There has been little innovation for many years in the treatment of some of our most common respiratory diseases.
New medicines for COPD have become more accessible, and convenient for the patient, but with little prospect of substantially improving on the current management and treatment of this serious disease.
COPD is a progressive respiratory disease for which there is no cure.
COPD damages the airways and the lungs and leads to shortness of breath, impacting a person’s ability to perform daily activities. Chronic inflammation is particularly prominent in the airways of COPD patients. COPD includes chronic bronchitis and emphysema. According to the World Health Organization, COPD is the third leading cause of death globally, with 210 million people worldwide suffering from the disease. It is estimated that there are 24 million people with COPD in the United States, only half of whom have been diagnosed. Total annual direct medical costs relating to COPD in the United States were estimated to be $32 billion in 2010 and are projected to rise to $49 billion in 2020. Global sales of drugs currently indicated for COPD are estimated to be $10.6 billion in 2016, growing to $15.6 billion in 2019.
COPD patients are commonly treated with bronchodilators, which seek to relieve airway constriction and make it easier to breathe, and corticosteroids, which seek to reduce lung inflammation. For more severe patients with recurrent exacerbations, an oral formulation of an anti-inflammatory PDE4 inhibitor is commonly used as treatment. Despite the wide availability of these therapies, many COPD patients continue to suffer acute periods of worsening symptoms, or exacerbations, which are estimated to cause approximately 1.5 million emergency department visits, 687,000 hospitalizations and 129,000 deaths per year in the United States alone. Furthermore, current therapies have not demonstrated an ability to change the progressive decline in lung function or reduce the mortality associated with COPD. There is an urgent and unmet medical need for new and more effective treatments for COPD to reduce the number and burden of symptoms, reduce the exacerbations and establish a consistent and durable treatment response.
Cystic fibrosis is the most common fatal inherited disease in the United States and Europe.
CF causes impaired lung function and is commonly associated with repeat and persistent lung infections that result in frequent exacerbations and hospitalizations. There is no cure for CF and the median age of death for CF patients is 37 years. CF is considered a rare, or orphan, disease by both the FDA and EMA, the respective United States and European regulatory authorities. According to the Cystic Fibrosis Foundation, more than 30,000 people in the United States and more than 70,000 people worldwide are living with CF and approximately 1,000 new cases of CF are diagnosed each year. CF patients require lifelong treatment with multiple daily medications, frequent hospitalizations and ultimately lung transplants in selected patients. The quality of life for CF patients is compromised as a result of spending significant time on self-care every day and frequent outpatient doctor visits and hospitalizations. CF patients take an average of seven medications daily. The FDA and the EMA provide incentives for sponsors to develop products for orphan diseases.