U.K. Awards Promising Innovative Medicine Designation to Savara’s Molgramostim for aPAP

The UK’s Medicines and Healthcare Products Regulatory Agency granted Promising Innovative Medicine (PIM) designation to Savara’s molgramostim for the treatment of aPAP, a rare lung disease with no approved pharmaceutical treatments.

PIM designation is an early indication that molgramostim is a promising candidate for the UK Early Access to Medicines Scheme (EAMS), a program that provides an opportunity for important therapies to be used in UK clinical practice in parallel with the later stages of the regulatory process. Medicines with a positive scientific opinion could be made available to UK patients 12 to 18 months before formal marketing authorization is granted.

“This PIM designation was granted on the basis of molgramostim nonclinical and clinical data and further reinforces the potential of molgramostim to provide significant benefit in the treatment of aPAP,” said Matt Pauls, chairman and CEO of Savara. “We look forward to working with the MHRA, on both the Innovation Passport and PIM designations, to advance this program and leverage the potential benefits of the EAMS program.”

Autoimmune pulmonary alveolar proteinosis (aPAP) is a rare lung disease that belongs to a family of distinct rare lung diseases collectively known as pulmonary alveolar proteinosis (PAP). aPAP represents about 90 percent of all patients with PAP. While aPAP can affect people of any age, race or sex, onset occurs most frequently in people between the ages of 30 and 40. PAP is characterized by the build-up of surfactant in the alveoli, or air sacs, of the lungs. The surfactant consists of proteins and lipids and is an important physiological substance that lines the inside of the alveoli to prevent the lungs from collapsing. The root cause of aPAP is an autoimmune response against GM-CSF, a naturally occurring protein in the body. Pulmonary macrophages need to be stimulated by GM-CSF to function properly, but in aPAP, GM-CSF is neutralized by antibodies against GM-CSF, rendering the macrophages unable to perform their tasks, including the clearance of surfactant from the alveoli. In aPAP, the feeling of having trouble breathing is the most common symptom. People with aPAP can also experience chronic cough, fatigue, sputum production, reduced ability to exercise and episodes of fever due to underlying pulmonary infections. There are currently no approved pharmaceutical treatment options for aPAP.

Molgramostim nebulizer solution, a novel inhaled biologic, is an inhaled granulocyte-macrophage colony-stimulating factor (GM-CSF) in phase 3 development for aPAP.

On June 15, 2022, molgramostim was awarded Innovation Passport designation by MHRA. In 2019, molgramostim was granted Fast Track and Breakthrough Therapy designation by the U.S. Food and Drug Administration for the treatment of aPAP, and in 2012 and 2013 was granted Orphan Drug designation for the treatment of aPAP in the United States and European Union, respectively.

For the MHRA to grant a PIM designation, medicinal products must meet the following criteria: the condition should be life-threatening or seriously debilitating with a high unmet need for which there is no method of treatment, diagnosis or prevention available or where existing methods have serious limitations; the medicinal product is likely to offer a major advantage over methods currently used in the UK; and the potential adverse effects of the medicinal product are likely to be outweighed by the benefits, allowing for the reasonable expectation of a positive benefit risk balance.

Author: Rare Daily Staff