How has Ultragenyx ramped up its orphan drugs pipeline?

In Clinical Development, Partnering & Investment, Regulation & Government by André SingerLeave a Comment

On the very first edition of the World Orphan Drug Congress USA in 2011 Emil Kakkis – then President of the Kakkis Every Life Foundation – spoke about the next steps to accelerate biotechnology for rare diseases. Looking now at how fast Ultragenyx, the biotech founded by Emil, is growing their rare disease pipeline, we can now say that the approaches he presented back in 2011 were successful. Ultragenyx’s pipeline includes four very promising mid-stage orphan drug candidates and multiple programs on early and pre-clinical stages also benefiting from a fast development pace. What has the company been doing to expedite its orphan drug development programs? Is is just assertive science? Or strategic acquisitions and partnerships? Maybe both? Well, this week’s partnership between Ultragenyx and Japanese drug maker Kyowa Hakko Kirin might weight for a combination of the factors described above. Ultragenyx will be co-developing an antibody designed to treat X-linked hypophosphatemia which is now wrapping a Phase I/II study at KHK. “The collaboration combines KHK's broad antibody-based discovery, manufacturing and development capabilities with our expertise in the clinical development of novel therapeutics for rare genetic diseases” said Emil Kakkis in a statement. While Ultragenyx steps in to be responsible for the development of the drug, both companies share its costs as they target to launch it for the US, Canada, Europa and Japanese markets. Collaborations like this might appear often, but aligning terms and responsibilities is usually a very complex effort. At the World Orphan Drug Congress USA, lots of panel discussions and case studies have addressed the relevance of enhancing multi-stakeholder collaborations, including regulators, patient groups, academia and payers, to a better orphan drug development strategy.

Emil Kakkis – then President of the Kakkis Every Life Foundation spoke about the next steps to accelerate biotechnology for rare diseases at the first World Orphan Drug Congress USA in 2011. Talk about practicing what you preach; Ultragenyx, the biotech founded by Emil, is fast-growing their rare disease pipeline.

Ultragenyx’s pipeline includes four very promising mid-stage orphan drug candidates and multiple programs on early and pre-clinical stages also benefiting from a fast development pace.

What has the company been doing to expedite its orphan drug development programs? Is is just assertive science? Or strategic acquisitions and partnerships? Maybe both?

Well, this week’s partnership between Ultragenyx and the Japanese drug maker Kyowa Hakko Kirin might hold the weight for a combination of the factors described above. Ultragenyx will be co-developing an antibody designed to treat X-linked hypophosphatemia which is now wrapping a Phase I/II study at KHK. “The collaboration combines KHK‘s broad antibody-based discovery, manufacturing and development capabilities with our expertise in the clinical development of novel therapeutics for rare genetic diseases” said Emil Kakkis in a statement. While Ultragenyx steps in to be responsible for the development of the drug, both companies share its costs as they target to launch it for the US, Canada, Europa and Japanese markets.

Collaborations like this might appear often, but aligning terms and responsibilities is usually a very complex effort. The World Orphan Drug Congress USA hosts panel discussions and case studies addressing the relevance of enhancing multi-stakeholder collaborations. This includes collaboration not just between drug-makers but with regulators, patient groups, academia and payers as well.

Here’s a good article that describes details of the abovementioned partnership.

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