At its November meeting, the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) recommended four drugs for approval, as well as seven line extensions, including three following re-examinations of previous negative opinions. Meanwhile, one company withdrew an application seeking a line extension. All of the decisions can be found detailed below.
Positive recommendations on new medicines:
Fexinidazole Winthrop (fexinidazole) for the treatment of human African trypanosomiasis. Sanofi noted that the drug, which could be distributed in endemic countries in 2019, is the first all-oral treatment shown to be efficacious for both stages of sleeping sickness.
Erleada (apalutamide) for the treatment of adults with non-metastatic castration-resistant prostate cancer who are at high risk of developing metastatic disease. Johnson & Johnson's oral androgen receptor inhibitor gained US approval in this indication in February.
Macimorelin Aeterna Zentaris (macimorelin) for the diagnosis of growth hormone deficiency in adults. Aeterna Zentaris' orally available ghrelin agonist was clearedby the FDA in this indication last year under the name Macrilen.
Silodosin Recordati (silodosin) for the treatment of the signs and symptoms of benign prostatic hyperplasia. Recordati's drug is a generic version of its own product Urorec.
Positive recommendations on extensions of therapeutic indications:
Kisqali (ribociclib) for the treatment of women with hormone receptor-positive, HER2-negative locally advanced or metastatic breast cancer in combination with fulvestrant as initial endocrine-based therapy and in women who have received prior endocrine therapy. Novartis' CDK4/6 inhibitor gained approval in the US in this indication in July, after the FDA reviewed it under its Real-Time Oncology Review and Assessment Aid pilot programmes.
MabThera (rituximab), marketed by Roche, in combination with glucocorticoids for the treatment of adults with severe, active granulomatosis with polyangiitis and microscopic polyangiitis.
Orkambi (lumacaftor/ivacaftor) for the treatment of people with cystic fibrosis aged 2 to 5 years old who have two copies of the F508del mutation. Vertex Pharmaceuticals noted that if approved, the drug will be the first medicine in Europe to treat the underlying cause of CF for approximately 1500 children with two copies of the F508del mutation.
Ravicti (glycerol phenylbutyrate), marketed by Horizon Pharma, for use as adjunctive therapy for the chronic management of patients with urea cycle disorders who cannot be managed by dietary protein restriction and/or amino acid supplementation alone.
Positive recommendations on extension of therapeutic indication following re-examination:
Blincyto (blinatumomab) for the treatment of adults with Philadelphia chromosome negative CD19 positive B-cell precursor acute lymphoblastic leukaemia in first or second complete remission with minimal residual disease greater than or equal to 0.1 percent. The CHMP issued a negative opinion on Amgen's bispecific CD19-directed CD3 T cell engager in July, with the re-examination beginning in September. The CHMP previously concluded that although Blincyto helped clear away residual cells in many patients, there was no strong evidence that it leads to improved survival in patients. However, after consulting a group of experts in cancer treatments, the committee agreed that, although there is no strong evidence of patients living longer, the available data from the main study indicate a good response to Blincyto. The CHMP requested that Amgen provide further data from ongoing studies once available.
Opdivo (nivolumab) plus low-dose Yervoy (ipilimumab) for the first-line treatment of adults with intermediate- and poor-risk advanced renal cell carcinoma. The CHMP initially rejected Bristol-Myers Squibb's combination therapy in July, with the re-examination starting in September. The committee previously issued a negative opinion due to questions regarding the benefit Yervoy added over Opdivo alone, whilst resulting in more side effects. Du...
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