Pulmovant Receives Orphan Drug Designation in Japan for Mosliciguat for the Treatment of Pulmonary Hypertension Associated with Interstitial Lung Disease (PH-ILD)

September 04, 2025 07:00 ET | Source: Pulmovant, Inc. Orphan drug designation from Japan’s Ministry of Health, Labour and Welfare provides key regulatory benefits, including priority review and extended market exclusivity for potential treatments targeting diseases for which there is no sufficient alternativeA global Phase 2 PHocus study of mosliciguat in PH-ILD is currently ongoing and will be highlighted in a study-in-progress poster at the European Respiratory Society International Congress 2025 on September 28th WALTHAM, Mass., Sept. 04, 2025 (GLOBE NEWSWIRE) -- Pulmovant, a clinical-stage biotechnology company committed to transforming the lives of patients with pulmonary diseases, and a Roivant (Nasdaq: ROIV) company, today announced that Japan’s Ministry of Health, Labour and Welfare (MHLW) has granted orphan drug designation (ODD) to mosliciguat, a novel, once-daily, inhaled soluble guanylate cyclase (sGC) activator, currently being studied for the treatment of Pulmonary Hypertension associated with Interstitial Lung Disease (PH-ILD), which is a progressive and life-threatening condition with significant unmet medical need. “Obtaining orphan drug designation for mosliciguat in Japan validates the significant unmet need that exists for people living with PH-ILD, a progressive and life-threatening cardiopulmonary condition with poor prognosis and limited or no treatment options,” said Drew Fromkin, Chief Executive Officer of Pulmovant. “We appreciate the MHLW’s recognition of this challenge for PH-ILD patients and look forward to continued collaboration with Japanese regulators as we advance mosliciguat through the next stages of clinical development.” ODD designation is based on the recommendation of Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) and provides key regulatory and development incentives, including priority consultation, reduced regulatory fees, and up to 10 years of market exclusivity following potential approval. For more information about Japan’s ODD, please visit the MHLW website. Mosliciguat is a potential first-in-class, once-daily, inhaled sGC activator with a differentiated mechanism of action designed to deliver targeted pulmonary vasodilation with limited systemic side effects for the treatment of PH-ILD. Mosliciguat is currently being evaluated in the Phase 2 PHocus clinical study (NCT06635850), a randomized, double-blind, placebo-controlled, global trial assessing its safety and efficacy in approximately 120 adult patients with PH-ILD. A study-in-progress poster outlining the design of PHocus will be presented at the European Respiratory Society (ERS) International Congress 2025 on September 28th. About Pulmonary Hypertension and Interstitial Lung DiseasePulmonary hypertension (PH) is a progressive and debilitating condition characterized by high blood pressure in the blood vessels of the lungs. This elevated pressure forces the heart to work harder to pump blood through the lungs, leading to symptoms such as shortness of breath, fatigue, chest pain, and dizziness. The WHO has classified PH into five groups based on their underlying causes, symptoms, and treatment approaches. Group 3 PH is a subtype of PH that arises from lung diseases, such as interstitial lung disease (ILD). ILD describes a large group of diseases that cause progressive damage to the lungs, making it difficult for patients to breathe. Up to 200,000 patients across the U.S. and Europe are living with PH-ILD, a subset of Group 3 PH, and have limited or no approved treatment options. For more information, please visit https://www.pulmovant.com/our-science. About MosliciguatMosliciguat is a potential first-in-class, once-daily, inhaled sGC activator with a differentiated mechanism of action, which may have broad application across the spectrum of pulmonary hypertension (PH). Mosliciguat targets sGC, a key enzyme in the nitric oxide (NO)/cyclic guanosine monophosphate (cGMP) signaling pathway that catalyzes cGMP production. Elevated cGMP levels are known to promote vasodilation, reduce inflammation and apoptosis, reverse vascular remodeling, and contribute to anti-fibrotic effects. Unlike sGC stimulators, which require reduced heme and NO to exert their effect, mosliciguat is an sGC activator that is thought to work independently of heme and NO. In the Phase 1b ATMOS study of mosliciguat, a single dose of inhaled mosliciguat in PH patients was well tolerated and led to clinically meaningful, mean peak reduction in pulmonary vascular resistance (PVR) of up to 38%, one of the highest reductions seen in pulmonary hypertension trials to date. For information on the Phase 2 PHocus study of mosliciguat, please visit https://phocusstudy.com. About PulmovantPulmovant is a clinical-stage biotechnology company committed to transforming the lives of patients with pulmonary diseases and is a Roivant (Nasdaq: ROIV) company. Pulmovant’s first investigational candidate, mosliciguat, is designed to provide a novel, once-daily, inhaled treatment option for patients with pulmonary hypertension associated with Interstitial Lung Disease (PH-ILD). Mosliciguat is a potential first-in-class soluble guanylate cyclase activator with a differentiated mechanism of action currently being evaluated in the Phase 2 PHocus global clinical trial in PH-ILD. For more information, please visit https://www.pulmovant.com. About Roivant Roivant (Nasdaq: ROIV) is a biopharmaceutical company that aims to improve the lives of patients by accelerating the development and commercialization of medicines that matter. Roivant’s pipeline includes brepocitinib, a potent small molecule inhibitor of TYK2 and JAK1 in development for the treatment of dermatomyositis, non-infectious uveitis and cutaneous sarcoidosis; IMVT-1402 and batoclimab, fully human monoclonal antibodies targeting FcRn in development across several IgG-mediated autoimmune indications; and mosliciguat, an inhaled sGC activator in development for pulmonary hypertension associated with interstitial lung disease. We advance our pipeline by creating nimble subsidiaries or “Vants” to develop and commercialize our medicines and technologies. Beyond therapeutics, Roivant also incubates discovery-stage companies and health technology startups complementary to its biopharmaceutical business. For more information, visit www.roivant.com. Forward-Looking Statements This press release contains forward-looking statements. Statements in this press release may include statements that are not historical facts and are considered forward-looking, which are usually identified by the use of words such as "anticipate," "believe," "continue," "could," "estimate," "expect," "intends," "may," "might," "plan," "possible," "potential," "predict," "project," "should," "would" and variations of such words or similar expressions. The words may identify forward-looking statements, but the absence of these words does not mean that a statement is not forward-looking. Our forward-looking statements include, but are not limited to, statements regarding our or our management team's expectations, hopes, beliefs, intentions or strategies regarding the future, and statements that are not historical facts, including statements about the clinical and therapeutic potential of our product candidate. In addition, any statements that refer to projections, forecasts or other characterizations of future events or circumstances, including any underlying assumptions, are forward-looking statements. Although we believe that our plans, intentions, expectations and strategies as reflected in or suggested by those forward-looking statements are reasonable, we can give no assurance that the plans, intentions, expectations or strategies will be attained or achieved. Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a number of risks, uncertainties and assumptions. Moreover, we operate in a very competitive and rapidly changing environment in which new risks emerge from time to time. These forward-looking statements are based upon the current expectations and beliefs of our management as of the date of this press release and are subject to certain risks and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. Except as required by applicable law, we assume no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise. © 2025 Pulmovant, Inc. All Rights Reserved. All trademarks are the property of their respective owners. Contactinquiries@pulmovant.com

Pulmovant Receives Orphan Drug Designation in Japan for Mosliciguat for the Treatment of Pulmonary Hypertension Associated with Interstitial Lung Disease (PH-ILD)

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