March 26, 2024
Nobelpharma Co., Ltd.
“Sargmalin® for inhalation 250 µg” Approved for
Autoimmune Pulmonary Alveolar Proteinosis
Nobelpharma Co., Ltd. (Headquarters: Chuo-ku, Tokyo, Managing Director & CEO:Jin Shiomura) announced today that “Sargmalin® for inhalation 250 µg” has been
approved for the indication of autoimmune pulmonary alveolar proteinosis (this“Product”).
We are deeply grateful for all of the efforts of the principal investigator, Dr. KohNakata, University Medical and Dental Hospital Advanced Clinical Research Center
Specially Appointed Professor, and many physicians participated in the investigatorinitiated studies, as well as for the cooperation of the patients in the clinical studies andthe assistance from Japan Agency for Medical Research and Development (AMED) andNational Institute of Biomedical Innovation, Health and Nutrition, owing to which theProduct was approved.
This Product is a “sargramostim (genetically modified)” based inhalation with
granulocyte-macrophage colony-stimulating factor (GM-CSF), one of the cytokine
having a promoting effect for proliferation and differentiation of myeloid precursor,produced with fermentum.

This Product has been in the US market since 1991 as an

injectable drug for neutrophil recovery following induction chemotherapy and myeloid
cell recovery following peripheral stem-cell transplantation in patients with acute
myeloid leukemia on the grounds that it increases and functionally activates neutrophils,
eosinophils and monocytes; however, it is not approved for the indication of
autoimmune pulmonary alveolar proteinosis nor as an inhalation.
Autoimmune pulmonary alveolar proteinosis is developed as the hyperproduced antiGM-CSF autoantibody blocks mature alveolar macrophage from decomposing waste
products including alveolar surfactant. There have been no drugs approved in any
country for autoimmune pulmonary alveolar proteinosis and only established treatment
is segmental lung lavage or whole lung lavage that removes accumulated waste products
by injecting saline into lung under general anesthesia.

While post-operative rapid symptomatic improvement can be expected with whole
lung lavage, there exist clinical challenges of being highly invasive and requiring
hospitalization as well as the therapeutic opportunities limited to facilities withexperienced specialists.
This Product, as inhaled, acts directly on alveolar macrophage to accelerate its
maturation and promotes decomposition of the waste products of alveolar surfactant bysuch matured macrophage, leading to improvement in lung functions.
The inhalation therapy of this Product is less invasive compared to segmental andwhole lung lavage and we are confident this will provide a new treatment option for theclinical practice in addition to the existing treatment.
In Japan, autoimmune pulmonary alveolar proteinosis has become a designatedintractable disease since 2015 and the number of patients is expected to be 730 to 770.
We are gratified the approval of this Product will assist the treatment of autoimmune
pulmonary alveolar proteinosis.
We are committed to continue contributing to society by providing critical but
neglected pharmaceuticals and medical devices.

[Contact for Inquiries]
Noboru Kudo, Head of Communications
Nobelpharma Co., Ltd.
1-17-24, Shinkawa, Chuo-ku, Tokyo 104-0033
Tel: 03-6670-3800