CID-078 Receives FDA Orphan Drug Designation in Small Cell Lung Cancer
US FDA
- CID-078 has been granted FDA orphan drug designation for the treatment of small cell lung cancer (SCLC).
- SCLC is an aggressive cancer with limited treatment options, particularly in relapsed/refractory settings.
- The orphan drug designation provides incentives for CID-078's development, including market exclusivity and tax credits, as the drug progresses through a phase 1 clinical trial for advanced solid tumors, including SCLC.
The investigational therapeutic candidate CID-078 has been granted orphan drug designation by the FDA for the treatment of SCLC. This designation underscores the significant unmet medical need in SCLC and provides critical incentives to accelerate the development of CID-078, which represents a novel approach to targeting this aggressive malignancy.
“The orphan drug designation from the FDA underscores both the seriousness of small cell lung cancer and the lack of effective treatment options,” said Michael C. Cox, PharmD, MHSc, BCOP, senior vice president and head of early development at Circle Pharma, in a press release. “We are committed to accelerating the clinical development of CID-078 to offer new hope for patients who face limited therapeutic choices.”
The FDA’s orphan drug designation program is designed to encourage the development of drugs for rare diseases or conditions that affect fewer than 200,000 people in the United States. While SCLC itself affects more than 200,000 people,2 the specific criteria for orphan drug designation can also apply to a subset of a common disease that meets the rare disease prevalence, or if there is no reasonable expectation that the cost of developing and making available the drug for such disease or condition will be recovered from sales in the US.3
For CID-078 in SCLC, this designation provides several important development incentives. These include 7 years of market exclusivity upon regulatory approval, tax credits for qualified clinical trial costs, and eligibility to apply for FDA-administered research grants. These provisions aim to offset the financial challenges often associated with developing therapies for smaller patient populations or difficult-to-treat diseases.
CID-078 is currently being evaluated in a phase 1 clinical trial (NCT06577987). The trial is designed to assess the safety, tolerability, pharmacokinetics, and preliminary signs of antitumor activity of CID-078 in patients with advanced solid tumors, including those with SCLC.4 The study is currently recruiting an estimated 220 patients aged 12 years or older across 10 sites in the US.
Small cell lung cancer is a particularly aggressive form of lung cancer, accounting for approximately 13%-15% of all lung cancer diagnoses.2 It is characterized by rapid growth and early metastasis and has a strong association with tobacco exposure. Despite initial responsiveness to chemotherapy and radiation, most patients with SCLC experience relapse, often with resistant disease, leading to a median survival that remains poor, particularly for those with extensive-stage disease. The limited therapeutic options in the relapsed and refractory settings highlight the urgent need for innovative treatments.
