FDA Grants Orphan Drug Status to LP-184 for Pancreatic Cancer

The FDA has granted an orphan drug designation to LP-184 as a potential therapeutic option for patients with pancreatic cancer.

The FDA has granted an orphan drug designation to LP-184 as a potential therapeutic option for patients with pancreatic cancer, according to an announcement from Lantern Pharma.1

The next-generation alkylating agent was designed to preferentially damage DNA in cancer cells that overexpress certain biomarkers or that harbor mutations in DNA repair pathways. The agent is under development for several cancer indications beyond this disease.

“Receipt of orphan drug designation is an important accomplishment for the LP-184 program and for our company. This designation further validates our data-driven approach to oncology drug development as well as the groundbreaking collaborative R&D approach we are advancing with leading institutions such as Fox Chase Cancer Center,” Panna Sharma, president and chief executive officer of Lantern Pharma, stated in a press release. “Orphan drug designation is designed to provide a number of benefits, including 7 years of market exclusivity, which complements our growing portfolio of patents that provide us additional commercial and market protections.”

Preclinical findings have shown that treatment with LP-184 resulted in a significant and rapid tumor shrinkage by over 90% in in-vivo mouse models in 8 weeks.2 In contrast, tumors in the untreated mice grew by over eleven-fold in volume during the same time period.

Moreover, the efficacy of the agent was also demonstrated in 6 pancreatic cancer cell lines and 5 more patient-derived xenograft ex-vivo tumor models. Across all cell lines and models, LP-184 resulted in a substantial reduction of cancer cells and tumor cell growth; IC50 values were in the nanomolar range of 45 nM to 270 nM.

“The orphan designation is one of any upcoming milestones that we expect to achieve for our LP-184 program in pancreatic cancer. We recently reported that LP-184 demonstrated significant and rapid pancreatic tumor shrinkage, by over 90%, within in-vivo mouse models over 8 weeks…We are excited to advance this groundbreaking research to help patients suffering from this devastating disease where the benefits of current treatment options are very limited.”

The clinical-stage oncology-focused biopharmaceutical company has announced that they have begun to discuss the design of first-in-human clinical trials for the agent.


  1. FDA grants Lantern Pharma orphan drug designation for drug candidate LP-184 in the treatment of pancreatic cancer. News release. Lantern Pharma. August 11, 2021. Accessed August 16, 2021. https://prn.to/3gbw3uG
  2. Lantern Pharma announces significant positive preclinical data in pancreatic cancer with drug candidate LP-184. News release. Lantern Pharma. July 20, 2021. Accessed August 16, 2021. https://bit.ly/3yV3hWL