Eleven medicines leap closer to EU approval

Santen’s Verkazia (ciclosporin) has been recommended for severe vernal keratoconjunctivitis (VKC), a rare form of chronic eye allergy that can lead to corneal ulcers and loss of sight, affecting 1 to 3 people out of 10,000 in the European Union.

VKC occurs mostly in young children and teenagers with other allergic conditions, such as eczema and asthma, and there is a need for additional treatments as those currently authorised - including corticosteroids - are not always effective in controlling severe forms of the condition.

Verkazia offers an alternative treatment approach in regulating the immune system to limit the progress of autoimmune conditions such as allergies, the CHMP noted.

Four other orphan medicines were put forward by the Committee: Merck and Pfizer’s Bavencio (avelumab) for the treatment of Merkel Cell Carcinoma; Advanced Accelerator Applications’ Lutathera (lutetium oxodotreotide), for the treatment of gastro-entero-pancreatic neuroendocrine tumours; Novartis’ Rydapt (midostaurin), for acute myeloid leukaemia, aggressive systemic mastocytosis, systemic mastocytosis with associated haematological neoplasm and mast cell leukaemia; and Ipsen Pharma’s Xermelo (telotristat ethyl), for carcinoid syndrome.

Elsewhere, Sanofi’s Dupixent (dupilumab) received a positive opinion for the treatment of moderate-to-severe atopic dermatitis.

Dupixent, which is already available to some patients in the UK via the Early Access to Medicines Scheme (EAMS), can improve the skin condition and reduce itching in patients with the condition. The most common side effects are injection site reactions, conjunctivitis, blepharitis, and oral herpes.

Janssen-Cilag’s Symtuza - a fixed-dose combination of darunavir, cobicistat, emtricitabine and tenofovir alafenamide - has been recommended for the treatment of HIV infection.

The benefits with Symtuza are its ability to achieve effective antiretroviral response in a once daily, single pill regimen, said the EMA, while the most common side effects are diarrhoea, nausea, fatigue and rash.

Roche’s immunotherapy Tecentriq (atezolizumab) has won backing for the treatment of locally advanced or metastatic urothelial carcinoma (UC) and locally advanced or metastatic non-small cell lung cancer (NSCLC).

The monoclonal antibody potentiates T-cell response, including anti-tumour response, through blockade of PD-L1 binding to PD-1 ligand, and was shown to induce durable responses in first-line cisplatin-ineligible and second-line urothelial carcinoma as well as an improvement in survival in the NSCLC indication.

The most common side effects observed with the drug are fatigue, decreased appetite, nausea, dyspnoea, diarrhoea, rash, pyrexia, vomiting, arthralgia, asthenia and pruritus, but it is also linked with immune-related adverse reactions including pneumonitis, hepatitis, colitis, hypothyroidism or hyperthyroidism, adrenal insufficiency, hypophysitis, type 1 diabetes mellitus, Guillain-Barré syndrome, meningoencephalitis and pancreatitis.

Tecentriq is also already available via the EAMS to patients with advanced cancer of the bladder and urinary system, which has spread and come back after one course of standard cancer chemotherapy.

Lastly, three generic medicines also picked up a positive opinion from the CHMP: Accord Healthcare’s Entecavir Accord (entecavir) and Mylan’s Entecavir Mylan (entecavir), both for the treatment of chronic hepatitis B; and Accord’s Lacosamide Accord (lacosamide), for the treatment of epilepsy.

On the downside, negative opinions were handed down for Vanda Pharmaceuticals’ schizophrenia drug Fanaptum (iloperidone), on a range of safety and efficacy concerns, and Nektar Therapeutics’ Onzeald (etirinotecan pegol) for breast cancer with brain metastases, because its benefit had not been demonstrated.