The US Food and Drug Administration (FDA) has approved Kiniksa Pharmaceuticals’ interleukin-1 alpha (IL-1α) and interleukin-1 beta (IL-1β) inhibitor Arcalyst (rilonacept) for the treatment of recurrent pericarditis and reduction in risk of recurrence in adults and children 12 years and older. Recurrent pericarditis is an autoinflammatory cardiovascular disease that often presents with chest pain, and is associated with changes in electrical conduction and build-up of fluid around the heart. The FDA approval is based on positive data from the RHAPSODY trial in recurrent pericarditis. Data from this trial showed that Arcalyst treatment improved clinically meaningful outcomes associated with the significant unmet medical need in recurrent pericarditis. There were rapid and sustained reductions in both reported pain and inflammation as early as after the first dose of Arcalyst. “The approval of Arcalyst in recurrent pericarditis is an extremely important achievement for those suffering from this disease as there can be significant ...
Daiichi Sankyo’s cholesterol-lowering drugs Nilemdo and Nustendi have been approved by the UK National Institute for Health and Care Excellence (NICE) for use on the NHS. Nilemdo (bempedoic acid) and Nustendi (bempedoic acid/ezetimibe) have been recommended for the treatment of primary hypercholesterolaemia or mixed dyslipidaemia (heterozygous familial and non-familial). It is authorised as an adjunct to diet and when statins are contraindicated or not tolerated, or when the standard cholesterol treatment ezetimibe alone does not control low-density lipoprotein cholesterol (LDL-C) adequately. “Today’s decision is a landmark step forward for patients and the NHS in working towards a national ambition of preventing cardiovascular disease and events in line with the Long Term Plan,” said Manuel Reiberg, managing director, Daiichi Sankyo UK. “We are now more dedicated than ever to reduce the impacts of the UK’s biggest killer through our ongoing collaboration with the medical community and the NHS,” he added. In the UK, ...
A partnership of frontline health charities has signed an open letter encouraging people with underlying conditions to book in for a COVID-19 vaccine. The charities, including Mencap, Diabetes UK and Lupus UK, have urged people with conditions including cancer, diabetes and heart disease to protect themselves and others around them from the coronavirus by receiving a vaccine. Although over half of those in this priority group – known as cohort six – have already received their first vaccine dose, the charities’ letter is ‘hoped’ to further increase take-up among this group. Cohort six includes individuals aged 16 to 64 years old who have certain long-term conditions, identified by the Joint Committee on Vaccination and Immunisation (JCVI), that puts them at a higher clinical risk from COVID-19. It also includes carers who are eligible for a carer’s allowance, as well as those who are the sole or primary carer of an ...
AstraZeneca (AZ) has issued an update on the safety of its COVID-19 vaccine, saying a review showed no evidence of an increased risk of blood clots among people vaccinated with its jab. In a statement, AZ said that across the EU and UK, there have been 15 events of deep vein thrombosis (DVT) and 22 events of pulmonary embolism reported among people given its vaccine – based on the number of cases the company has received as of 8 March. AZ added that a ‘careful’ review of all available safety data – including more than 17 million people vaccinated in the EU and UK with its COVID-19 vaccine – has shown ‘no evidence of an increased risk of pulmonary embolism, DVT or thrombocytopenia, in any defined age group, gender, batch or in any particular country.’ The cases reported so far are ‘much lower’ than what would be expected to naturally ...
GlaxoSmithKline (GSK) is set to leverage the Cell and Gene Therapy (CGT) Catapult’s facility in Stevenage to expand its own clinical trial manufacturing capacity for cell and gene therapy. GSK will use the Stevenage facility to perform GMP cell processing to accelerate its pipeline of cell and gene therapy for clinical trials and streamline technical transfer. “The UK already has significant capabilities in cell and gene therapy, and this agreement illustrates how strategic investments by GSK can make them even stronger,” said Tony Wood, senior vice president, medicinal science and technology, GSK. “Working more closely with CGT Catapult will help us advance our promising cell and gene therapy programmes, and bring these transformative medicines more quickly to the patients who desperately need them,” he added. There has been a 48% increase in the UK GMP manufacturing space for cell and gene therapies in 2020, according to CGT Catapult. It added that ...
A new study has found that BRCA1-positive breast cells appear to undergo changes before becoming cancerous, which could help identify which patients can benefit from preventative surgery. The study in mice, funded by Cancer Research UK and published today in Nature Communications, found that breast cells with the BRCA1 gene mutation develop changes similar to those seen in late pregnancy before becoming cancerous. The researchers suggest that women with BRCA1 mutations could be screened in the future to monitor changes to their breast cells. This could help to inform decision-making revolving around preventative surgery, by showing who could benefit from this option. After analysing the mammary tissue of mice at various ages carrying the BRCA1 mutation, the researchers analysed breast cells from 12 women who had a BRCA1 mutation and had undergone preventative surgery. The team found that four out of the 12 women had detectable levels of markers of early stages ...
Albireo Pharma has submitted odevixibat to the US Food and Drug Administration (FDA) and European Medicines Agency (EMA), seeking approval for the treatment of patients with progressive familial intrahepatic cholestasis (PFIC). The US-based biopharmaceutical company has previously received fast track, rare paediatric disease and orphan drug designations for odevixibat in the US for PFIC. The drug also has orphan drug designations for the treatment for Alagille Syndrome, biliary atresia and primary biliary cholangitis. Odevixibat is a one-daily non-systemic ileal bile acid transport inhibitor (IBATi) currently being developed for the treatment of a number of rare paediatric liver diseases, such as PFIC, biliary atresia and Alagille syndrome. In a phase III trial evaluating the efficacy and safety of odevixibat in PFIC, the drug met two primary endpoints, demonstrating the ability to reduce serum bile acid responses and improve pruritus assessments with a single digit diarrhoea rate. “With randomised, placebo-controlled PEDFIC data, ...
The UK’s National Institute for Health and Care Excellence (NICE) has recommended selective internal radiation therapy (SIRT) for the treatment of advanced liver cancer in final draft guidance. NICE’s draft guidance recommends the use of SIR-Spheres – made by SIRTEX – and TheraSphere – made by Boston Scientific – for treating advanced liver cancer that can’t be removed surgically and when transarterial therapy is not appropriate. The recommendation also specifies that these two therapies should only be considered for people with Child-Pugh grade A liver impairment. Despite limited clinical trial data for SIRTs compared with other treatment options, NICE’s appraisal committee concluded that SIRT may have fewer and more manageable side effects compared to systemic therapy sorafenib. Although SIR-Spheres and TheraSphere are slightly less clinically effective than sorafenib, they cost less. The cost savings mean that these two therapies can be recommended as cost-effective options for people with Child-Pugh grade ...
Oxford, UK-based Genomics plc has completed a $30m funding round, which the company will use to expand its work on its patient-centric, population health platform. The funding round included significant investments from life sciences investors Foresite Capital and F-Prime Capital, with existing backers Oxford Sciences Innovation and Lansdowne Partners also participating. Genomics plc uses large-scale genetic information to develop precision healthcare tools, working within the new area of genomic prevention. Genomic prevention allows for personalised estimates of risk for common diseases, ahead of actual disease manifestation. This enables targeted interventions and tailored screening to either prevent disease entirely or to catch it early when outcomes remain favourable. “We are delighted to be supported by leading life sciences investors who share our vision. We have known for over 50 years that genetics plays a key role in risk for all the common chronic diseases and cancers, and now we have a ...
The Competition Appeal Tribunal (CAT) has maintained a decision made last year by the UK’s Competition and Markets Authority (CMA) to fine pharmaceutical firm Lexon £1.2m after finding the company guilty of breaking competition law. Last year, the CMA found that Lexon, along with King Pharmaceuticals and Alissa Healthcare Research, had illegally shared commercially sensitive information in an attempt to drive up prices of the antidepressant nortriptyline. The CMA concluded that from 2015 to 2017 – when the cost of the drug was decreasing – the three companies exchanged information about prices, the volumes they were supplying and Alissa’s plans to enter the market. The CMA then fined all three companies, fining Lexon a total of £1.2m – however, Lexon maintained it had not broken the law and appealed the decision and fine. However, the CAT has decided to uphold the CMA’s original finding that Lexon broke competition law and ...
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