New medicine for two types of pulmonary fibrosis
Jascayd slowed loss of lung function in patients with idiopathic or progressive pulmonary fibrosis
NewsHumanMedicines
EMA has recommended granting a marketing authorisation in the European Union (EU) for Jascayd (nerandomilast) to treat adults with idiopathic pulmonary fibrosis (IPF) or progressive pulmonary fibrosis (PPF), two serious lung diseases that involve progressive and irreversible scarring (fibrosis) of the lung tissue.
There are limited treatment options for people suffering from IPF or PPF. Both conditions can cause severe symptoms, including difficulty breathing, leading to hospitalisation and ultimately death within a few years of diagnosis due to a progressive decline in lung function.
Jascayd has a different mechanism of action from authorised medicines for IPF and PPF and therefore represents a new treatment option for patients with these conditions. The active substance in Jascayd, nerandomilast, belongs to the class of PDE4 inhibitors and preferentially blocks the activity of an enzyme called PDE4B. This enzyme is expressed in the lungs and plays an important role in fibrosis and inflammation. By blocking PDE4B activity, Jascayd reduces both fibrosis and inflammation in the lungs.
EMA’s recommendation is based on data from two main studies involving 2,355 patients with IPF or PPF. In both studies, the main measure of effectiveness was the decline in lung function over the course of 1 year of treatment, measured by forced vital capacity (FVC). FVC is the maximum amount of air a person can breathe out forcefully after taking in a deep breath; FVC is reduced in IPF and PPF and decreases as the patient’s condition gets worse.
Patients received either Jascayd or placebo. Some patients received Jascayd alone and others received it in addition to their existing background treatment for IPF or PPF. At the start of the studies, about 78% of patients with IPF and 44% of patients with PPF were receiving background treatment for their disease.
In both studies, the decline in FVC was significantly less in patients taking Jascayd than in patients taking placebo, indicating that treatment slowed down disease progression.
In the study involving patients with IPF, the average decrease in FVC after 1 year was around 115 ml in patients taking 18 mg Jascayd twice a day and 139 ml in those taking 9 mg Jascayd twice a day, compared with 183 ml in patients taking placebo. In the study involving patients with PPF, these figures were around 99 ml and 85 ml, respectively, compared with around 166 ml for patients taking placebo.
The results from both studies also suggested that treatment with Jascayd reduces mortality.
Jascayd is taken orally and the recommended dose is 18 mg twice a day. The dose may be reduced to 9 mg twice a day if the patient does not tolerate the treatment due to diarrhoea or weight decrease. However, the 18 mg dose should not be reduced in patients who are also taking another fibrosis medicine called pirfenidone, as this medicine reduces the level of nerandomilast in the body.
The most common side effects reported with Jascayd were diarrhoea and weight loss.
The opinion adopted by the CHMP is an intermediary step on Jascayd’s path to patient access. The opinion will now be sent to the European Commission for the adoption of a decision on an EU‑wide marketing authorisation. Once a marketing authorisation has been granted, decisions about price and reimbursement will take place at the level of each Member State, taking into account the potential role or use of this medicine in the context of the national health system of that country.
Note: The applicant for Jascayd is Boehringer Ingelheim International GmbH.