CAR-T before the bourse: Kymriah OK will ease fundraising but price an issue says ISCT

By Gareth Macdonald contact

- Last updated on GMT

Kymriah OK will ease fundraising but price an issue says ISCT
US approval for Novartis’s CAR-T cancer treatment Kymriah will help cell and gene therapy firms attract the investors they need to fund development according to International Society for Cellular Therapy (ISCT).

The ISCT – which represents clinicians, researchers, technologists and industry partners – made the comment a few day​s after Kymirah – also known as ​CTL019 (tisagenlecleucel) – became the first cell therapy licensed by the US Food and Drug Administration (FDA).

Miguel Forte, MD, PhD, Chief Commercialization Officer, ISCT told us “development of medicinal products always come with a risk attached. Cell therapy, in view of the complexity of a living medicinal product comes with higher potential but also higher risk.

For investors the approval of this product makes the success a reality and gives a sense and understanding of the risk. Brings cell therapy closer to traditional drug development. This better understanding, the risk mitigation and the multiple competing opportunities will enable the investors to support further products.​”

This was echoed by Bruce Levine, a member of ISCT Commercialization and Immuno & Gene Therapy Committees, who told us “the approval of the well-developed and well supported CART Novartis product shows that it is possible to take such a product to market. This makes the development of cell therapy product real and more in line with traditional expectations.​”

The ISCT also predicted support industries – contract manufacturing organisations (CMOs), contract research organisations (CROs), equipment suppliers and logistics firms will invest to increase capacity.

Work to do

However, while it welcomed Kymirah’s approval as positive, the ISCT cautioned industry and investors that there is still more to do to ensure that such high priced therapies – the Novartis product is going to cost $475,000 (€400,000) ​– are available to patients.

According to Forte “The price appears high but cost effective in comparison to averted costs and independent pharmacoeconomic studies. The value of long term benefit is very significant in patients with relapsing and remitting disease with no alternatives.

The long term benefit requires a new approach to the payment and reimbursement of these therapies in line with the affordability of these therapies to the health care systems globally despite geographical variations​.”

The price of Kymirah and other cell therapies should be assessed in terms of the overall disease burden according to Levine.

He told us “the cost averted is not only the cost of other therapies, including transplant, but the costs of the associated treatments, mitigation of short and long term toxicities, cost upon the families in missed work, stress, depression, PTSD, etc. 

Additionally, competition in CAR T cells amongst the 40 odd companies developing engineered T cells or NK cells should put pressure on developing more efficient manufacturing, analytics, logistics and support solutions​” Levine said.

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