Credit - CGT Catapult
Life-changing new products
from Europe’s pharma industry It’s been a year of ‘firsts’ for the approval of novel classes of drug in Europe, Sarah Houlton reports.
T
he EU has given the green light to several new therapeutics in the past year. The first RNAi product to reach the market, Tegsedi (inotersen) from Ionis and Akcea Therapeutics, is designed to treat hereditary transthyretrin amyloidosis, an inherited, progressive rare disease for which treatment options are limited. The drug is an antisense oligonucleotide. These products are short single strands of a nucleic acid that are designed to bind to the messenger RNA produced by a faulty gene, which blocks it from being able to replicate, and therefore effectively turns it off. Europe’s first cell therapy products were also approved. While potentially life-changing for patients, they come at a significant cost – hundreds of thousands of euros per patient. Kite/Gilead’s Yescarta and Kymriah from Novartis are both CAR-T products designed to treat blood cancers, and manufacturing them represents a huge challenge. 6 Industry Europe
The process is very different from those used to make small molecule drugs or biologic protein products, where large batches are made and distributed. Here, the first step is harvesting T-cells from the blood of individual patients. The cells are then genetically modified using a viral vector to insert a gene that codes for a chimeric antigen receptor, or CAR, which arms the cells to recognise and kill cancerous cells. They are then expanded to make more cells, before being returned to the patient. This means that every patient receives an individually tailored product – and therefore each dose has to be manufactured individually. Both companies are ramping up production capability in anticipation of product launches around Europe. Novartis will make the cells in-house in Switzerland plus at CellforCure in France and the Fraunhofer Institute in Germany, while Kite has a facility in the Netherlands.
Altering the cells is not the only manufacturing challenge – the virus used to insert the new gene into the cells needs to be made too. There is a real shortage of capacity for manufacturing viruses, which are also used in gene therapy, and in May the UK government’s Cell & Gene Therapy Catapult opened an incubator in Stevenage for the scale-up and manufacture of these products. It has six independent cleanroom modules for biotech companies to use, and the addition of further modules is already being planned. Four companies are already in situ – Adaptimmune and Freeline making viral vectors, and Autolus and Cell Medica producing cell therapies. The facility is adjacent to a cryo storage and distribution centre, run by Fisher Scientific. The UK government’s Industrial Strategy is leading to further investment in pharma manufacturing, too, not least the Medicines Manufacturing Innovation Centre that is being
