The US FDA Has Granted the Orphan Drug Status to Lumosa’s LT2003, an Anti-tumor Targeting Fusion Protein, for the Treatment of Pancreatic Cancer

(March 2, 2020, Taipei) Lumosa Therapeutics Co., Ltd.  announced today that it had received Orphan Drug Designation (no. DRU-2019-7291) from the US Food and Drug Administration (FDA) for its LT2003, an anti-tumor targeting fusion protein, for the treatment of pancreatic cancer. LT2003 is a cancer targeting therapy capable of enzyme-prodrug conversion. LT2003 can target specific types of tumor cells and converts non-cytotoxic prodrugs to deliver concentrated potent cytotoxic drugs precisely at the tumor site efficiently without concerns in systemic side effects.

Results of animal pharmacodynamics study conducted by Lumosa showed that LT2003 achieved statistical significance (p<0.05) in several cancer categories when compared to conventional therapy. LT2003 demonstrated significant tumor suppressing capability and animals receiving LT2003 showed no severe adverse effects.

The US FDA encourages companies to develop drugs for rare diseases through the orphan drug designation by awarding the product with a 7-year marketing exclusivity upon approval, regardless of patent status. In drug development perspective, the designation allows the drug developers the chance to receive consultations for clinical development, or incentives to shorten the time to market; such as expedited review in Fast-track designation or shortened reviewing period in Priority Review designation (from 10 months reviewing period to 6 months). Other benefits include reimbursements for research and development costs, waiver for drug application fees and tax reduction.

Many best-selling drugs obtained orphan drug designation before receiving marketing approval. Anti-tumor drugs such as BMS’s Opdivo or Merck’s Keytruda used the orphan drug status to gain market authorization first before extending their indications to other categories or expand drug sales to other markets. Besides the US, all major pharmaceutical markets offer similar orphan-drug regulatory pathway to help companies in subsequent clinical trial or licensing negotiations.

After the orphan drug designation is granted, Lumosa will take the opportunity to propel LT2003 to the clinical stage through licensing or co-development model with potential partners and shorten the time needed to market.