FDA approves Jascayd for adults with progressive pulmonary fibrosis
Oral therapy shown to slow lung function decline in PFF patients in trial
Written by |
The U.S. Food and Drug Administration (FDA) has approved Jascayd (nerandomilast), taken as oral tablets, as a treatment for adults with progressive pulmonary fibrosis (PPF), in which lung scarring worsens over time.
“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,” Shashank Deshpande, chairman of the board of managing directors and head of human pharma at Boehringer Ingelheim, which sells Jascayd, said in a company press release announcing the FDA’s decision.
“My gratitude goes to patients, investigators and our teams whose dedication made this milestone possible,” Deshpande added. “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.”
The recommended dosage of the medication is 18 mg twice daily, taken approximately 12 hours apart, with or without food.
Scott Staszak, president and CEO of the Pulmonary Fibrosis Foundation, said this approval “is a welcomed milestone for the community.”
“People living with progressive pulmonary fibrosis often carry a heavy burden that others don’t always see,” Staszak said. “A progressive disease condition process like PPF can worsen lung function quickly, and patients have been eagerly awaiting additional treatment options.”
Approval is second for oral therapy in US this year
Pulmonary fibrosis refers to scarring in the lungs, which makes it harder for them to function properly. PPF refers to lung scarring that gets progressively worse over time.
The FDA’s decision, which makes Jascayd the only approved PPF therapy in its class, comes just a few months after the agency approved the medication for the treatment of idiopathic pulmonary fibrosis (IPF). In IPF, the disease’s underlying cause is unclear.
Jascayd works to block an inflammatory enzyme called phosphodiesterase 4B (PDE4B), which is expected to help limit lung inflammation and scarring.
Shervin Assassi, MD, director of rheumatology at the McGovern Medical School at UTHealth Houston in Texas, noted that PPF “is linked to underlying clinical ILD [interstitial lung disease] diagnoses including autoimmune ILDs — which can be caused by disorders like rheumatoid arthritis or systemic sclerosis — as well as hypersensitivity pneumonitis.” Other conditions can also play a role, Assassi noted.
“These underlying conditions often lead to the lungs being overlooked, yet lung scarring may lead to [a] debilitating and irreversible impact on lung function,” Assassi said. “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 Jascayd.”
The FDA’s approval of Jascayd for PPF was based mainly on data from the Phase 3 FIBRONEER-ILD trial (NCT05321082), which enrolled more than 1,000 people with the disease. The study tested two doses of the medication — 9 mg or 18 mg, both given twice daily — against a placebo. Most of the trial’s participants were already taking the pulmonary fibrosis treatment Ofev (nintedanib).
The trial met its main goal, showing that, after one year, Jascayd was better than the placebo at slowing the decline in a standard measure of lung function known as forced vital capacity. Data from the trial also demonstrated that Jascayd significantly reduced the risk of acute disease flares, and tended to reduce rates of hospitalizations for respiratory problems. A pooled analysis that used data from FIBRONEER-ILD and a separate trial in IPF, called FIBRONEER-IPF (NCT05321069), showed that Jascayd appears to reduce the risk of death in people with either condition.
According to its prescribing information, the most common side effects of Jascayd include diarrhea, COVID-19, and upper respiratory tract infection. Depression, weight loss, decreased appetite, nausea, fatigue, headache, vomiting, back pain, and dizziness also can occur. The prescribing information does not list any contraindications or warnings. In FIBRONEER-ILD, diarrhea was the most common side effect associated with treatment discontinuation, and occurred most frequently in patients treated with Jascayd at 18 mg (4%) or 9 mg (3%) when combined with background therapy.
