Pharming and Novartis Collaborate to Develop and Commercialize CDZ173 for Activated PI3K Delta Syndrome (APDS)

On August 14, 2019, VCBeat (WeChat ID: vcbeat) learned from foreign media reports that biopharmaceutical company Pharming Group (Pharming) announced an agreement with Novartis to jointly develop and commercialize CDZ173, a therapeutic drug for immunodeficiency disorders. Upon completion of this collaboration, Pharming will obtain the exclusive license for the drug, holding its sole distribution rights.

CDZ173 is a highly potent small-molecule inhibitor of phosphoinositide 3-kinase delta (PI3Kδ), primarily indicated for the treatment of activated PI3K delta syndrome (APDS). Novartis has currently completed all clinical trials for this drug and will next explore additional indications for CDZ173 while submitting a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA). Pharming will collaborate with Novartis to complete the NDA process; upon approval, Pharming will commercialize CDZ173 leveraging its established presence in European and American markets. The drug was expected to be launched in 2021.

APDS is a primary immunodeficiency disorder caused by mutations in the PIK3CD gene, inherited in an autosomal dominant pattern. Mutations in the PIK3CD gene lead to hyperactivation of PI3Kδ, resulting in abnormal differentiation of cells involved in immune responses and consequently causing immunodeficiency in patients. APDS typically presents at an early age, with main clinical manifestations including recurrent respiratory infections, hepatosplenomegaly, lymphadenopathy, and viremia caused by cytomegalovirus or Epstein-Barr virus. Some pediatric patients may also develop autoimmune phenomena and lymphoma. This is an extremely rare disease, with a global incidence of approximately one in a million.

Pharming, headquartered in the Netherlands, is a publicly listed company specializing in biotechnology and pharmaceuticals. The company primarily focuses on developing innovative therapies for rare diseases, providing patients with safe and effective treatment options. Pharming possesses a GMP-certified technology platform for the production of human recombinant proteins. Compared to other cell line-based production methods, this platform not only enables large-scale manufacturing of high-quality recombinant proteins but also significantly reduces production costs.

Pharming’s flagship drug, Ruconest, is the first recombinant C1 esterase inhibitor and was approved by the FDA for market launch in July 2014. The drug is indicated for the treatment of acute attacks in adult patients with hereditary angioedema (HAE), and regulatory approval for its use in pediatric HAE patients is currently underway. Furthermore, Pharming plans to explore additional indications for Ruconest.

Pharming has established a long-term partnership with the China State Institute of Pharmaceutical Industry (CSIPI) to jointly develop new products. CSIPI will oversee and supervise Pharming’s drug development processes and provide corresponding financial support.

Novartis, founded in 1996 and headquartered in Basel, Switzerland, is a multinational corporation in the pharmaceutical and healthcare industry. The company boasts a diversified business portfolio, developing innovative patented drugs, eye care products, generic medicines, vaccines, and diagnostic equipment, among other products, and holds leading positions worldwide in multiple fields. As one of the top three pharmaceutical companies globally, Novartis operates in more than 150 countries and regions and employs hundreds of thousands of people. The company was listed on the New York Stock Exchange in May 2000 under the ticker symbol NVS.

Sijmen de Vries, CEO of Pharming, stated, “The development and commercialization of CDZ173 will help Pharming expand its market; this collaboration can be considered a significant milestone. We are highly interested in the characteristics and mechanism of action of this drug, which provides new treatment options for patients with APDS. We look forward to completing the development of CDZ173 together with Novartis.”

(Compiled by Xu Xiaoxue)