ALPHA 1 LUNG DISEASE MARKET ANALYSIS

Market News

Recent Developments

New Product Launches & Approvals

• On September 21, 2023, Krystal Biotech Inc., a commercial-stage biotechnology company focused on the discovery, development, and commercialization of genetic medicines to treat diseases with high unmet medical needs, announced that the U.S. Food and Drug Administration (FDA) cleared the Investigational New Drug Application (IND) for KB408 for the treatment of alpha-1 antitrypsin deficiency (AATD).
• On May 16, 2023, Kamada Ltd., a commercial stage global biopharmaceutical company, announced that Swissmedic, the national authorization and supervisory authority for drugs and medical products has granted marketing authorization for Glassia [Alpha-1 Proteinase Inhibitor (Human)] in Switzerland for chronic augmentation and maintenance therapy in adults with clinically evident emphysema due to severe hereditary Alpha-1 antitrypsin deficiency (AATD).
• On May 11, 2023, Grifols, S.A., a leader in plasma-derived medicines and innovative diagnostic solutions, announced it has launched its AlphaID At Home Genetic Health Risk Service (AlphaID At Home), the first-ever free direct-to-consumer program in the U.S. to screen for the genetic risk of alpha1-antitrypsin deficiency (alpha-1).
• In October 2022, Mereo BioPharma Group plc., a clinical-stage biopharmaceutical company, announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for its investigational oral neutrophil elastase inhibitor, alvelestat (MPH-966) as a treatment for AATD-associated lung disease.
• In July 2021, Grifols, S.A., a leading producer of plasma-derived medicines, and OrphanPacific, Inc., a provider of drugs for rare diseases and disorders in Japan, announced the beginning of the commercialization of Lynspad (intravenous infusion 1000mg), an alpha-1 proteinase inhibitor to treat severe alpha-1 antitrypsin deficiency (AATD) in the country.

Acquisition, Collaborations, and Partnerships

• On September 26, 2023, Alfasigma S.p.A, a leading pharmaceutical company, announced the acquisition of Intercept Pharmaceuticals, Inc., a leading biopharmaceutical company in rare and serious liver diseases, to expand Alfasigma S.p.A’s gastrointestinal and hepatology portfolio and its presence in the U.S. market.
• In July 2022, Vertex Pharmaceuticals Incorporated, a pharmaceutical company, announced the acquisition of ViaCyte, a privately-held biotechnology company for alpha-1 lung disease for potential cure of lung diseases
• In December 2022, GSK plc, a pharmaceutical company, announced a collaboration with Wave Life Sciences Ltd., a clinical-stage genetic medicines company, to advance oligonucleotide therapeutics, including Wave Life Sciences Ltd.’s preclinical RNA editing Programme targeting alpha-1 antitrypsin deficiency (AATD), WVE-006. The discovery collaboration has an initial four-year research term. It combines GSK plc’s unique insights from human genetics, as well as its global development and commercial capabilities, with Wave Life Sciences Ltd’s proprietary discovery and drug development platform, PRISMTM.
• In October 2020, Takeda Pharmaceutical Company Limited, a pharmaceutical company, announced a collaboration with Arrowhead Pharmaceuticals Inc., a pharmaceutical company, to develop ARO-AAT, a Phase 2 investigational RNA interference (RNAi) therapy in development to treat alpha-1 antitrypsin-associated liver disease (AATLD). ARO-AAT is a potential first-in-class therapy designed to reduce the production of mutant alpha-1 antitrypsin protein, the cause of AATLD progression.