The U.S. Food and Drug Administration (FDA) has approved nerandomilast (brand name: Jascayd) for adults with idiopathic pulmonary fibrosis (IPF) — a rare but severe, progressive disease with no cure and limited treatment options. It becomes the first new IPF therapy approved in more than 10 years.
“The FDA approval of nerandomilast is exciting news for people living with idiopathic pulmonary fibrosis and their caregivers,” said Scott Staszak, president and CEO of the Pulmonary Fibrosis Foundation, in a statement. “There has been a long-standing need for new treatment options for IPF within our community, and nerandomilast provides an important addition to the care landscape.”
Nerandomilast previously demonstrated efficacy and safety in the phase 3 randomized, double-blind, placebo-controlled FIBRONEER-IPF trial. The primary endpoint was the absolute change from baseline in forced vital capacity (FVC) at week 52. Results showed that participants who took nerandomilast 18 mg and 9 mg had a significantly smaller FVC decline compared to those who took the placebo.
The drug was also evaluated in a smaller phase 2 trial that included 147 patients with or without previous antifibrotic treatments (nintedanib or pirfenidone). Patients who took nerandomilast at 18 mg twice daily experienced a FVC decline of 91 mL at 12 weeks.
Nerandomilast is an oral, preferential inhibitor of phosphodiesterase 4B (PDE4B). According to the drug’s manufacturer, Boehringer Ingelheim, this represents a novel mechanism of action that exerts both antifibrotic and immunomodulatory effects, thereby slowing the decline in lung function in IPF patients. The recommended dosage is 18 mg orally twice a day, approximately 12 hours apart. The amount may be reduced for intolerability to 9 mg twice daily, except in patients also taking pirfenidone.
The most common side effects (≥5%) reported in nerandomilast- and placebo-treated patients were diarrhea, COVID-19, upper respiratory tract infection, depression, decreased weight, decreased appetite, nausea, fatigue, headache, vomiting, back pain and dizziness.
Regulatory submissions for nerandomilast in IPF are also under review in China, Japan and the European Union.
