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THOUSAND OAKS, Calif.--( BUSINESS WIRE )--AliveGen USA Inc. (AliveGen), a clinical stage biopharmaceutical company, announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to ALG-801 for the treatment of pulmonary arterial hypertension (PAH), a rare, serious, and life-threatening condition characterized by high blood pressure in the lung arteries that can lead to right-sided heart failure. “FDA’s Orphan Drug Designation for ALG-801 underscores the need for identifying better treatment options for patients suffering from PAH,” said Dr. HQ Han, CEO of AliveGen. “The designation represents another milestone in AliveGen’s efforts to discover and develop first-in-class/best-in-class therapeutics for high unmet medical needs. We believe that ALG-801, which has demonstrated superior efficacy and improved safety profile in preclinical and clinical Phase 1 studies, holds a remarkable potential to become a best-in-class medicine for PAH. We look forward to progressing ALG-801 clinical development.” The FDA Orphan Drug Designation program is granted to drugs and biologics intended for the safe and effective treatment, diagnosis, or prevention of rare diseases or conditions affecting fewer than 200,000 people in the United States. Orphan Drug Designation provides benefits to sponsors designed to support the development of drugs and biologics for small patient populations with unmet medical needs. These benefits include tax credits for clinical costs, exemptions from certain FDA fees, and potential seven years of marketing exclusivity. About ALG-801 ALG-801 is a novel next-generation ActRIIA/IIB hybrid ligand trap designed to selectively sequester a specific set of Smad2/3 pathway-activating ligands, which are critically involved in the pathogenesis of PAH as well as of many other diseases. ALG-801 has successfully completed its Phase 1a and 1b clinical trials in healthy volunteers and is ready for Phase 2 clinical development. About AliveGen AliveGen is a privately held clinical-stage biotechnology company located in Thousand Oaks, California. The company aims to discover first-in-class/best-in-class biotherapeutics for treating diseases with high unmet medical needs such as cardiometabolic disorders, neuromuscular diseases, age-related osteosarcopenia, and various wasting disorders. The company aspires to improve the care and quality of life of patients by developing innovative therapies that are safe and highly effective. Learn more about AliveGen on its website: www.alivegen.com .

The UK Medicines and Healthcare products Regulatory Agency (MHRA) announced on Tuesday the approval of a new drug developed by Merck for the treatment of a rare lung disease. This approval further solidifies the pharmaceutical company’s success in the potential blockbuster product sector. The drug, named sotaterept, was previously approved in the US in March 2024 and is marketed under the brand name Winrevair. It has also received approval in the EU and over 30 other markets.The MHRA's approval is based on research data involving 323 patients with pulmonary arterial hypertension. In this study, sotaterept demonstrated significantly better effects on improving patients' exercise capacity compared to a placebo. The development of sotaterept is part of Merck's acquisition of Acceleron Pharma for $11.5 billion in 2021. Through this acquisition, Merck obtained sotaterept and has been expanding its cardiovascular drug portfolio to address potential sales impacts from the expiration of patents on its bestselling cancer treatment Keytruda by the end of the century. Regulators noted that sotaterept can be used to treat adult patients with moderate to significant physical activity limitations to help improve their exercise capacity. The recommended dosing schedule is a self-injection every three weeks. According to the National Health Service (NHS) data, there were 9,263 active pulmonary arterial hypertension patients in the UK as of March 2024. Pulmonary arterial hypertension refers to elevated blood pressure in the vessels supplying blood to the lungs, making it more difficult for the heart to pump blood and potentially leading to organ failure. Sotaterept works by targeting the activation protein, which plays a key role in pulmonary artery narrowing. Since its launch in March, Winrevair has achieved $149 million in sales. According to data compiled by LSEG, analysts expect its sales to exceed $1 billion by 2025. In the US, the therapy is priced at $14,000 per vial. Merck has not yet responded to inquiries from reporters regarding the pricing and availability of the therapy in the UK.

MSD acquired the therapy from Acceleron Pharma. Credit: Copyright © 2024 Merck & Co., Inc., Rahway, NJ, USA and its affiliates. All rights reserved. MSD has received the UK Medicines and Healthcare products Regulatory Agency (MHRA) approval for sotatercept, marketed as Winrevair, to treat pulmonary arterial hypertension (PAH) in the adult population. The therapy is used along with other PAH medications to improve exercise capacity in patients with moderate to severe limitations in physical activity. Designed to address the underlying causes of PAH, which involves high blood pressure in the pulmonary arteries that can lead to right heart damage, sotatercept operates by easing the narrowing of these arteries, helping the heart pump blood more efficiently to the lungs and improving physical activity capabilities. A pivotal study involving 323 PAH subjects demonstrated the therapy’s effectiveness compared to placebo in enhancing exercise capacity. The main measure of this was the change in the six-minute walk distance (6MWD) after 24 weeks of treatment. Subjects who received the therapy, in addition to their standard PAH therapy, could walk approximately 34 metres farther than before treatment, while those on placebo improved by only one metre. The median treatment difference between the therapy and placebo groups stood at 40.8 metres. The UK regulator has recommended a dosing schedule of one injection every three weeks, which patients can self-administer. Common adverse events reported in more than one in 10 subjects include low blood platelet count, elevated haemoglobin levels and skin rash. The safety and effectiveness of the therapy will continue to be monitored closely by the agency. The approval of sotatercept came through the International Recognition Procedure (IRP). MSD’s acquisition of Acceleron Pharma in September 2021 for $11.5bn, at $180 per share, led to the addition of sotatercept to its portfolio. MHRA healthcare quality and access interim executive director Julian Beach stated: “Keeping patients safe and enabling their access to high quality, safe and effective medical products are key priorities for us. We’re assured that the appropriate regulatory standards for the approval of this medicine have been met. As with all products, we will keep its safety under close review.”