Significant strides have been made across the country towards delivering the commitments made in 2013’s UK Strategy for Rare Diseases, but the first progress report since its launch also shows that much remains to be done to improve the care of patients.
News
Genentech bags US nod for Gazyvaro in follicular lymphoma
Roche group Genentech has secured US approval for cancer drug Gazyvaro/Gazyva as a treatment for some patients with previously treated follicular lymphoma, the most common type of indolent non-Hodgkin’s lymphoma.
Six medicines step closer to EU approval
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has put forward six new medicines for marketing authorisation, including two with orphan drug status.
AZ hands CMS rights to Imdur, Plendil for $500m
AstraZeneca has handed China Medical System (CMS) certain rights to its heart and blood pressure drugs Imdur and Plendil in deals worth some $500 million.
UCB launches new epilepsy drug in the UK
UCB has rolled out Briviact across the UK, offering a new treatment approach for patients with uncontrolled epilepsy.
Success for rare disease access programme register
Global pharmaceutical and services company Clinigen and news and information provider CheckOrphan have announced the 128th addition to a unique online access programme register for rare diseases, on the first anniversary of its launch.
Analysis shows Boehringer’s Ofev cuts IPF exacerbations
Boehringer Ingelheim’s Ofev cut the risk of acute exacerbations by around 50% in people with the rare lung disease idiopathic pulmonary fibrosis (IPF), according pooled analysis of data from the TOMORROW and INPULSIS trials.
EU reviews Gilead’s TAF for hep B infection
European regulators have accepted an application from Gilead seeking permission to market tenofovir alafenamide as a single agent treatment for adults with chronic hepatitis B virus (HBV) infection.
Baxalta, Precision in $1.7 billion CAR T pact
Baxalta has signed a pact with gene-editing group Precision BioSciences to develop a broad series of allogeneic chimeric antigen receptor (CAR) T cell therapies that address major unmet need in multiple cancers.
EU approves Shield’s anaemia drug for IBD patients
The European Commission has issued a green light for Shield Therapeutics’ Feraccru for the treatment of iron deficiency anaemia (IDA) in patients with inflammatory bowel disease (IBD).
US nod for Pfizer’s once-daily Xeljanz
US regulators have approved an extended-release version of Pfizer’s Xeljanz for the treatment of rheumatoid arthritis (RA), giving patients access to the first once-daily, oral JAK inhibitor for the condition.
CRUK starts trial of Amgen’s cancer immunotherapy
Cancer Research UK has launched a mid-stage trial in England to explore whether an investigational immunotherapy in development at Amgen can unmask cancer cells and thereby facilitate their detection and destruction by the immune system.
MHRA expands use of MSD’s Ezetrol
The Medicines and Healthcare Products Regulatory Agency has approved a new indication for MSD’s cholesterol buster Ezetrol (ezetimibe), expanding its use to include reducing the risk of cardiovascular events in certain patients.
NHS England approves Cancer Drugs Fund plans
The highly controversial Cancer Drugs Fund will be replaced by a new fund controlled by the National Institute of Clinical Excellence from July this year, NHS England has confirmed.
International Clinical Researcher Awards, Early bird tickets out now
The level of engagement in the 2016 International Clinical Researcher of the Year competition has been fantastic, with the number of candidates increasing yet again. The Awards ceremony, held at the luxurious Lancaster London, is a must-attend event for anyone in the pharma industry. Entrants will be informed whether they have made it through to the Finals early next week.
