FDA News

FDA grants orphan drug designation to OBI-999 for pancreatic cancer

The FDA granted orphan drug designation to the antibody-drug conjugate OBI-999 for the treatment of pancreatic cancer, according to a press release from the agent’s manufacturer.

OBI-999 (OBI Pharma) targets Globo H, a glycolipid antigen expressed in up to 15 epithelial cancers.

Preclinical xenograft animal models of pancreatic, lung, gastric and breast cancers showed OBI-999 induced profound tumor shrinkage at various doses. The agent also appeared well-tolerated with a favorable safety profile in preclinical single and repeated dose toxicology studies.

Researchers have initiated enrollment in a phase 1/phase 2 clinical trial to assess the safety and preliminary efficacy of OBI-999 among patients with locally advanced or metastatic solid tumors, including pancreatic, gastric, colorectal and esophageal cancers.

“We are very excited about the potential value that OBI-999 may provide to patients with pancreatic cancer, given both the high potency we have observed using OBI-999 in pancreatic cancer xenograft models and because many pancreas cancers highly overexpress Globo H, the glycolipid target of OBI-999, using the validated immunohistochemistry assay that will be available for selecting patients for the phase 2 portion of this first-in-human clinical trial,” Tillman Pearce, MD, chief medical officer of OBI Pharma, said in the press release.

The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.

 

The FDA granted orphan drug designation to the antibody-drug conjugate OBI-999 for the treatment of pancreatic cancer, according to a press release from the agent’s manufacturer.

OBI-999 (OBI Pharma) targets Globo H, a glycolipid antigen expressed in up to 15 epithelial cancers.

Preclinical xenograft animal models of pancreatic, lung, gastric and breast cancers showed OBI-999 induced profound tumor shrinkage at various doses. The agent also appeared well-tolerated with a favorable safety profile in preclinical single and repeated dose toxicology studies.

Researchers have initiated enrollment in a phase 1/phase 2 clinical trial to assess the safety and preliminary efficacy of OBI-999 among patients with locally advanced or metastatic solid tumors, including pancreatic, gastric, colorectal and esophageal cancers.

“We are very excited about the potential value that OBI-999 may provide to patients with pancreatic cancer, given both the high potency we have observed using OBI-999 in pancreatic cancer xenograft models and because many pancreas cancers highly overexpress Globo H, the glycolipid target of OBI-999, using the validated immunohistochemistry assay that will be available for selecting patients for the phase 2 portion of this first-in-human clinical trial,” Tillman Pearce, MD, chief medical officer of OBI Pharma, said in the press release.

The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.