Chinese AI-Powered Biotech Metabiopharma Secures FDA Rare Pediatric Disease Designation for Novel PH Therapy META-001-PH

August 5, 2024META Pharmaceuticals Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation to its investigational new drug META-001-PH.（Rare Pediatric Disease Designation, RPDD）, used for the treatment of primary hyperoxaluria（Primary Hyperoxaluria, PH）。

Primary Hyperoxaluria is a rare genetic disorder that can cause kidney stones and kidney failure, and in severe cases, it can be life-threatening. RPDD aims to promote the development of drugs and biologics for serious and life-threatening pediatric rare diseases. The FDA grants RPDD to therapeutic options for diseases that affect fewer than 200,000 people in the U.S. and primarily occur in patients aged 18 years or younger.

This designation is based on Section 529(a)(3) of the Federal Food, Drug, and Cosmetic Act (FD&C Act) (21 U.S.C. 360ff(a)(3)).

Primary Hyperoxaluria (PH) is an autosomal recessive metabolic disorder caused by enzyme defects responsible for oxalate metabolism in the liver and other organs, leading to excessive oxalate production and deposition in the body.

Patients typically present with kidney stones, nephrocalcinosis, renal insufficiency, 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. Primary hyperoxaluria mainly affects children, with symptoms usually manifesting between 0-3 years of age. Without intervention, most patients will progress to end-stage renal disease during adolescence, posing a severe threat to life.

It is estimated that the incidence rate of primary hyperoxaluria is 1/58,000, affecting over ten thousand people in the United States and the European Union, and more than twenty thousand people in China. Currently, there is no specific drug available to cure the disease. Existing treatments mainly involve supportive care, including increasing fluid intake to dilute oxalate concentration in urine and using medications such as pyridoxine (vitamin B6) to reduce oxalate production. However, patients' symptoms and disease progression cannot be effectively controlled.

META-001-PH is a globally pioneering small-molecule drug independently developed by META Pharmaceuticals Inc. for the treatment of primary hyperoxaluria. Preclinical animal disease model experiments have shown that, by inhibiting the most critical step in the synthesis process—the metabolism of glyoxylate into oxalate—META-001-PH can significantly reduce urinary oxalate excretion by up to 80%.

Existing therapeutic drugs are unable to control oxalate concentration in patients' urine effectively over the long term. However, META-001-PH, taken orally on a daily basis, can continuously maintain oxalate at normal levels, thereby demonstrating the potential for long-term control of kidney stone formation in patients with hyperoxaluria. This is also the most promising therapy currently under research for complete control of the disease.

In preclinical animal models, META-001-PH also demonstrated good tolerability and safety. It is currently undergoing toxicology studies for clinical application, with plans to initiate Phase I clinical trials for safety assessment in healthy volunteers in Australia in the first half of 2025.

RPDD (Rare Pediatric Disease Designation) is a qualification granted by the U.S. FDA for rare pediatric diseases affecting fewer than 200,000 patients in the United States and posing a life-threatening risk to children under the age of 18. The RPDD and Priority Review Voucher program aim to recognize the significant need for therapies addressing rare pediatric diseases and encourage the development of new treatments for these serious or life-threatening conditions.

Under this program, sponsors will be eligible for a Priority Review Voucher (PRV) after the approval of a New Drug Application (NDA) or Biologics License Application (BLA) for pediatric rare diseases. The PRV can be used for the marketing application of any subsequent product, reducing the review time by 4-6 months, or it can be sold to a third party, with an average transfer price exceeding 100 million US dollars in recent years.

META is an innovative pharmaceutical company focused on the research and development of drugs for autoimmune and metabolic diseases. Based on cutting-edge emerging biological theories, META effectively modulates immune system functions and other physiological system functions by regulating cellular metabolic activity. The company is dedicated to developing safer and more effective treatments for autoimmune and metabolic diseases, with a primary focus on first-in-class novel drug research and development, aiming to provide urgently needed medications for patients worldwide.