Vicore Pharma's Buloxibutid Receives Orphan Drug Designation in Japan for Idiopathic Pulmonary Fibrosis Treatment
Key Insights
Japan's Ministry of Health, Labor and Welfare (search) has granted Orphan Drug designation to buloxibutid, a first-in-class angiotensin II type 2 receptor (search) agonist for treating idiopathic pulmonary fibrosis (search).
The designation provides incentives including reduced consultation and review fees and extended market exclusivity upon approval in Japan.
Buloxibutid is currently being evaluated in the global Phase 2b ASPIRE trial, a 52-week study enrolling 270 patients across 14 countries.
Vicore Pharma Holding AB (search) announced that Japan's Ministry of Health, Labor and Welfare (search) (MHLW) has granted Orphan Drug designation to buloxibutid for the treatment of idiopathic pulmonary fibrosis (search) (IPF (search)). The designation recognizes the significant unmet medical need in IPF and the potential of this first-in-class therapy to offer improvements over existing treatments.
Novel Mechanism of Action
Buloxibutid is a first-in-class angiotensin II type 2 receptor (search) agonist that activates an upstream mechanism to drive alveolar repair, resolve fibrosis, and promote pulmonary vascular function. This represents a new class of drugs called angiotensin II type 2 receptor agonists (ATRAGs) that Vicore Pharma is developing for respiratory and fibrotic diseases.
"This milestone underscores the significant unmet medical need in IPF (search) and reinforces the potential of buloxibutid to offer significant improvement over existing therapies," said Bertil Lindmark, Chief Medical Officer of Vicore. "We look forward to working closely with our partners at Nippon Shinyaku and with the MHLW to support the development of buloxibutid in Japan."
Regulatory Recognition and Partnership
The Orphan Drug designation in Japan follows previous regulatory recognition in other major markets. Buloxibutid was granted Orphan Drug designation by the European Commission in 2016 and by the United States Food and Drug Administration (FDA) in 2017, as well as Fast Track designation by the FDA in 2025.
In February 2024, Vicore entered into an exclusive licensing agreement with Nippon Shinyaku to develop and commercialize buloxibutid in Japan. The Orphan Drug designation provides incentives such as reduced consultation and review fees, and extended market exclusivity upon approval in Japan.
Current Clinical Development
Buloxibutid is being evaluated in the global Phase 2b ASPIRE trial for the treatment of IPF (search). ASPIRE is an ongoing 52-week, randomized, double-blind, placebo-controlled clinical trial designed to assess the efficacy and safety of buloxibutid in IPF patients who are either not currently on treatment or receiving background nintedanib standard of care.
Participants are randomized to receive one of two doses of buloxibutid (100 mg or 50 mg taken orally twice daily) or placebo. The primary endpoint is change from baseline in forced vital capacity, the registrational endpoint for IPF (search). Secondary endpoints include safety, tolerability, and the proportion of patients with disease progression over the trial period. The trial will enroll 270 patients from approximately 100 sites across 14 countries, including the United States.
Addressing Unmet Medical Need
IPF (search) is a progressive and lethal fibrotic lung disease impacting approximately 250,000 people across the United States and Europe. The average life expectancy following diagnosis is 3-5 years, and currently approved therapies only slow the decline of lung function. While there are two anti-fibrotic therapies available today, a large proportion of patients do not initiate treatment, and those who do often discontinue due to limited efficacy and significant tolerability issues. With a growing patient population, there is a clear need for new disease-modifying treatments.