Regulatory Index News: 11/01/2018

Welcome to your Regulatory Index News update today. Stay up to date with the recent EU scope expansion, breakthrough status designation and UK launch here.

EU regulators clear Vertex’ Orkambi for CF therapy in younger patients

The EMA have expanded the scope of Orkambi to include children from age six up to eleven with cystic fibrosis that have two copies of F508del mutation. This expansion is due to data based on two Phase III trials which show statistically significant improvements in lung function in children which are treated with Orkambi rather than placebo. The study also reports improvements in BMI and the Cystic Fibrosis Questionnaire-Revised (CFQ-R) respiratory domain score.  Safety of the therapy in children was also deemed acceptable. This expansion of scope is good news as there are approximately 3,400 European children ages 6-11 who have two copies of the F508del mutation. Vertex is now focusing on country reimbursement processes to ensure access. If you would like to read more, please click here for a PharmaTimes article.

MSD/Eisai cancer combo wins US breakthrough status

MSD and Eisai are developing a combination therapy for kidney cancer which has now been awarded breakthrough designation in the US. The drug makers are combining Eisai’s multiple receptor tyrosine kinase inhibitor Lenvima with Merck’s anti-PD-1 therapy Keytruda in patients with advanced and/or metastatic renal cell carcinoma (RCC) to test its affects. Interim results from the Phase II study, 111, found that the combination treatment achieved a confirmed objective response rate (ORR) at week 24 of 63%, while disease control rate was 96%. This combination breakthrough designation may now allow for expedited development and regulatory review, with the firms also having access to more intensive guidance. Please click here for more details from a PharmaTimes article.

UK launch for MSD Clostridium difficile antitoxin

Zinplava has been launched in the UK, offering patients a novel therapeutic option for the prevention of clostridium difficile infection (CDI) recurrence. Whilst the antibody drug itself is not an antibacterial and it is not indicated to treat infection, it is designed to neutralise C. difficile toxin B which can damage the gut wall, causing inflammation and diarrhoea. This makes it the only EC licensed non-antibiotic option indicated for CDI in high risk-adults where around one-in-four patients experience a recurrence, and 40% of those have further recurrences. Pivotal phase III clinical studies showed that infection recurrence rates were significantly lower in those patients given Zinplava than those treated with placebo. If you would like to read further on this launch, please click here for an article from PharmaTimes. 



Max Lymbery

Date Published

11th January 2018

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