Novartis licenses small molecule treatment for a genetic ultra-rare disease

By Maggie Lynch contact

- Last updated on GMT

(Image: Getty/rawpixel)
(Image: Getty/rawpixel)

Related tags: Small molecule, Small molecule drugs, Rare disease, Novartis, Genetic testing, License

Novartis enters a licensing and development agreement with Pharming group for ultra-rare disease treatment for more than $20m.

Per the agreement, Pharming will collaborate to develop and commercialize Novartis’ CDZ173, a small molecule phosphoinositide 3-kinase delta inhibitor to treat patients with activated phosphoinositide 3-kinase delta syndrome (APDS).

The specialty pharmaceutical company focused on rare disease treatments, Pharming, will pay Novartis $20m upfront. The drugmaker is eligible to receive regulatory and commercial milestones and double-digit royalties on net sales as well.

Novartis has completed all preclinical and clinical work to date and will continue to run ongoing registration-enabling trial, as well as the current open-label extension study.

With this agreement, Pharming will work with Novartis to complete enrollment of the ongoing trial. Upon approval of the drug candidate, Pharming will commercialize CDZ173 through its existing commercial infrastructure in the US and EU.

Sijmen de Vries, CEO of Pharming stated, “This is a great example of solid science and deep disease understanding coming together to create a real and personalized treatment option for patients with no prospect of treatment.”

An immune deficiency caused by a mutation in the PIK3CD gene, ADPS can cause patients to suffer early cell death and have difficulty reacting to infections. ADPS is an ultra-rare disease with rates of 1-2 per million across the world.

The gene mutation in PIK3CD increases activity in the phosphoinositide 3-kinase delta inhibitor which is a promotor of activity in the immune system, therefore, the cells involved in immune response can fail to be differentiated properly in patients with ADPS.

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