Phase 1 Clinical Trial of Dornase Alfa Biosimilar to Begin March 2018

March 5, 2018
The Center for Biosimilars Staff

The proposed biosimilar references Genentech’s Pulmozyme, a biologic first approved in the early 1990s in both the European Union and the United States. Pulmozyme is used in conjunction with other therapies to improve pulmonary function in patients with cystic fibrosis.

JHL Biotech has announced that the Dutch Healthcare Authority has approved a clinical trial application for the company’s proposed biosimilar of the cystic fibrosis drug dornase alfa. JHL will begin the phase 1 clinical trial of JHL1922 in the Netherlands beginning this month, March 2018.

The proposed biosimilar references Genentech’s Pulmozyme, a biologic first approved in the early 1990s in both the European Union and the United States. Pulmozyme is used in conjunction with other therapies to improve pulmonary function in patients with cystic fibrosis. A recombinant human deoxyribonuclease 1 enzyme that selectively treats cystic fibrosis based on genetic markers, dornase alfa has also been shown to reduce the risk of respiratory tract infections that require parenteral antibiotics. The biologic is administered daily using a rapid nebulizer system or a jet nebulizer connected to an air compressor.

If JHL1922 is eventually approved, a cheaper biosimilar could expand access to dornase alfa treatment for the approximately 100,000 patients with cystic fibrosis worldwide. According to JHL’s own estimates, the annual cost of Pulmozyme per patient ranges from $12,000 to $40,000.

However, just as a potential biosimilar nears for this drug that has become a mainstay in treating the symptoms of cystic fibrosis, newer therapies that target the underlying cause of the disease are reaching the market; drug maker Vertex announced in February that the FDA had approved its tezacaftor/ivacaftor and ivacaftor combination (Symdeko) in patients with at least 1 genetic mutation that is responsive to tezacaftor plus ivacaftor. The newly approved drug addresses the trafficking and processing defect of a gene that causes cystic fibrosis.

While the therapy offers a new treatment option for patients, it comes as a cost far higher than that of dornase alfa; Vertex’s drug carries a list price of $292,000 per patient per year. Vertex is also the sponsor of other high-cost cystic fibrosis therapies, including the $311,000-per-patient-per-year ivacaftor (Kalydeco), and multiple investigational drugs currently in phase 3 studies.