FDA approves Jascayd as first new IPF treatment in over 10 years
Boehringer's oral therapy nerandomilast aims to slow lung function decline
The U.S. Food and Drug Administration (FDA) has approved nerandomilast, an oral medication developed by Boehringer Ingelheim, to treat adults with idiopathic pulmonary fibrosis (IPF) — when the lung disease has no known cause.
The therapy will be marketed under the brand name Jascayd. According to a press release from the FDA, the decision marks the first approval of a new IPF treatment in more than a decade.
“[This] approval furthers [the] FDA’s longstanding commitment to support treatment options for patients and advances in health care for the American public,” the release stated.
IPF is a chronic disease marked by inflammation and fibrosis, or scarring, in the lungs, which leads to symptoms such as shortness of breath, coughing, and fatigue that tend to get worse over time.
Jascayd is an orally available molecule designed to block the activity of an inflammatory enzyme called phosphodiesterase 4B (PDE4B). This is expected to reduce inflammation and tissue scarring in IPF, thereby slowing or stopping lung function decline.
The medication is available in tablets of 9 or 18 mg, which can be swallowed whole or dispersed in water. The recommended dosage is 18 mg taken twice daily, approximately 12 hours apart, with or without food, according to the FDA.
The agency noted that lower doses may be used in patients who experience intolerable side effects, except if they are taking Jascayd alongside pirfenidone, an approved IPF medication sold as Esbriet and generics.
US approval of Jascayd was supported by data from 2 global clinical trials
The FDA’s approval of Jascayd was mainly supported by data from two global clinical trials.
The first was a Phase 2 study (NCT04419506) that tested the therapy against a placebo in 147 IPF patients. Participants could continue to receive standard IPF medications, namely pirfenidone and Ofev (nintedanib).
After three months of treatment, results showed Jascayd slowed decline in forced vital capacity (FVC), a common measure of lung function based on how much air someone can blow out in a forceful breath.
The second trial was a Phase 3 study called FIBRONEER-IPF (NCT05321069) that enrolled 1,177 adults with IPF. Participants in this study were randomly assigned to receive Jascayd at a dose of 9 or 18 mg, or a placebo, for at least one year. Individuals in this trial were also allowed to continue on pirfenidone and Ofev.
FIBRONEER-IPF met its primary goal of showing that Jascayd was significantly better than the placebo at slowing FVC decline after a year of treatment. Data from the Phase 3 study also indicated that Jascayd may reduce the risk of death for people with IPF.
In both studies, Jascayd’s efficacy was generally similar in patients who were or were not taking other IPF treatments. Only the group taking pirfenidone in the FIBRONEER-IPF trial failed to benefit from the 9 mg dose, the data showed.
Therapy label shows diarrhea, respiratory infections as common side effects
According to the therapy’s prescribing information, common side effects of Jascayd include diarrhea, COVID-19, upper respiratory tract infection, and depression. Additional common side effects are weight loss, decreased appetite, nausea, fatigue, headache, vomiting, back pain, and dizziness. The therapy’s prescribing information does not list any warnings or contraindications.
Boehringer is also developing nerandomilast to treat progressive pulmonary fibrosis, and Phase 3 data have demonstrated the therapy may slow lung function decline and extend survival in that population.
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