Approval
Medicines recently licensed by the European Medicines Agency.
Bazedoxifene and conjugated oestrogens
New medicine Menopausal symptoms
A combination preparation containing bazedoxifene, a selective oestrogen receptor modulator, and conjugated oestrogens (Duavive; Pfizer) has been licensed to treat oestrogen deficiency symptoms in postmenopausal women with a uterus who are unsuitable for progestin-containing therapy. The treatment was assessed in the SMART studies, which involved nearly 7,500 women. The SMART-2 study found the incidence of hot flushes at week 12 was reduced from baseline by 74% in patients taking Duavive and by 51% with placebo. In SMART-3, patients in the Duavive group reported improved sexual function and total scores on a quality of life questionnaire at week 12 (P<0.001) compared with placebo. Increases in lumbar spine bone mineral density after one year in SMART-1, 4 and 5 were significantly greater in patients taking Duavive compared with placebo (P≤0.001). The incidence of endometrial bleeding and breast tenderness was similar to that with placebo, and significantly lower than with medroxyprogesterone and conjugated oestrogens (P<0.001). The most common side effect is abdominal pain, occurring in more than 10% of women.
Ramucirumab
New medicine Gastric cancer
Ramucirumab (Cyramza; Eli Lilly) has been approved to treat adults with advanced gastric cancer or gastro-oesophageal junction adenocarcinoma whose disease has progressed following chemotherapy. It can be used alone or in combination with paclitaxel. Ramucirumab blocks human vascular endothelial growth factor receptor 2, which plays an important role in forming new blood vessels in tumours. The REGARD study (n=355) found median overall survival was 5.2 months in patients given intravenous ramucirumab and 3.8 months in patients given placebo (P=0.047). In the RAINBOW study, in which 665 patients were randomised to ramucirumab or placebo, plus paclitaxel, median overall survival was 9.6 months and 7.4 months, respectively (P=0.017). Adverse effects include fatigue, neutropenia, leukopenia, diarrhoea, nose bleeds and hypertension.
Positive opinion
Medicines that have been recommended for licensing by the European Medicines Agency.
Collagenase clostridium histolyticum
New indication Peyronie’s disease
Collagenase clostridium histolyticum (Xiapex; Swedish Orphan Biovitrum) has been given a positive opinion for a licence extension to treat Peyronie’s disease (PD) in men with a palpable plaque and curvature deformity of the penis of at least 30 degrees.
Meta-analysis of two 52-week studies involving a total of 832 men found intra-lesional injections of collagenase (two every six weeks; 79% of patients receiving the maximum eight injections) produced a 34% mean improvement in penile curvature, compared with 18% for placebo (mean change per patient -17.0 vs. -9.3 degrees respectively; P<0.0001). Mean symptom bother scores also improved significantly with collagenase compared with placebo (-2.8 vs. -1.8; P=0.004). Penile bruising, swelling and pain occurred in 80%, 55% and 45%, respectively, of men given collagenase compared with 26%, 3% and 9% given placebo.
Dalbavancin
New medicine Skin infection
Dalbavancin (Xydalba; Durata), a second-generation once-weekly intravenous glycopeptide antibiotic, has been recommended for a licence to treat acute bacterial skin and skin structure infections in adults. It is active against methicillin-resistant Staphylococcus aureus and some S. aureus strains with reduced susceptibility to glycopeptides.
Pooled analysis of the DISCOVER 1 and 2 studies (n=1,312) showed two doses of dalbavancin were non-inferior to vancomycin (given for three days with the option of switching to oral linezolid for a total of 10-14 days); 79.7% in the dalbavancin group and 79.8% in the vancomycin-linezolid group had an early clinical response at 48 to 72 hours. Outcomes were similar at the end of therapy. Common side effects are nausea, diarrhoea and headache.
Naltrexone and bupropion
New formulation Obesity
A treatment containing naltrexone and bupropion (Mysimba; Orexigen) has been given a positive opinion for a licence to be used for weight management in adults who are obese or those who are overweight and have weight-related co-morbidities. Naltrexone (an opioid receptor antagonist) and bupropion (a dopamine and noradrenaline reuptake inhibitor) act on two areas within the brain to control food intake and energy expenditure, and moderate food cravings.
In a study involving 1,742 patients with a body mass index (BMI) of 30-45kg/m2 or a BMI 27-45kg/m2 and dyslipidaemia or hypertension, mean body weight at week 56 decreased from baseline by 5% with Mysimba compared with 1.3% with placebo (P<0.0001). A total of 39% of patients taking Mysimba achieved a weight loss of 5% or more, compared with 16% taking placebo (P<0.0001). Mysimba has also been shown to reduce weight and improve glycaemic control in patients with type 2 diabetes. The most frequently reported side effects are nausea, constipation, vomiting, dizziness and dry mouth.
Application
Medicines for which a licensing application has been submitted to the European Medicines Agency
Sebelipase alfa
New medicine Lysosomal acid lipase deficiency
Sebelipase alfa, a recombinant human lysosomal acid lipase (LAL), is being developed by Synageva BioPharma to treat LAL deficiency. This inherited disease affects less than two in every 100,000 people and results in fats accumulating within cells and tissues. It can be fatal and currently no effective treatments are available. In a phase II study, eight adults given sebelipase alfa infusions every other week for a year had mean reductions from baseline of 60% in low-density lipoprotein, 39% in total cholesterol and 36% in triglycerides. The phase III ARISE study in 66 children and adults is expected to be completed in 2015.
