consistent with the known safety profiles of the individual agents.
The most common adverse reactions (≥ 10%) in patients receiving dostarlimab plus chemotherapy were rash, hypothyroidism (underactive thyroid), increased alanine aminotransferase or increased aspartate aminotransferase (increased liver enzyme levels in the blood), pyrexia (fever) and dry skin.
Dr Mirza, the RUBY trial’s principal investigator, commented: ’The results from the RUBY trial, which led to this approval, underscore the practice-changing potential of dostarlimab for these patients.’
24th November 2023
Zanubrutinib (brand name Brukinsa) has been granted marketing authorisation by the European Commission (EC) for use in combination with obinutuzumab for eligible patients with follicular lymphoma, its manufacturer BeiGene has announced.
This highly selective, oral Bruton’s tyrosine kinase (BTK) inhibitor is now approved for the treatment of adult patients with relapsed or refractory (R/R) follicular lymphoma who have received at least two prior lines of systemic therapy.
The EC approval is based on positive results from the phase 2 ROSEWOOD study which looked at zanubrutinib plus obinutuzumab compared with the anti-CD20 monoclonal antibody obinutuzumab alone.
Some 217 patients with R/R follicular lymphoma who had received at least two prior lines of systemic therapy were included in the global, randomised, open-label study.
The primary endpoint was overall response rate (ORR) by independent central review. Secondary endpoints included duration of response (DOR), progression-free survival (PFS), overall survival, and safety.
The researchers found the ORR to be 69.0% in the zanubrutinib plus obinutuzumab arm versus 45.8% in the obinutuzumab arm (P = 0.0012), with a median follow-up of approximately 20 months.
Responses were durable with 18-month landmark DOR of 69.3% in the zanubrutinib combination arm. Additionally, the median PFS for patients treated in the combination arm was 28.0 months, compared to 10.4 months for patients treated with only obinutuzumab (HR: 0.50 [95% CI: 0.33, 0.75]; P = 0.0007).
Zanubrutinib plus obinutuzumab was generally well-tolerated, with safety results consistent with previous studies of both medicines.
Dr Pier Luigi Zinzani, full professor of haematology at the Institute of Haematology ‘Seràgnoli’, University of Bologna, Italy, said: ‘People living with follicular lymphoma often experience relapse and have poor responses to subsequent lines of therapy, making it imperative to improve outcomes.
‘The results from the ROSEWOOD trial demonstrated a significant clinical benefit of Brukinsa plus obinutuzumab for patients with relapsed or refractory follicular lymphoma. Brukinsa is a chemotherapy-free, oral treatment option that can be a practice-changing option for eligible patients with relapsed or refractory follicular lymphoma.’
This is the fourth approved indication for this drug in the EU, which gives it ‘the broadest label of any medicine in its class globally’, according to Mehrdad Mobasher, chief medical officer, hematology at BeiGene.
In addition to R/R follicular lymphoma, zanubrutinib is approved in the EU as monotherapy for the treatment of adult patients with: chronic lymphocytic leukaemia, marginal zone lymphoma who have received at least one prior anti-CD20-based therapy, and Waldenström’s macroglobulinemia who have received at least one prior therapy, or in firstline treatment for patients unsuitable for chemo-immunotherapy.
The use of zanubrutinib in R/R follicular lymphoma is currently under review by regulatory authorities in Switzerland and the United Kingdom as part of the Access Consortium New Active Substance Work-sharing Initiative.
23rd October 2023
The injectable bispecific antibody epcoritamab (brand name Tepkinly) has been granted conditional marketing authorisation by the Medicines and Healthcare products Regulatory Agency (MHRA), its manufacturer Abbvie has announced.
This follows its conditional marketing authorisation by the European Commission in late September 2023.
The conditional marketing authorisations recommend epcoritamab as a monotherapy for adult patients with relapsed or refractory (R/R) diffuse large B-cell lymphoma (DLBCL) after two or more systemic therapies.
Epcoritamab is the first and only off-the-shelf, subcutaneously administered CD3xCD20 T-cell-engaging bispecific antibody. It is designed to simultaneously bind to both the cluster of differentiation (CD)3 proteins on immune T cells and the CD20 proteins on cancerous B cells and activate the T cell to destroy the B cell.
Unlike existing therapeutic options for DLBCL, epcoritamab does not require cell collection and engineering before eligible patients can start treatment and it can be delivered in broader settings. It therefore has the potential for greater geographical accessibility and more timely administration.
Professor Chris Fox, professor of haematology at the University of Nottingham’s School of Medicine, and honorary consultant haematologist at Nottingham University Hospitals NHS Trust, UK, said: ‘Despite recent therapeutic advances, treatment options for patients with R/R DLBCL after two previous therapies are limited. For such patients living with this type of aggressive blood cancer, many experience disease progression and have poor prognosis.
‘As a novel bispecific antibody, given as a subcutaneous injection, epcoritamab offers a new treatment option for this difficult-to-treat patient group.’
Anna Sureda, head of clinical hematology department, Institut Català d’Oncologia – L’Hospitalet in Barcelona, Spain, said: ‘R/R DLBCL is an aggressive cancer and patients can face a difficult and emotional treatment journey. At this point in the journey, a patient may have had multiple lines of therapy and will already have experienced relapse.
‘This European Commission approval represents an important moment for the DLBCL patient community and brings with it a potential opportunity for effective disease management for a condition with limited available treatment options.’
The conditional marketing authorisations are based on the results of the single-arm Phase 1/2 EPCORE NHL-1 trial, which investigated epcoritamab as monotherapy for 139 patients with R/R DLBCL after two or more lines of systemic therapy.
Phase 1 of the study looked at dose escalation, which determined the recommended phase 2 dose, and the phase 2 expansion treated additional patients to explore safety and efficacy. The primary endpoint was overall response rate determined by the 2014 Lugano criteria as assessed by an independent review committee.
Secondary endpoints included duration of response, complete response rate, progression-free survival, overall survival, time to response, time to next therapy and rate of minimal residual disease negativity.
Patients achieved an overall response rate of 61.9% (n=86/139) and a complete response rate of 39.6% (n=54/139).
The researchers also found that epcoritamab prevented the growth or spread of the cancer for an average of 15.6 months, and the patients lived for an average of 19.4 months from the start of epcoritamab therapy.
The trial results demonstrated a manageable safety profile, with cytokine release syndrome being the most common serious adverse event (31.1%), followed by pneumonia (7.2%), upper respiratory tract infections (2.4%), febrile neutropenia, immune effector cell-associated neurotoxicity syndrome (ICANS) (2.4%) and pyrexia (2.4%).
Four patients (2.4%) experienced a fatal adverse reaction – ICANS in one patient (0.6%) and pneumonia in three patients (1.8%).
Further data from the trial are expected in due course.
Commenting on the MHRA conditional marketing authorisation, Belinda Byrne, medical director at AbbVie UK, said: ‘AbbVie is committed to advancing care for people living with blood cancer. Today’s news is an important step forward in enabling us to provide this hard-to-treat patient group with an innovative subcutaneous treatment.
‘We are working with the NHS and relevant authorities to bring access to eligible patients and clinicians throughout the UK as quickly as possible.’
Epcoritamab is delivered to eligible patients by clinicians as a weekly subcutaneous injection for 12 weeks, then moves to every other week for 24 weeks (12 injections), before continuing as one injection every four weeks until treatment is discontinued due to cancer progression or side effects.
Epcoritamab is the first injectable bispecific antibody to be approved for use in DLBCL. It follows the recent MHRA approval and National Institute for Health and Care Excellence recommendation of glofitamab (brand name Columvi), a bispecific antibody administered via infusion.
Glofitamab also gained conditional marketing authorisation in the EU in July 2023.
1st September 2023
Nivolumab (brand name Opdivo) has been approved by the European Commission for use as a monotherapy for the adjuvant treatment of stage IIB or IIC melanoma, its manufacturer Bristol Myers Squibb has announced.
The approved indication is for use in adults and adolescents 12 years of age and older with stage IIB or IIC melanoma, or melanoma with involvement of lymph nodes or metastatic disease who have undergone complete resection.
Combining relatlimab with nivolumab is known to increase progression-free survival compared to the use of nivolumab alone in patients with metastatic or unresectable melanoma.
The EC approval was based on the findings of the CheckMate -76K trial, which evaluated adjuvant nivolumab compared to placebo in patients with completely resected stage IIB or IIC melanoma.
CheckMate -76K was a randomised phase 3, double-blind study directly comparing adjuvant nivolumab at a dose of 480 mg Q4W for up to 12 months versus placebo in patients with completely resected stage IIB or IIC melanoma.
The primary endpoint of the trial was recurrence-free survival (RFS), with secondary endpoints including overall survival (OS), distant metastases-free survival (DMFS), progression-free survival on next-line therapy (PFS2) and safety endpoints.
Findings from the trial showed that adjuvant nivolumab, after a minimum follow-up of 7.8 months, reduced the risk of recurrence or death by 58% versus placebo (hazard ratio, HR = 0.42, 95% CI 0.30 – 0.59, p < 0.0001).
In addition, the 12-month RFS rate was 89% compared to 79% for placebo. This RFS benefit was observed across predefined subgroups in the trial, including T category and disease stage.
In terms of safety, the pooled dataset of nivolumab as monotherapy found that some of the most frequent adverse reactions, occurring in more than 10% of patients, were fatigue (44%), musculoskeletal pain (28%), diarrhoea (26%), rash (24%) and cough (22%). However, the majority of these adverse reactions were mild to moderate (Grade 1 or 2) in severity.
Peter Mohr, chief physician and head of the Skin Cancer Center, Buxtehude, Department of Dermatology, Elbe-Kliniken, Germany, said: ‘Patients with stage IIB or IIC melanoma are at a high risk of disease recurrence following surgery. This can be a very impactful event for patients. This approval reinforces the benefit that nivolumab may offer when used after resection, potentially preventing the disease from recurring.‘
31st August 2023
Mitazalimab has received orphan drug designation status by the European Medicines Agency (EMA) for the treatment of patients with pancreatic cancer, its manufacturer Alligator Bioscience has announced.
A human CD40 agonistic antibody targeting CD40, mitazalimab kickstarts the cancer-immunity cycle by priming and activating tumour-specific T cells. Targeting CD40 with mitazalimab has the potential to augment responses to chemotherapy.
Pancreatic ductal adenocarcinoma (PDAC) is known to be the fourth-leading cause of cancer related mortality in the world and has a poor prognosis, with a five-year survival rate of below 5%.
To qualify for the EMA’s orphan designation, a medicine must be intended for the treatment, prevention or diagnosis of rare, life-threatening or chronically debilitating diseases that affect fewer than five in 10,000 persons in the EU. Medicines that meet these criteria are eligible for financial and regulatory incentives that include 10 years of marketing exclusivity in the EU after product approval.
The EMA orphan drug designation follows a similar approval by the FDA in May 2023.
Commenting on the EMA approval for orphan drug designation status, Søren Bregenholt, CEO of Alligator Bioscience, said: ‘We are very pleased that the European Medicines Agency has granted orphan designation to our lead asset mitazalimab in the treatment of pancreatic cancer.
‘It is our second orphan designation this year following the FDA‘s decision to grant us [orphan drug designation] in May, meaning mitazalimab now has stronger commercial protection through market exclusivity in these two key markets. This latest designation adds to the momentum we are building in our efforts to bring this promising drug candidate to market.‘
Mitazalimab is currently being evaluated in the phase 1b/2 OPTIMIZE-1 trial in combination with mFOLFIRINOX chemotherapy for adult patients with previously untreated metastatic PDAC.
In the trial, participants receive mitazalimab and mFOLFIRINOX via intravenous infusions following a 14-day cycle schedule. Mitazalimab is administered two days after mFOLFIRINOX, except for the first cycle of 21 days, where the drug is administered on days one and 10 with infusion of mFOLFIRINOX starting on day eight.
Interim results from OPTIMIZE-1 released in June 2023, showed a deepening of tumour response and an increase in the objective response rate (ORR) from 52% to 57% in a cohort of 23 patients.
In the full study cohort of 57 patients, there was an interim ORR of 44%, and this is expected to further improve with longer follow-up. A median duration of response of 8.7 months was also reported.
24th August 2023
Talquetamab has been granted a conditional marketing authorisation by the European Commission as monotherapy for the treatment of adult patients with relapsed and refractory multiple myeloma.
Talquetamab (brand name Talvey) is approved as a weekly or biweekly subcutaneous injection after an initial step-up phase. It can be used as monotherapy for the treatment of patients relapsed and refractory multiple myeloma who have had an inadequate response to at least three prior therapies, including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody. They must also have demonstrated disease progression on the last treatment.
The treatment is a bispecific engaging antibody that binds to CD3, on the surface of T-cells, and G protein-coupled receptor class C group 5 member D (GPRC5D), a novel target highly expressed on the surface of multiple myeloma cells and hard keratinised tissues.
Multiple myeloma is a malignancy of terminally differentiated plasma cells that typically presents with bone marrow infiltration of clonal plasma cells and monoclonal protein in the serum and/or urine.
Commenting on the EC decision, Edmond Chan, senior director EMEA therapeutic area lead haematology, Janssen-Cilag Limited, said: ‘[This] brings a new off-the-shelf option, with a novel cellular target and the immediate option of biweekly dosing, to an area of high unmet clinical need.
‘The high overall response rates in patients with heavily pretreated multiple myeloma, including those with prior T-cell redirection therapy, are encouraging, and we believe talquetamab has the potential to offer physicians flexibility and versatility when determining the optimal treatment regimen for their patients.‘
The EC approval follows a similar FDA approval in the US in August 2023.
The talquetamab approval was based on efficacy data from the phase 1 and phase 2 MonumenTAL-1 study. In the trial, patients had received a median of five prior lines of therapy and talquetamab was given at a weekly dose of 0.4 mg/kg or biweekly 0.8 mg/kg.
For both dosage regimens, patients showed meaningful overall response rates. After a median follow-up of 12.7 months, 71.7% of response-evaluable patients treated at the 0.8 mg/kg biweekly dose achieved a response. Some 60.8% achieved a very good partial response (VGPR) or better and 38.7% achieved a complete response (CR) or better.
After a median follow-up of 18.8 months, 74.1% of response-evaluable patients treated with the 0.4 mg/kg weekly dose of talquetamab achieved a response; 59.5% a VGPR or better and 33.6% achieved a CR or better.
An estimated 76.3% and 51.5% of patients maintained a response for at least nine months at the 0.8 mg/kg biweekly and 0.4 mg/kg weekly talquetamab doses, respectively.
Maria-Victoria Mateos, consultant physician in haematology at the University Hospital of Salamanca in Spain, said: ‘As multiple myeloma progresses and patients cycle through treatments, the disease becomes
more difficult to treat and remission periods shorten. Targeting GPRC5D has been shown to deliver deep responses, and unlike many other targets for multiple myeloma, its expression is limited on immune cells providing an important new approach to targeting this heterogenous disease.‘
2nd August 2023
Eculizumab has been approved by the European Commission for use in those aged six to 17 years who have refractory generalised myasthenia gravis (gMG) and who are also anti-acetylcholine receptor (AChR) antibody-positive (Ab+).
The use of eculizumab (brand name Soliris) has been extended in the European Union (EU) to include the treatment of refractory gMG in children and adolescents, having already been licensed for the treatment of adults with refractory gMG who are AChR antibody-positive. This latest approval represents the first and currently only targeted therapy approved for the treatment of paediatric patients with the disease in the EU.
The extension of eculizumab’s license for use in children and adolescents was based on findings from the Phase III trial of Soliris. Here, eculizumab demonstrated a clinical benefit in paediatric patients with refractory gMG who previously failed immunosuppressive treatment and who continued to experience significant unresolved disease symptoms.
The drug demonstrated a statistically significant improvement in the primary endpoint, which was a change from baseline in the Quantitative Myasthenia Gravis (QMG) total score – a physician-reported scale assessing disease severity and function – at week 26 (-5.8, 95% CI -8.4, -3.13, p < 0.0004).
Dr John Brandsema, from the Children’s Hospital of Philadelphia and the primary investigator in the study, said: ‘This approval represents a significant advance in care for paediatric patients with refractory gMG, who previously had no targeted treatment options to help manage their condition. Soliris showed clinical benefit and sustained improvements in disease severity through 26 weeks in a Phase III trial, offering potential to improve quality of life and redefine disease management for children and adolescents living with this rare neurological disease.‘
Marc Dunoyer, chief executive officer, Alexion, the manufacturer of Soliris, said: ‘The impact of gMG on children can be devastating, and families have long been awaiting solutions. This approval of our first-in-class C5 inhibitor Soliris for paediatric patients with refractory gMG in the EU exemplifies our efforts to deliver transformative medicines that help address unmet medical needs for rare disease communities. Soliris offers hope for improved outcomes for children and adolescents impacted by gMG and we are committed to increasing access for these families as quickly as possible.‘
Generalised myasthenia gravis is a rare autoimmune disorder characterised by loss of muscle function and severe muscle weakness. Some 80% of patients with gMG are AChR antibody positive, which means that they produce specific antibodies that bind to signal receptors at the neuromuscular junction – the connection point between nerve cells and the muscles they control. This effect activates the complement system, causing the immune system to attack the neuromuscular junction and leading to inflammation and a breakdown in communication between the brain and the muscles.
A recent phase 1b/2a study found that using RNA CAR T cell therapy improves clinical symptoms in adult patients with myasthenia gravis.