FDA Grants Certepetide Orphan Drug Designation For Cholangiocarcinoma

The U.S. Food and Drug Administration has granted Lisata Therapeutics’ Certepetide an Orphan Drug Designation to Treat Cholangiocarcinoma, a rare and aggressive form of bile duct cancer. The company made the announcement via press release here.

The disorder represents a substantial challenge to patients as the treatment options are extremely limited. Dr. Kristen Buck, Lisata’s chief medical officer, commented that receiving orphan drug designation is an opportunity to provide patients with many more innovative options.

Currently, Lisata is conducting a pivotal Phase 2a trial in the United States named the BOLSTER trial that is investigating cholangiocarcinoma.

Orphan Drug Designation (ODD)

In the United States, rare diseases are characterized as disorders that affect fewer than 200,000 individuals. Orphan Drug Designation affords recipients up to seven years of marketing exclusivity that includes exempting user fees and granting tax credits for clinical trials that meet requisite guidelines.

Additionally, closer collaboration with the FDA presents opportunities for shorter development timelines.

Cholangiocarcinoma: Rare and Difficult to Diagnose

Cholangiocarcinoma is cancer of the bile ducts, which are vital to digestion. The American Cancer Society estimates that approximately 8,000 individuals in the U.S. receive a cholangiocarcinoma diagnosis every year, but this number is possibly higher.

The five-year rate of survival for the disease is under 5% and is indicative of the urgency surrounding the need for effective treatments.

The investigational drug, certepetide, is designed to activate a transport mechanism. The result is the efficient co-administration of anti-cancer drugs while penetrating and then building up within the tumor.

More About Certepetide

According to Lisata, certepetide weakens the tumors immunosuppressive effect and keeps the tumor from spreading. Thus far, study data demonstrates effective delivery of anti-cancer regimens.

This includes an abundance of activity in ongoing trials demonstrating its ability to improve standard of care chemotherapy for pancreatic cancer together with the combination of immunotherapy and chemotherapy that is used in various solid tumors.

The list of certepetide’s awards includes:

• Orphan Drug Designation (Pancreatic cancer in the European Union and U.S.)
• Orphan Drug Designation for osteosarcoma, glioma, and cholangiocarcinoma (U.S)
• Rare Pediatric Disease Designation for Osteosarcoma (U.S.)
• US Fast Track designation

Lisata Therapeutics is focused upon commercialization of novel therapies to treat solid tumors and various major diseases. The Company is planning to announce various milestones within the next year.

Orphan Drug research

Last modified: September 16, 2024