TechFW client AyuVis receives Orphan Drug Designation from FDA
Published on January 27, 2021
AyuVis Research Inc., a TechFW client, has been granted Orphan Drug Designation from the U.S. Food and Drug Administration for its small-molecule drug AVR-48, which prevents bronchopulmonary dysplasia.
Bronchopulmonary dysplasia is a chronic lung disease that can develop in babies born prematurely. It can lead to long-term respiratory issues, psychological disabilities or death. Babies with the disease require mechanical ventilation or other supplemental oxygen in order to survive, but mechanical ventilation can also damage their underdeveloped lungs or cause inflammation and tissue damage. A damaged lung can’t supply sufficient oxygen to other organs in the body – including the brain – and most babies with the condition grow into adulthood with asthma-like systems and poor brain development.
The AVR-48 drug produced by AyuVis helps modulate the immune system, decreasing its inflammatory response to mechanical ventilation and oxygen toxicity. This prevents the development of bronchopulmonary dysplasia, resulting in lives being saved, improvement in the quality of life of survivors and reduced hospital costs.
The Orphan Drug Designation is given by the FDA to drugs and biologics that treat, prevent or diagnose a rare disease or condition that affects fewer than 200,000 people in the United States. This designation also comes with financial incentives, including a partial tax credit for clinical trial expenditures, waiver of the new drug application fee (approximately $2.2 million) and eligibility for seven years of additional marketing exclusivity beyond the timeframe afforded by a strong patent position.
“We are very excited and encouraged that the FDA approved AVR-48 for a condition that has a huge medical need globally,” said Dr. Suchismita Acharya, founder and CEO of AyuVis Research Inc.
“The recognition by the FDA is validation of our science and will facilitate our strategic partnership discussions. The designation also can mark young companies as preferable to investors because it might be expected to have reduced competition,” Acharya said. “This is great motivation to our extraordinarily talented scientific, clinical and business team to march toward our goal to the clinic and beyond.”
AyuVis has plans for its first-in-human Phase 1 clinical trial in 2021. To reach the marketplace, AyuVis will complete GMP manufacturing of AVR-48 for clinical trials, finish the remaining IND-enabling pre-clinical studies and conduct Phases 1, 2a and 2b clinical trials. With positive clinical results, there is the opportunity to receive Accelerated Approval from the FDA, which could preclude a Phase 3 trial.