IO Frontiers World 2019

European drug makers take the first footsteps into the US gene therapy market

Michael Adeniya

Published on September 1, 2017 by: 



Novartis have made history as the first pharma company to gain approval of a CAR-T product by the FDA and can be crowned definitive winners of what has been an eagerly followed race over recent years.


It’s an exciting time for our colleagues in cell and gene therapy. This truly cements the possibilities of ex vivo gene therapy treatments following Gilead’s purchase of Kite Pharma and recent EMA approvals.

Phacilitate Leaders Europe

Carl June of UPenn, collecting the Translation Pioneer Award at Cell & Gene Therapy World last January. Novartis bought the rights to the Penn treatment for just $20 million up front.

The Swiss drug giant’s product, to be marketed as Kymriah, is a treatment for children and young adults with relapsed acute lymphoblastic leukemia but priced at $475,000 (£367,000) per dose, is one of the world’s most expensive medicines. Will all patients who need this drug be able to access it?

I’d put a bet on it.

As a salesperson, I'm often in the crossfire of "cost vs value" conversations and this is a a great example of it. Innovative drugs deserve innovative pricing and by beating most Wall Street analyst’s predictions, Kymriah does appear to be a relative bargain. Especially when compared to the current standard of care, bone marrow transplants can cost as much as $800k.  Moreover, the clinical results are so impressive with 83% of patients being cancer free after three months.

Stephen Grupp, who treated the first child with a CART put it well when he said, "I don't want to be an apologist for high drug prices in the U.S., but if it's the last treatment they need, that's a really significant one-time investment in their wellness, especially in kids who have a whole lifetime ahead of them."

However, there are some risk factors. The reality of the marketplace for such an unprecedented treatment paradigm could mean several hurdles for patients, payers and practitioners alike. For example, Novartis has said it is working on a system whereby the government pays for the treatment only if patients respond within a month; there are only 32 sites across the US where patients can be treated; it takes 22 days to process and deliver the treatment; the price tag covers the drug only and does not include hospitalization or travel or adjunct therapy costs.

So, time will reveal the true commercial success of Kymriah but, either way, FDA approval is a huge success for the European advanced therapies community too. Not only are Novartis a huge European biopharma but Oxford Biomedica, a UK-based biotech and CMO has an exclusive partnership for the supply of lentiviral vector used in manufacture of the treatment. Speaking to the Guardian, John Dawson, chief executive of the British firm, said: “This innovative immunocellular therapy will offer critically ill patients with an aggressive disease a new treatment option. We look forward to continuing our collaboration with Novartis.”

Oxford Biomedica

Oxford Biomedica and three Novartis leaders are on the agenda for this month’s Phacilitate Leaders Forum in Berlin, I’m personally looking forward to bringing the community together, sharing and developing new strategies and of course celebrating another major milestone in healthcare.

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  • "Great venue to solidify and harmonize on the current challenges facing this sector - in real time. The caliber of the speakers and session contents was premier. “
    Dolores Baksh
    Innovation Leader, GE Healthcare
  • “A great event to learn about the hurdles that are needed to be overcome to get through the clinic.”
    Devin Bridgen
    Scientist, strategic partnerships, SQZ Biotech
  • "It was a great opportunity to share current data and thinking about the development of therapeutics directed at modulating the immune response to cancer."
    Lance Leopold
    Vice President, Head of Oncology Development, Incyte Corporation
  • "Phacilitate provides a unique forum, bringing together research, process development, and commercial leaders on the cutting edge of cell, gene, and immunotherapy. A great conference for anyone wanting a comprehensive view of the field."
    Dr James Trager
    Vice President, Research & Product Development, Dendreon
  • “In the rapidly moving Advanced Therapy market, Phacilitate remains one of the premier events.”
    David James
    CEO, Scinogy