Novartis: using rare diseases as pathways to treating common diseases

It’s been encouraging to see the big pharma companies focus increasingly on rare diseases over the past year. Novartis particularly stands out as it seems to have adopted rare disease research as a central part of its strategy. Each time I’ve heard someone from Novartis speak, I’ve been impressed by their commitment to understanding the science behind the diseases, not just focusing on the marketing and distribution.

In fact, a report came out in March showing that Novartis, unlike many of the other big pharma companies, is increasing its spending on research and development. Last year, it’s R&D budget went up by 10%.

FierceBiotech.com and CheckOrphan.com have just published an article about how Novartis’s CEO Joe Jimenez took a minute during a recent interview with Fortune to explain the company’s drug development strategy. Rather than go after a blockbuster indication, Novartis scientists are more focused on pathways.

By pursuing a particular disease pathway with a drug designed to treat a rare disease, Novartis improves its shot at gaining an approval from a tougher set of regulators on both sides of the Atlantic, the Novartis CEO said. Then once the pharma giant has that initial approval it expands on the treatment label by adding a more common disease that is affected by the same pathway.

‘A good example of this is Ilaris, which is for a rare set of autoimmune diseases called cryopyrin-associated periodic syndrome,’ Jimenez told Fortune magazine. ‘I think there are only 6,000 patients globally with this disease. But the mechanism, the pathway of this disease, is the same pathway that is prevalent in gout. And there are 3 million patients with acute gout. We have developed the drug in gout and we’re awaiting FDA approval for this compound.

‘This is a perfect example of how you would have multiple indications for the same drug and that’s the way the drug would become, let’s say, a blockbuster. Not that Ilaris will become a blockbuster but there will be multiple indications and multiple diseases through this fundamentally different approach.’

From our point of view, AKU is an extreme model of osteoarthritis, and we’re pretty convinced that discoveries in AKU will have significant impacts on OA in the future. In fact, we’re doing a symposium at the Liverpool Medical Institute on Friday 20 May all about this. I’ll be posting up more information about this soon in case you’d like to attend.