勃林格殷格翰宣布JASCAYD®（nerandomilast）片获FDA批准用于治疗成人进行性肺纤维化

19/12/2025-Boehringer Ingelheim’s JASCAYD® (nerandomilast) tablets has been approved by the U.S. Food and Drug Administration (FDA) for the treatment of progressive pulmonary fibrosis (PPF) in adults.5 This represents a new treatment option in the U.S. for this debilitating lung condition, with JASCAYD being the first and only preferential phosphodiesterase 4B (PDE4B) inhibitor with immunomodulatory and antifibrotic effects approved in this indication.5 The FDA approval is based on results from the pivotal Phase III FIBRONEERTM-ILD clinical trial, the largest clinical trial program in PPF to date.5 Results showed that nerandomilast effectively slowed lung function decline in PPF with similar permanent discontinuation rates to placebo.*

“Progressive pulmonary fibrosis is linked to underlying clinical ILD diagnoses including autoimmune ILDs – which can be caused by disorders like rheumatoid arthritis or systemic sclerosis – as well as hypersensitivity pneumonitis, among other conditions,” said Shervin Assassi, M.D., Prof., Director of Rheumatology, McGovern Medical School, UTHealth Houston. “These underlying conditions often lead to the lungs being overlooked, yet lung scarring may lead to debilitating and irreversible impact on lung function. This can have a detrimental effect on patients’ lives and highlights the need for new treatment options that can help reduce the decline in lung function, as has been observed with nerandomilast.”

The primary endpoint in FIBRONEERTM-ILD was the absolute change from baseline in Forced Vital Capacity (FVC), a measure of lung function, in mL at Week 52.5 Nerandomilast demonstrated a significantly smaller reduction in FVC decline from baseline compared to placebo.5 Specifically, 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, versus -152 mL in the placebo group.5 The respective treatment difference compared with the placebo group was 65 mL (95% CI: 30, 101) and 83 mL (95% CI: 48, 118).5 The most common adverse reactions in patients with PPF treated with nerandomilast were generally consistent with those observed in patients with IPF.5

“Progressive pulmonary fibrosis is a life-threatening condition with a high unmet medical need. The U.S. approval of JASCAYD is an important step forward to help slow lung function decline for people living with PPF, providing a new, well-tolerated treatment option,” said Shashank Deshpande, Chairman of the Board of Managing Directors and Head of Human Pharma at Boehringer Ingelheim. “My gratitude goes to patients, investigators and our teams whose dedication made this milestone possible. We will now work closely with stakeholders to enable access and work tirelessly to ensure patients around the world can benefit from JASCAYD as quickly as possible.”

“People living with progressive pulmonary fibrosis often carry a heavy burden that others don’t always see,” said Scott Staszak, President and CEO of the Pulmonary Fibrosis Foundation. “A progressive disease condition process like PPF can worsen lung function quickly, and patients have been eagerly awaiting additional treatment options. The FDA approval of nerandomilast for PPF is a welcomed milestone for the community.”

Additional FIBRONEER-ILD Data

In the FIBRONEERTM-ILD clinical trial, 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 (up to 109 weeks): acute ILD exacerbation, hospitalization for respiratory cause, or death.5 Overall, there was no statistically significant treatment difference in the hazard ratio (HR) for the nerandomilast 18 mg or 9 mg groups compared to placebo for the key secondary composite endpoint (nerandomilast 18 mg or 9 mg groups, respectively, compared to placebo: HR 0.77 [95% CI: 0.59, 1.01] and HR 0.88 [95% CI: 0.68, 1.14]).5 Further results for nerandomilast 18 mg in respect to the key secondary endpoint components, from an exploratory analysis, include:

• Nerandomilast 18 mg showed a nominally significant reduction in the risk of acute ILD exacerbations over the blinded trial duration (up to 109 weeks) compared to placebo (HR 0.60 [95% CI: 0.38, 0.94]).5

• Nerandomilast 18 mg showed a numerical reduction in the risk of hospitalization for respiratory cause over the blinded trial duration (up to 109 weeks) compared to placebo (HR 0.82 [95% CI: 0.61, 1.11]).5

Additionally, in the FIBRONEERTM-ILD trial, there was an overall survival trend in favor of nerandomilast.5 The HRs for all-cause mortality until the end of the trial (up to 114 weeks) for nerandomilast 18 mg and 9 mg compared to placebo were 0.51 [95% CI: 0.34, 0.78] and 0.51 [95% CI: 0.34, 0.78], respectively.5 These results were not prespecified for multiplicity control.5

Overall, nerandomilast was well-tolerated in patients with PPF.5 Diarrhea was more common in patients using nerandomilast with concomitant nintedanib. In patients taking background nintedanib, diarrhea occurred 49%, 50%, and 37% of patients treated with nerandomilast 18 mg twice daily, nerandomilast 9 mg twice daily, and placebo, respectively. In patients without concomitant use of background nintedanib, diarrhea occurred in 27%, 16%, and 16% of patients using nerandomilast 18 mg twice daily, nerandomilast 9 mg twice daily, and placebo, respectively5.

Diarrhea was the most common adverse reaction associated with treatment discontinuation and occurred most frequently in patients treated with nerandomilast 18 mg or (4%) or nerandomilast 9 mg (3%) with background antifibrotic therapy, versus patients receiving placebo (1%) and background antifibrotic therapy.5 Importantly, in patients not receiving concomitant nintedanib, diarrhea led to treatment discontinuation in 1% of patients treated with nerandomilast 18 mg and in no patients receiving nerandomilast 9 mg or placebo.5  In most patients treated with nerandomilast, diarrhea was of mild to moderate intensity and generally occurred within the first 3 months of treatment.5

There is no ‘Warnings and Precautions’ section in the FDA approved product label.5 

*In FIBRONEER™-ILD, adverse events led to permanent discontinuation of the trial regimen in 10.0% of the nerandomilast 18 mg group, 8.1% in the nerandomilast 9 mg group, and 10.2% in the placebo group.

References

1. Kondoh Y, Inoue Y. Progressive Pulmonary Fibrosis: Current Status in Terminology and Future Directions. Adv Ther. 2025;42(7):2988–3001.

2. Cen Z, et al. Outcomes and predictors of progression in progressive pulmonary fibrosis. Ann Med. 2024;56(1):2406439.

3. Montero IE, Hernandez-Gonzalez F, Sellares J. Epidemiology and prognosis of progressive pulmonary fibrosis: a literature review. Pulm Ther. 2025;11(3):347-363. doi:10.1007/s41030-025-00302-5.

4. Cottin V, et al. The Burden of Progressive-Fibrosing Interstitial Lung Diseases. Front Med (Lausanne). 2022;9:799912.

5. JASCAYD® (nerandomilast). Prescribing Information. Ridgefield, CT: Boehringer Ingelheim Pharmaceuticals, Inc; 202. (Last updated: Dec 2025).

6. Raghu G, Remy-Jardin M, Richeldi L, et al. Am J Respir Crit Care Med. 2022;205(9). DOI: 10.1164/rccm.202202-0399ST.

7. Chaudhuri et al. Respiratory Research 2024; 25:364

8. Wijsenbeek M et al. Current Medical Research and Opinion 2019; 35 (11): 2015-2024