The US Food and Drug Administration (FDA) has granted orphan drug designation for Lisata Therapeutics’s LSTA1 (certepetide) for the treatment of osteosarcoma, a rare type of blood cancer.

The US-based pharma company will be eligible for tax credits for qualifying US-based clinical trials and, pending approval, seven years of market exclusivity in the designated indication.

Orphan drug designations are handed out by the FDA for drugs or biologics that target diseases that affect fewer than 200,000 people in the US.

Osteosarcoma is most often diagnosed in children and young adults, accounting for 2% of all cancers in those aged up to 14 years old, and 3% of all cancers in teens aged 15-19 years old.

LSTA1 is designed to increase the efficacy of co-administered anti-cancer drugs penetrating solid tumours. The cyclic peptide activates the CendR uptake pathway that allows bounded medicine to accumulate in the tumour. Lisata says its compound can also modify the tumour microenvironment to make tumours more susceptible to immunotherapies.

The orphan drug designation follows an FDA rare paediatric disease designation earlier this month and fast track designation awarded in 2022. The former could point to the company receiving a priority voucher upon approval while the latter could potentially expedite development and review. Also with orphan drug status for osteosarcoma is Radiopharm Theranostics’s monoclonal antibody DUNP19. The Australian biotech received the designation in September 2022.

Lisata is also targeting other cancers with its asset. LSTA1 has FDA orphan drug status for glioblastoma multiform treatment, as well as FDA and European orphan status for pancreatic cancer.

According to analysis by GlobalData, the drug is being evaluated across several Phase II clinical trials, including the Phase II BOLSTER study (NCT05712356) for solid tumour treatment, the Phase II ASCEND trial for pancreatic cancer treatment, and a Phase II trial for patients with newly diagnosed glioblastoma multiforme (GBM).

“We are thrilled to have received another favourable regulatory designation from the FDA. Osteosarcoma, while rare, is a type of bone cancer that is often associated with high morbidity, early metastasis, rapid progression, and poor prognosis. Receiving an orphan drug designation from the FDA is an important milestone as we plan for future clinical expansion of LSTA1,” said Lisata’s chief medical officer, Dr Kristen Buck in a 9 April press release.