In a pair of high-impact regulatory moves, the U.S. Food and Drug Administration (FDA) approved two innovative therapies that address complex, under-treated conditions. Between March 25 and 26, 2025, Novartis and Theratechnologies secured FDA approval for iptacopan (branded as Fabhalta) and tesamorelin F8 (branded as Egrifta WR), respectively. These approvals mark significant advancements in treatment strategies for rare kidney disease and HIV-associated fat redistribution.
Novartis’ iptacopan has now received its third FDA approval, this time for the treatment of C3 glomerulopathy (C3G), a rare and serious complement-mediated kidney disease. Fabhalta becomes the first oral treatment available for this condition, offering new hope for patients who previously faced limited and often invasive therapeutic options. C3G, caused by dysregulation in the complement system, often progresses to end-stage renal disease. With Fabhalta, patients now have access to a non-invasive, lifelong treatment regimen that may be integrated into standard nephrology care. The oral formulation is expected to improve patient adherence, reduce clinical burdens, and potentially delay disease progression.
Theratechnologies’ Egrifta WR (tesamorelin F8) also represents a milestone as the first long-acting formulation of a growth hormone-releasing factor specifically designed for adults living with HIV-associated lipodystrophy. This condition is characterized by an abnormal accumulation of visceral fat, contributing to increased cardiovascular and metabolic risks. The new formulation reduces the frequency of injections, a significant improvement over previous therapies that required daily dosing. As a long-acting version, Egrifta WR aims to enhance patient adherence while delivering sustained metabolic benefits and improved quality of life for people living with HIV.
These back-to-back FDA approvals highlight the agency’s commitment to expediting treatments that address unmet medical needs, particularly in the areas of rare diseases and chronic metabolic conditions. Analysts point to the FDA’s accelerated review pathways, such as Breakthrough Therapy and Fast Track designations, as key mechanisms in bringing these therapies to market swiftly. Industry observers note that these approvals demonstrate a clear trend toward prioritizing patient-centric, innovative treatment modalities that align with modern healthcare goals of convenience, adherence, and long-term outcomes.
Both Novartis and Theratechnologies are expected to move rapidly in launching these drugs, with healthcare providers already preparing for their integration into treatment protocols. Patients and advocacy groups have expressed optimism that these therapies will significantly enhance care and quality of life for those affected by these challenging conditions.
