Zydus Lifesciences on Thursday announced that the US Food and Drug Administration (USFDA) has granted Orphan Drug Designation (ODD) to its investigational product Desidustat for the treatment of beta-thalassemia. The recognition marks an important milestone for the Ahmedabad-based pharmaceutical company as it expands its research footprint into therapies targeting rare and life-threatening conditions.

Beta-thalassemia is a genetic blood disorder characterized by reduced hemoglobin levels, impairing oxygen transport across the body and often leading to severe anemia, fatigue, organ damage, and other serious complications. Patients with the condition rely on frequent blood transfusions and treatments to manage iron overload, which significantly affect quality of life. Desidustat, a hypoxia inducible factor (HIF)-prolyl hydroxylase inhibitor (PHI), is being developed to boost hemoglobin and red blood cell production, potentially offering a safer and more sustainable alternative for patients.

“This Orphan Drug Designation from the USFDA underlines the urgent medical need to develop Desidustat to address beta-thalassemia,” said Zydus Lifesciences Managing Director Sharvil Patel in a regulatory filing. The company emphasized that the designation validates its research focus on addressing rare and underserved diseases.

Also Read: Zubeen Garg Fans Rage as Murder Accused Shifted to Empty Jail with VIP Treatment

Receiving orphan status provides Zydus with several key incentives under the FDA’s regulatory framework. These include eligibility for tax credits on clinical research, exemption from certain prescription drug user fees, and the potential for seven years of market exclusivity in the United States upon approval. Experts say such designations are vital to encouraging innovation in areas where patient populations are too small to attract widespread commercial investment.

Zydus Lifesciences, known for its work in chronic disease and specialty drug development, continues to expand its global pipeline with an emphasis on innovative molecules in hematology and oncology. The company’s stock was trading 0.71 percent lower at Rs 965.95 per share on the BSE following the announcement. Nonetheless, the latest development positions Zydus among Indian pharmaceutical firms making strides in rare disease research on the international stage.

Also Read: Kerala Election Officer Mauled by Pet Dog During Voter List Duty in Kottayam