Therapy Areas: Devices
Nobelpharma's inhaled use of Leukine receives Japanese regulatory approval to treat aPAP
4 April 2024 -

US-based Partner Therapeutics, Inc. (PTx), an integrated biotechnology company, announced on Wednesday that its Japan-based partner Nobelpharma has received approval for the inhaled use of Leukine (sargramostim), branded in Japan as Sargmalin, from the Japanese Pharmaceuticals and Medical Device Agency (PMDA).

Leukine is intended for the treatment of Autoimmune Pulmonary Alveolar Proteinosis (aPAP). PTx licensed rights for certain indications in Japan to Nobelpharma in 2022.

Leukine is a glycosylated recombinant human granulocyte-macrophage colony-stimulating factor (rhu GM-CSF) produced by recombinant DNA technology in yeast. Leukine is not approved outside Japan for aPAP or as an inhalation therapy. Partner Therapeutics will manufacture Sargmalin for Nobelpharma at its manufacturing facility in Lynnwood, WA.

The Sargmalin approval is based on data from the PAGE trial, a phase 2 multicentre, randomised, double-blind, placebo-controlled study to evaluate Leukine in 64 patients with mild to moderate aPAP led by sponsor-investigator Koh Nakata MD, PhD, project professor, Division of Pioneering Advanced Therapeutics, Center for Medical Innovation of Niigata University Medical Dental Hospital.