Have you ever heard of Orphan Drug designation? This designation is intended to spur research and development into drugs or biologics for rare diseases. Orphan Drug designation is granted to therapies intended to treat, diagnose, or prevent rare conditions; rare means affecting fewer than 200,000 Americans. As a benefit, drug developers receive incentives such as fee waivers, tax credits, and 7 years of market exclusivity upon approval. An August 19 article from OncLive shares that a combination capsule of senaparib and temozolomide received Orphan Drug designation for the treatment of adults with small cell lung cancer (SCLC).
Study Results
The Orphan Drug designation comes following results from the Phase 1b/2 study evaluating the combination treatment for SCLC. Altogether, 14 patients with advanced solid tumors enrolled in the dose escalation portion of the trial, and were split into four cohorts. Patients either received 40mg senaparib and 20mg temozolomide; 60mg senaparib and 20mg temozolomide; 80mg senaparib and 20mg temozolomide; or 80mg senaparib and 30mg temozolomide. To enroll, patients must not have responded to other therapeutic options.
Out of 4 evaluable patients, researchers found that:
- The median time to reach a response was 7.3 months, with a median response duration of 3.6 months. Disease control rate was found to be 83.3%.
- 3 patients out of the 4 evaluable experienced partial responses.
- 80mg senaparib and 20mg temozolomide was found to be both the maximum tolerated dose and the recommended dose for future studies.
Additionally, 14 patients with late-stage SCLC are also enrolled for part two of the study. Within this patient group, there were 7 evaluable patients. 42.9% saw objective responses.
Adverse reactions did occur, including nausea, fatigue, anemia (low red blood cell count), neutropenia (low white blood cell count), and thrombocytopenia (low platelet count). However, the therapy was still found to be relatively safe and well-tolerated.
Researchers are continuing to recruit patients for the trial’s second phase.
About Small Cell Lung Cancer (SCLC)
Small cell lung cancer (SCLC) is an aggressive and fast-growing form of lung cancer. SCLC is estimated to account for around 15% of all lung cancer diagnoses. It is typically considered to be either small cell carcinoma or combined small cell carcinoma. While researchers are unsure of exactly what causes SCLC, risk factors include smoking, being older in age, a family history of lung cancer, secondhand smoke exposure, radiation or chemical exposure, HIV, or air pollution. Typically, symptoms do not appear until later stages of this cancer. When symptoms do appear, they can (but do not always) include:
- Fatigue
- Chest pain
- Chronic cough (that may produce blood)
- Difficulty breathing
- Wheezing
- Facial swelling
- Swollen neck veins
- Unintended weight loss
- Hoarseness
- Appetite loss
- Pleural effusion
