HONG KONG, Aug. 5, 2024 /PRNewswire/ — META Pharmaceuticals Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) to its investigational new drug META-001-PH for the treatment of primary hyperoxaluria (PH). Primary hyperoxaluria is a rare genetic disorder that can lead to kidney stone formation, renal failure, and can be life-threatening in severe cases. The RPDD is intended to facilitate the development of drugs and biologics for serious and life-threatening rare pediatric diseases that affect fewer than 200,000 people in the U.S. and predominantly occur in patients aged 18 years and younger. This designation is pursuant to section 529(a)(3) of the Federal Food, Drug, and Cosmetic Act (FD&C Act) (21 U.S.C. 360ff(a)(3)).
About Primary Hyperoxaluria (PH)
Primary Hyperoxaluria (PH) is an autosomal recessive metabolic disorder in which oxalate is overproduced and deposited in the body due to defects in enzymes responsible for oxalate metabolism in the liver and other organs. Patients typically present with kidney stones, nephrocalcinosis, renal failure, and oxalate deposition in other organs. Severe cases can lead to end-stage renal disease (ESRD) requiring dialysis, kidney transplantation, or combined liver-kidney transplantation. Symptoms of the disease usually appear at the age of 0 to 3. Without intervention, most patients will develop end-stage renal disease during adolescence, which severely threatens their lives. The incidence of PH is estimated to be 1/58,000, affecting more than 10,000 people in the United States and the European Union, and more than 20,000 people in China. Currently, there is no cure for primary hyperoxaluria. Existing treatments are primarily supportive care, including increased fluid intake to dilute oxalate in the urine, and medications such as pyridoxine (vitamin B6) to reduce oxalate production. However, patients’ symptoms and disease progression cannot be effectively controlled.
About META-001-PH
META-001-PH is a groundbreaking small molecule drug developed by META for the treatment of primary hyperoxaluria. This innovative molecule is the product of a collaborative effort between META’s chemistry team and XtalPi’s (2228.HK) AI drug discovery team, who worked together from the initial scaffold screening to the preclinical compound nomination. XtalPi’s automated robotic chemical synthesis lab was responsible for the chemical synthesis of the related molecule series, ensuring a precise and efficient production process. Preclinical experiments in animal disease models have shown that META-001-PH can significantly reduce urinary oxalate excretion by up to 80%. While existing therapeutic agents are unable to effectively control urinary oxalate levels in the long term, META-001-PH, administered orally daily, can maintain oxalate at normal levels, thus demonstrating the potential for better long-term control of kidney stone formation in patients with PH. META-001-PH has also demonstrated good tolerability and safety in preclinical animal models and is undergoing IND-enabling toxicology studies with a clinical Phase I safety assessment on healthy subjects planned for 2025H1 in Australia. This collaboration between META and XtalPi marks a significant advancement in the development of effective treatments for primary hyperoxaluria.
About Rare Pediatric Disease Designation (RPDD)
The Rare Pediatric Disease Designation (RPDD) is an eligibility determination for rare pediatric diseases that affect fewer than 200,000 patients and pose a serious life-threatening risk to children under the age of 18. The RPDD and Priority Review Voucher (PRV) program aims to recognize the significant need for therapies for rare pediatric diseases and to encourage the development of new treatments for these serious or life-threatening conditions. Under the program, sponsors will be eligible to receive a PRV upon approval of a New Drug Application (NDA) or Biologics License Application (BLA) for a rare pediatric disease. PRVs can be used for any subsequent product approval application, reducing the review time by 4-6 months, or can be traded with an average transfer price of more than $100 million in recent years.
About META Pharmaceuticals Inc.
META Pharmaceuticals Inc. (META) is an innovative biopharmaceutical company dedicated to discovering and developing urgently needed breakthrough treatments for a broad range of autoimmune disorders, metabolic diseases, and cancer. As the only company in the Asia Pacific Region leveraging the groundbreaking theory of immuno-metabolism, META leads the way in creating safer and more effective therapies that modulate cellular metabolism to regulate immune system function and other pathways. META has developed two distinct chemical series targeting META-001 for the treatment of autoimmune diseases and primary hyperoxaluria, respectively, which hold the potential to become the next-generation oral treatments for these conditions, addressing significant unmet clinical needs. META Pharmaceuticals Inc. is jointly incubated by XtalPi (2228.HK), the leader in the AI-pharmaceutical industry, Forcefield Ventures, and IMO Ventures, with investments from Tiantu Capital (1973.HK), Yael Capital, Fangyuan Capital, Lead Rich International, Decent Capital, and Bopu Capital. For more information, please visit http://en.metabiopharma.com/ and follow us on LinkedIn.
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