We have established a portfolio of de-risked assets addressing multiple potential indications.
* TARA-002 Granted Rare Pediatric Disease designation. OK-432 Granted Orphan Drug Designation by the U.S. FDA, applicable under established comparability.
** Granted Orphan Drug and Fast Track Designations by the U.S. FDA
IV Choline Chloride for IFALD
IV choline chloride is an investigational, intravenous (IV) phospholipid substrate replacement therapy initially in development for patients receiving parenteral nutrition (PN) who have intestinal failure-associated liver disease (IFALD).
Choline is a known important substrate for phospholipids that are critical for healthy liver function. Because PN patients cannot sufficiently absorb adequate levels of choline and no available PN formulations contain sufficient amounts of choline to correct this deficiency, PN patients often experience a prolonged progression to hepatic failure and death, with the only known intervention being a dual small bowel / liver transplant. If approved, IV choline chloride would be the first approved therapy for IFALD. It has been granted Orphan Drug Designations (ODDs) by the FDA for the treatment of IFALD and the prevention of choline deficiency in PN patients.
Protara had an end of Phase 2 meeting with the FDA in late 2018 and received the FDA’s support to advance IV choline chloride into a registration-enabling study for the treatment of IFALD.
Intestinal failure-associated liver disease (IFALD), which occurs in patients dependent on PN support, is characterized by choline deficiency, hepatic steatosis, cholestasis, and rapid progression of liver disease through to hepatic failure and death in the absence of intestine-liver transplant. IFALD carries a relatively poor prognosis, with a 15-34% death rate within 1-4 years. When IFALD presents with symptoms of liver disease in children, mortality is even higher (23–40%).
IFALD is uniquely characterized by the presence of both steatosis (toxic fat accumulation in liver cells) and cholestasis (damage to the biliary system in the liver) in patients who are chronic (greater than six months) PN users. A patient is considered to have IFALD if she/he is dependent on PN for more than six months (e.g., has chronic intestinal failure); has evidence of steatosis, determined by imaging techniques or histologic assessments; has evidence of cholestasis (e.g., elevated alkaline phosphatase (ALP), elevated bilirubin and/or histology); and may have evidence of ongoing, progressive liver injury on the basis of multiple abnormal liver function tests, in conjunction with findings of fibrosis, cirrhosis, and/or end-stage liver disease (ESLD).
Expanded Access Policy
Protara is committed to identifying and advancing transformative therapies for the treatment of cancer and rare diseases with significant unmet needs.
Expanded access, also called compassionate use, makes an investigational product available for treatment outside of clinical trials when no comparable or satisfactory alternative therapy option is available.
Protara is committed to advancing its investigational therapies, TARA-002 and IV Choline Chloride, by continuing to study them in clinical trials designed to confirm potential safety, tolerability and efficacy. We believe the best way to make our therapies available to patients is by enrolling and completing all of our clinical trials and, if those trials are successful, pursuing regulatory approval. Protara is not making its medicines available via expanded access at this time. We are focused on advancing our clinical development programs and more information about our clinical trials can be found on ClinicalTrials.gov when those trials are underway. We believe that participation in our clinical trials will be the most appropriate way to access our investigational medicines prior to their approval.
Protara will continually assess our current expanded access policy and provide updates to this website. We look forward to collaborating with the U.S. FDA and making these important therapies available to patients. Please contact us with any questions at email@example.com.