Shervin Assassi, MD, Director, Division of Rheumatology at McGovern Medical School, discusses the approval of Jascayd (nerandomilast) tablets for treatment of patients with progressive pulmonary fibrosis (PPF).
PPF is a subcategory of interstitial lung disease (ILD) characterized by progressive scarring of the walls of the air sacs in the lung. PPF is not a specific diagnosis, but a disease behavior that can occur in many types of ILD. Signs and symptoms include shortness of breath, cough, fatigue, depression, and anxiety.
The U.S. Food and Drug Administration (FDA) has approved nerandomilast tablets for the treatment of adults with PPF. Nerandomilast is the first preferential PDE4B inhibitor with immunomodulatory and antifibrotic effects. The approval is based on results from the pivotal phase III FIBRONEER-ILD clinical trial.
The primary endpoint of the trial was the absolute change from baseline in Forced Vital Capacity (FVC) at week 52. Nerandomilast demonstrated significantly smaller reduction in FVC from baseline compared to placebo. The adjusted mean decline in absolute change from baseline in FVC in patients receiving nerandomilast 18 mg or 9 mg was -86 mL and -69 mL, respectively, compared to -152 mL in the placebo group.
The key secondary composite endpoint was the time to the first occurrence of any of the components of the composite endpoint over the blinded duration of the trial: acute ILD exacerbation, hospitalization for respiratory cause, or death. Overall, there was no statistically significant treatment difference in the hazard ratio for the nerandomilast 18 mg or 9 mg groups compared to placebo for the key secondary composite endpoint.
Further results for nerandomilast 18 mg in respect to the key secondary endpoint components, from an exploratory analysis, were also published. Treatment showed a significant reduction in risk of acute ILD exacerbations over the blinded trial duration compared to placebo and a reduction in the risk of hospitalization for respiratory cause over the blinded trial duration compared to placebo.
Additionally, a prespecified analysis of overall survival at the end of the trial showed a trend in favor of nerandomilast. The hazard ratios for all-cause mortality until the end of the trial for nerandomilast 18 mg and 9 mg compared to placebo were 0.51 and 0.51, respectively.
Overall, nerandomilast was well-tolerated in patients with PPF. Diarrhea was the most common adverse reaction associated with treatment and treatment discontinuation and occurred most frequently in patients treated with 18 mg or 9 mg nerandomilast with background antifibrotic therapy, versus patients receiving placebo and background antifibrotic therapy. In most patients treated with nerandomilast, diarrhea was of mild to moderate intensity and generally occurred within the first 3 months of treatment.
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To learn more about PPF, ILD, and other rare lung conditions, visit https://checkrare.com/diseases/lung-diseases/
