Protara Therapeutics, Inc. (Nasdaq: TARA) has been granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for its Intravenous (IV) Choline Chloride. This investigational therapy is aimed at addressing choline deficiency in adult and adolescent patients receiving parenteral support (PS) when oral or enteral nutrition is not feasible, insufficient, or contraindicated.
The FDA's Fast Track program is designed to expedite the development and review of drugs that treat serious conditions and fulfill unmet medical needs. This designation underscores the urgent requirement for a safe and effective treatment for choline deficiency in PS patients, a condition that can lead to severe hepatic injury, neuropsychological impairment, muscle damage, and thrombotic abnormalities.
Addressing Choline Deficiency in Parenteral Support Patients
Approximately 40,000 patients in the U.S. rely on long-term parenteral support. According to Protara Therapeutics, around 80% of these patients experience choline deficiency. This deficiency arises because PS patients cannot synthesize choline from enteral nutrition sources, and currently, there are no available PS formulations containing choline. The American Society for Parenteral and Enteral Nutrition, along with the European Society for Clinical Nutrition and Metabolism, recommends IV choline for patients receiving PS.
"Receipt of Fast Track designation underscores the urgent need in these patients and our belief that IV Choline Chloride has the potential to serve as the first FDA-approved IV choline therapy for patients dependent on PS," said Jesse Shefferman, Chief Executive Officer of Protara Therapeutics.
THRIVE-3 Clinical Trial
Protara Therapeutics is planning to initiate the registrational THRIVE-3 clinical trial in the first quarter of 2025. This Phase 2b/3 trial will assess the safety and efficacy of IV Choline Chloride in adolescents and adults receiving parenteral support. The trial design includes a dose confirmation phase followed by a double-blinded, randomized, placebo-controlled phase. The primary endpoint of the trial is the change in plasma choline concentration from baseline compared to placebo.
Potential Impact and Future Directions
If approved, IV Choline Chloride could become the first FDA-approved IV choline formulation for PS patients. The therapy has already received Orphan Drug Designation from the FDA for the prevention of choline deficiency in PS patients. Protara holds a U.S. patent for a choline composition, which is set to expire in 2041.
Protara recently presented results from the THRIVE-1 study, a prospective, observational study, which found that 78% of patients dependent on PS were choline deficient, with 63% of these patients demonstrating liver dysfunction, including steatosis, cholestasis, and signs of hepatobiliary injury. The Fast Track designation and the planned THRIVE-3 trial represent significant steps toward addressing this critical unmet need.
