Within the United Kingdom, there’s a program titled the Innovative Licensing and Access Pathway (ILAP), which is meant to give patients earlier access to developing treatments. The first step to entering this program is called the Innovation Passport – and an investigational treatment called molgramostim was recently awarded it. This means that it has the chance to move through the regulatory process at a faster pace, eventually making it to autoimmune pulmonary alveolar proteinosis (aPAP) patients sooner.
The Medicines and Healthcare Products Regulatory Agency (MHRA) is responsible for granting this designation, and their decision follows that of the European Union and US FDA to award the Orphan Drug designation back in 2013 and 2012, respectively. In addition, the FDA has also granted its Breakthrough Therapy and Fast Track designation to molgramostim. Hopefully, all of these decisions move molgramostim – a novel inhaled biologic – towards aPAP patients at a faster rate so they can reap the benefits sooner.
You can read the full press release from Biospace.
According to NORD, autoimmune pulmonary alveolar proteinosis is a rare autoimmune disorder of the lung. In fact, it is the most common form of all pulmonary alveolar proteinosis (PAP). Symptoms include shortness of breath upon exertion, difficulty breathing, fatigue, cough, weight loss, and chest pain. In rare instances, patients may cough up blood, experience cyanosis, and see the swelling of the fingertips. As the name suggests, the cause is autoimmune in nature. IgG antibodies block something called the granulocyte-Macrophage Colony Stimulating Factor (GM-CSF) effect, which includes a protein that is necessary to clear surfactants off of alveolar macrophages. When this process is blocked, the surfactants are able to build up and cause the characteristic symptoms. An additional risk factor is age, as most patients are between the ages of 20 and 50.