Potential New Treatment for Bronchiectasis Receives Positive Suggestion
A new drug, Brinsupri (brensokatib), is a step closer to becoming the first approved treatment specifically for bronchiectasis without cystic fibrosis, following a positive recommendation from the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP). The CHMP recommends approval for use in the european Union for patients aged twelve and older who have experienced two or more worsening episodes of the disease in the past year.
Bronchiectasis, a severe chronic lung disease affecting potentially up to three million people in the EU, shares symptoms with cystic fibrosis (CF) but isn’t caused by the same genetic mutations. It can be triggered by factors like respiratory infections, autoimmune diseases, and immunodeficiency. The condition is characterized by a chronic cough, excessive mucus production, and widening of the bronchi, leading to periods of worsening symptoms - typically one to four per year – that contribute to declining lung function, reduced quality of life, and increased mortality. currently, treatment focuses on airway clearance, antibiotics, and anti-inflammatory medications.
Brinsupri is an oral tablet that works by inhibiting the enzyme dipeptidyl peptidase 1. This enzyme plays a role in activating neutrophils, a type of white blood cell. Over-activation of neutrophils contributes to airway damage, mucus overproduction, chronic inflammation, and weakened immune defenses in bronchiectasis patients. By blocking this enzyme, brensokatib aims to interrupt this damaging process.
The recommendation is based on a clinical trial involving 1,767 patients, conducted as a randomized, double-blind, placebo-controlled study. Results showed patients treated with Brinsupri experienced 19.4 percent fewer worsening episodes compared to those receiving a placebo. A substantially higher proportion of patients in the Brinsupri group also remained free of worsening episodes for a year or more.
Common side effects observed during trials included headache, gingivitis, and various skin issues such as hyperkeratosis (thickened skin), dermatitis (skin swelling and irritation), rash, and dry skin.
Brinsupri benefited from the EMA’s PRIority MEdicines (PRIME) program, which provides enhanced support for promising new medicines addressing unmet medical needs. The CHMP also reviewed the request under an accelerated timeline, recognizing the drug’s potential public health benefit.
While the CHMP recommendation is a significant step, the final decision regarding EU approval rests with the European Commission, which generally follows the CHMP’s guidance.
