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Novartis sells rights to drug for ultra-rare disease

Pharming Group NV bought the rights to develop Novartis AG's CDZ173, an investigational treatment for an ultra-rare disease that affects the immune system.

Under the collaboration and licensing deal, Novartis will receive an up-front payment of $20 million. Additionally, the company is eligible to receive regulatory and commercial milestone payments, as well as tiered, double digit royalties on net sales.

CDZ173 is being evaluated to treat activated phosphoinositide 3-kinase delta syndrome, or APDS, a disorder caused by a mutation in the PIK3CD gene that leads to the impairment of the immune system. Patients with APDS often have low white blood cells, resulting in a functional inability to prevent infections.

APDS is an ultra-rare disorder that affects about 1 to 2 people per million.

Switzerland's Novartis, which has completed all preclinical and clinical work for CDZ173, will continue a registration-enabling phase 2/3 clinical study that is enrolling patients in the U.S. and Europe, as well as an ongoing open label extension trial for the drug.

Pharming will be responsible for commercializing CDZ173 once it receives regulatory approval in the U.S. and Europe. The Dutch company intends to seek ways to make the treatment available in other global markets.