A gene therapy developed by Spark Therapeutics Inc for an inherited retinal disease has received a paediatric designation from the US Food and Drug Administration. The decision is the agency’s way of highlighting the treatment’s potential for children and young adults.
Ablynx NV has secured a collaboration with Sanofi SA to develop products directed at immune-mediated inflammatory diseases. The deal, which includes an upfront payment of €23 million, is the latest endorsement of the Belgian company’s therapeutic protein platform.
Novartis expects that the decline in sales and operating profit that the company experienced in 2016 will be mostly halted in 2017. This year, it expects sales will be largely unchanged and the operating profit will be flat or slightly lower. Growth is expected to resume next year. This follows a productivity drive and the successful launch of new products.
The US Food and Drug Administration has approved Nerlynx (neratinib), a new drug for lowering the risk of recurrent breast cancer in patients who have been treated with a regimen that includes trastuzumab, the targeted therapy for HER2-positive breast cancer.
An advisory committee to the US Food and Drug Administration has unanimously recommended that the agency approve the first engineered T cell therapy for cancer CTL019. The vote is not binding on the FDA, but the agency usually follows the recommendations of its advisory committees.
Results from a Phase 3 trial of emicizumab, an experimental treatment for haemophilia A, were published online in the New England Journal of Medicine on 10 July. They showed the treatment achieved a clinically meaningful reduction in bleeding episodes in patients with antibodies to factor VIII replacement therapy.
There are multiple strategies underway in oncology to exploit new knowledge about the immune system and destroy tumours. Many of these involve building compounds de novo. But every once in a while a company breathes new life into an existing molecule.
Sanofi has taken steps to broaden its vaccine portfolio with the planned acquisition of Protein Sciences Corp of Meriden, Connecticut, US which has a protein-based influenza vaccine recently approved for the US market.
Shire Plc is advancing a candidate gene therapy acquired from its 2016 purchase of Baxalta, with plans to take the product into a first human trial in patients with haemophilia A. The product is a factor VIII gene therapy delivered in an adeno-associated virus (AAV) vector.
The first new drug for sickle cell disease in nearly 20 years has been approved by the US Food and Drug Administration. The treatment, Endari (L-glutamine oral powder), lowers oxidant damage to red blood cells thereby reducing complications of the disease.