FDA Separately Grants Lantern Pharma and Codexis Rare Pediatric Disease Designations

The U.S. Food and Drug Administration granted Rare Pediatric Disease and Orphan Drug designations to Lantern Pharma’s treatment for a rare cancer of the central nervous system and to Codexis’ treatment for homocystinuria.

Photo: John Nicols, president and CEO of Codexis

Lantern Pharma received the designations for its experimental drug candidate LP-184 for the treatment of pediatric patients with Atypical Teratoid Rhabdoid Tumor (ATRT), an aggressive and rapidly growing cancer of the central nervous system. LP-184 is also being pursued as a potential new therapy across a range of genetically defined solid tumors, including pancreatic cancer and glioblastoma multiforme.

The National Cancer Institute classifies ATRT as grade IV tumors, meaning they are malignant, aggressive, and fast-growing. ATRTs are very aggressive childhood malignancies of the central nervous system. The underlying genetic cause are inactivating bi-allelic mutations in SMARCB1 (also called INI1) or in SMARCA4. Nearly 90 percent of pediatric ATRTs are caused by changes in the gene known as SMARCB1.

“Historical approaches to treating pediatric ATRT such as surgery, radiation, and chemotherapy have had largely unfavorable long-term outcomes for children, and new options are urgently needed,” said Kishor Bhatia, chief scientific officer of Lantern Pharma. Receiving Rare Pediatric Disease Designation from the FDA underscores the critical value of our growing focus on pediatric oncology indications at Lantern and represents another significant milestone for the LP-184 program.”

The FDA also granted Orphan Drug and Rare Pediatric Disease designations to Codexis’ CDX-6512 for the treatment of homocystinuria.

Homocystinuria is a rare inborn error of metabolism most commonly due to cystathionine beta-synthase (CBS) deficiency and is characterized by elevated levels of homocysteine in blood and urine that when left untreated may lead to learning and intellectual disabilities, cardiovascular disease, osteoporosis, and stroke. Homocysteine is a metabolite derived from methionine, an essential amino acid that enters the body as part of dietary protein. Strict, life-long adherence to a methionine-restricted diet, often paired with vitamin supplementation, is currently the only available therapy.

CDX-6512, which is in pre-IND development, is a gastrointestinal-stable enzyme specifically engineered to be highly resistant to both the acidic conditions of the stomach and to proteases of the upper intestines, enabling it to effectively degrade methionine that is liberated from protein digestion. Elevated levels of this amino acid and its metabolite homocysteine, leads to the various clinical manifestations of HCU.

“The Orphan Drug and Rare Pediatric Disease designations for CDX-6512 further build upon the momentum our engineered enzymes have generated as potential first-in-class oral therapeutics for inborn errors of metabolism,” said John Nicols, president and CEO of Codexis. “These designations represent an important step forward in the development of CDX-6512 as a potential treatment of homocystinuria and we look forward to continuing its advancement toward the clinic.”

Orphan Drug designation is granted by the FDA to drugs and biologics intended for the treatment, diagnosis or prevention of diseases or disorders that affect fewer than 200,000 people in the United States. The designation provides incentives for sponsors to develop products, which may include tax credits toward the cost of clinical trials and prescription drug user fee waivers. The ODD could also entitle a company to a seven-year period of marketing exclusivity in the United States should the company receive FDA approval for the product candidate.

The FDA grants Rare Pediatric Disease designation for serious and life-threatening diseases that primarily affect children aged 18 years or younger and impact fewer than 200,000 people in the United States. If a New Drug Application in the United States for IMX-110 is approved, ImmixBio may be eligible to receive a Priority Review Voucher (PRV) from the FDA.

The vouchers can be used to reduce the time of an FDA new drug approval review to six months from ten months. The vouchers are potentially lucrative because they are transferable. Most recently, Mirum Pharmaceuticals sold its voucher for $110 million.

Author: Rare Daily Staff