In the United States, the FDA grants Orphan Drug designation to drugs or biologics intended to treat, diagnose, or prevent rare conditions. A rare disease or condition is defined as one affecting under 200,000 Americans. As a benefit, the drug developer also receives incentives such as fee waivers, increased regulatory assistance, tax credits, and seven years of market exclusivity upon drug approval. In a news release from biopharmaceutical company Oryzon Genomics, S.A. (“Oryzon”), the company shared that its drug candidate iadademstat, designed for those with small cell lung cancer (SCLC), earned this designation.
Outside of SCLC, iadademstat has also received Orphan Drug designation for acute myeloid leukemia (AML) in both the United States and Europe.
About Iadademstat
So what exactly is iadademstat? According to Oryzon, iadademstat is:
a small oral molecule, which acts as a covalent and highly selective inhibitor of the epigenetic enzyme Lysine Specific Demethylase-1, LSD1 (DKM1A). Among other biological roles, LSD1 is involved in stemness in cancer and high levels of LSD1 are known to correlate with more aggressive types of cancer and poorer prognosis for patients.
This epigenetic therapy works to reprogram tumor cell genomics while strengthening the immune system so that it can destroy the SCLC. So far, in clinical studies of iadademstat, research has shown that the treatment is relatively safe and well-tolerated. Further studies are ongoing.
What is Small Cell Lung Cancer (SCLC)?
An estimated 15% of lung cancer diagnoses are small cell lung cancer (SCLC), a rare and aggressive cancer. Doctors are not exactly sure what causes SCLC. However, medical professionals do know that a family history of lung cancer, HIV, secondhand smoke or radiation exposure, air pollution, smoking cigarettes, or being older in age increase the risk of developing SCLC. Two main forms of SCLC exist: combined small cell carcinoma and small cell carcinoma. When symptoms of SCLC appear, these can include:
- Fatigue
- Unintended weight loss
- Appetite loss
- A hoarse voice
- Wheezing and/or a chronic cough
- Chest pain or discomfort
- Difficulty breathing
- Facial swelling and/or swollen neck veins
- Coughing up blood
- Pleural effusion
