| |JANUARY 20258NMD PHARMA RECEIVES US FDA ORPHAN DRUG DESIGNATION FOR NMD670 IN CMTGerman multinational pharmaceutical and biotechnology firm Bayer aims to bolster its presence in India by broadening its range of therapies and increasing collaborations throughout the healthcare ecosystem. Bayer has launched various treatments to tackle particular health issues in these regions. Recent product introductions, such as Kerendia (finerenone), Verquvo (vericiguat), and Nubeqa (darolutamide), exemplify this approach. Kerendia aids in preventing or postponing kidney failure in individuals with diabetes, whereas Verquvo aims to lower hospitalisation rates in those with deteriorating heart failure. Nubeqa, a therapy for prostate cancer, has broadened its approved uses since its introduction in July 2022. "Our focus in India's pharmaceutical market is on addressing critical healthcare needs across key therapeutic areas such as cardiovascular, diabetes, kidney health, women's health, oncology, and ophthalmology," said Shweta Rai, Managing Director India and Country Division Head South Asia, Bayer Pharmaceuticals."Currently, we are conducting approximately 17 clinical trials in India, spanning Phase II, III, and IV, with nearly 4,000 Indian participants. This ensures that our treatments are both effective and safe for the local population," Rai explained.India holds an important position in Bayer's worldwide activities, especially in the areas of research and development. The Global Capability Centre in Hyderabad of the company plays a role in R&D, data science, and AI-based healthcare solutions, aiding global Phase II and III trials. PONMD Pharma A/S, a biotech firm in the clinical stage focused on creating innovative and enhanced therapies for individuals with neuromuscular disorders, revealed that the U.S. Food and Drug Administration (FDA) has awarded orphan drug designation (ODD) to NMD670, a new, oral, small molecule blocker of the skeletal muscle-specific chloride ion channel ClC-1, aimed at addressing Charcot-Marie-Tooth disease (CMT).CMT is an inherited nerve disorder marked by muscle weakness and fatigue, for which there are no approved treatments. Approximately 136,000 people in the US are affected by CMT, and it is believed that more than 3 million people globally suffer from CMT. Clinical characteristics of this uncommon neuromuscular disorder involve motor signs and symptoms like muscle weakness, muscle wasting, and tiredness, along with sensory impairments that, together, can significantly diminish quality of life."NMD Pharma is committed to addressing the need of patients living with neuromuscular diseases such as CMT, and we are thrilled that the FDA has granted orphan drug designation to NMD670," commented Thomas Holm Pedersen, Chief Executive Officer of NMD Pharma. "Based on positive results from preclinical studies and the recently published ESTABLISH1 CMT observational study, this designation not only highlights the urgent need for novel, effective treatments for this rare disease, but also underscores the therapeutic potential of our skeletal muscle-specific ClC-1 inhibitor approach to address the associated muscle weakness and fatigue". POTOP STORIESBAYER BOLSTERS INDIA FOCUS WITH EXPANDED THERAPIES AND HEALTHCARE ECOSYSTEM
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