Protara Therapeutics Gets FDA Fast Track for IV Choline Chloride for Parenteral Support

Protara Therapeutics, Inc., a clinical-stage company focused on developing innovative treatments for cancer and rare diseases, announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to their investigational drug, Intravenous (IV) Choline Chloride. This therapy is intended for adult and adolescent patients who rely on parenteral support (PS) and are unable to consume adequate nutrition orally or enterally. In the U.S., about 40,000 patients depend on long-term parenteral support.

Jesse Shefferman, CEO of Protara Therapeutics, emphasized the importance of this designation, highlighting the urgent need for effective treatments among PS-dependent patients. He noted that around 80% of these patients suffer from choline deficiency, which can cause severe liver damage, neuropsychological issues, muscle damage, and other health complications. Currently, there are no approved IV choline products for these patients globally. Shefferman reiterated the company's commitment to starting the registrational THRIVE-3 clinical trial in the first quarter of 2025.

The American Society for Parenteral and Enteral Nutrition has recommended IV choline for patients on parenteral support. Similarly, the European Society for Clinical Nutrition and Metabolism has included it in its guidelines for home parenteral nutrition. Protara plans to evaluate the safety and efficacy of IV Choline Chloride in the THRIVE-3 trial, which will include a Phase 2b/3 seamless design with dose confirmation, followed by a double-blinded, randomized, placebo-controlled trial. The trial will focus on measuring changes in plasma choline concentration from baseline compared to a placebo.

The FDA’s Fast Track program is designed to accelerate the development and review of drugs that address serious conditions and unmet medical needs. This designation allows for early and frequent communication with the FDA, potentially leading to faster approvals and priority reviews if specific criteria are met. Additionally, it enables the submission of parts of a New Drug Application on a rolling basis.

IV Choline Chloride is an investigational therapy aimed at addressing the needs of patients receiving parenteral support, who cannot synthesize enough choline from their diet. Choline is crucial for maintaining healthy liver function, gene expression, cell membrane signaling, brain development, neurotransmission, muscle function, and bone health. In the U.S., approximately 40,000 patients on long-term parenteral support lack access to a choline-containing PS formulation, leading to widespread deficiencies and liver damage among these patients. IV Choline Chloride holds promise as the first FDA-approved IV choline formulation for PS patients. The FDA has already granted Orphan Drug Designation to IV Choline Chloride for preventing choline deficiency in these patients. Additionally, Protara holds a U.S. patent for a choline composition with a term expiring in 2041.

Protara recently shared results from their THRIVE-1 study, which highlighted the prevalence of choline deficiency and liver injury among PS-dependent patients. The study revealed that 78% of these patients were choline deficient, with 63% showing signs of liver dysfunction, including steatosis, cholestasis, and other hepatic injuries.

Protara Therapeutics is a biotechnology company dedicated to developing transformative therapies for cancer and rare diseases. Their portfolio includes TARA-002, an investigational cell-based therapy for non-muscle invasive bladder cancer (NMIBC) and lymphatic malformations (LMs), currently in Phase 2 trials. Additionally, they are advancing IV Choline Chloride for patients on parenteral support.