HOT & NOT

The EC has approved BMS’ blood disease therapy Reblozyl (luspatercept) for transfusion-dependent anaemia associated with myelodysplastic syndromes (MDS) or beta thalassaemia. The decision makes Reblozyl the first and only erythroid maturation agent approved in the EU, representing a new class of therapy for eligible patients. Clinical data showed a statistically significant improvement in red blood cell (RBC) transfusion burden with the drug, with 37.9% of treated patients achieving independence from RBC transfusions for at least eight weeks during the first six months of the trial, compared to 13.2% of the placebo arm.

The European Commission (EC) has approved Johnson & Johnson group Janssen’s Ebola vaccine regimen for the prevention of Ebola Virus Disease caused by the Zaire ebolavirus species in individuals aged one year and above. Two marketing applications were submitted to the European Medicines Agency (EMA) for the vaccines composing the two-dose regimen, Zabdeno (Ad26.ZEBOV) and Mvabea (MVA-BN-Filo). Ad26.ZEBOV, the first dose, is based on Janssen’s AdVac viral vector technology, and MVA-BN-Filo, the second dose, is based on Bavarian Nordic’s MVA-BN technology, administered approximately eight weeks later.

NICE final guidance backs NHS use of Roche's Kadcyla (trastuzumab emtansine) for adjuvant treatment of HER2 positive early breast cancer, that is in people who have already had therapy to shrink their tumour but have residual disease in the breast or surrounding lymph nodes prior to surgery to remove it. Clinical evidence shows that for this patient subset Kadcyla increases the time people remain free of disease compared with Herceptin (trastuzumab) alone, the Institute noted. At three years, 88.3% of people treated with Kadcyla did not have their breast cancer return compared to 77.0% treated with Herceptin, an absolute improvement of 11.3%.

The EMA's human medicines committee (CHMP) has adopted a negative opinion recommending the refusal of a marketing authorisation for Daiichi Sankyo Europe's Turalio (pexidartinib), which has been developed to treat tenosynovial giant cell tumours. The Agency voiced concern that although the main study found that tumours shrank in patients treated with Turalio, “there was only a small improvement in symptoms such as pain and the ability to use the joint”. Also, it was not clear how long this effect lasts, and there was serious concern about “unpredictable, potentially life-threatening effects of Turalio on the liver”. The drug won US approval last year.

NICE has turned down NHS funding in preliminary guidelines for use of Novatis' Mayzent (siponimod) as a treatment for secondary progressive multiple sclerosis (SPMS) with evidence of active disease. According to the Institute, clinical trial results show that the drug cuts the number of relapses and slows disability progression compared with placebo. However, it is uncertain how effective Mayzent is compared with interferon beta-1b – the only disease-modifying treatment available for people with active SPMS – because there is no evidence directly comparing them. Novartis said it is disappointed with the decision, and stressed that the drug addresses unmet need.

A Phase III trial assessing Sanofi/Regeneron's arthritis drug Kevzara (sarilumab) for COVID-19 patients requiring mechanical ventilation has been stopped after it failed to meet its primary and key secondary endpoints. The firms did note that “minor positive trends” were observed in the pre-specified analysis group (ventilated at baseline) that did not reach statistical significance but that these were countered by negative trends in a subgroup of critical patients who were not mechanically ventilated at baseline. A separate Sanofi-led trial outside of the US in hospitalised patients with severe and critical COVID-19 using a different dosing regimen of Kevzara is ongoing.