The Orphan Drug Act of 1983 was designed to facilitate the development of orphan drugs, or drugs designed for patients with rare or life-threatening conditions. Now, the FDA grants Orphan Drug designation to drugs or biologics which treat, prevent, or diagnose a rare condition; these are defined as conditions affecting fewer than 200,000 Americans. This designation comes with benefits for drug developers, such as fee waivers, tax credits, and 7 years of market exclusivity upon approval. According to an article published in Cancer Network, SY-5609, in conjunction with chemotherapy, was recently granted Orphan Drug designation for the treatment of those with relapsed metastatic prostate cancer.
Drug developer Syros Pharmaceuticals, Inc. (“Syros”) describes SY-5609 as:
a highly selective and potent oral inhibitor of the cyclin-dependent kinase 7 (CDK7) in development for select solid tumors. SY-5609 represents a novel targeted approach that we believe has potential in a range of difficult-to-treat cancers.
Currently, researchers are evaluating SY-5609 and chemotherapy in an ongoing Phase 1 study for patients with advanced solid tumors, including pancreatic cancer, breast cancer, and small cell lung cancer (SCLC). Patients within this study have progressed while on leucovorin calcium, fluorouracil, irinotecan hydrochloride, and oxaliplatin therapy. Altogether, 160 patients will enroll. During the study, researchers hope to determine the safety, efficacy, and tolerability of SY-5609 alone and in combination with other therapies (fulvestrant, gemcitabine, nab-paclitaxel).
What is Pancreatic Cancer?
As its name suggests, pancreatic cancer forms in the pancreas, an organ in your abdomen that sits just behind your stomach. Normally, the pancreas releases enzymes that aid in food digestion and hormones that aid in blood sugar management. There are multiple forms of pancreatic cancer depending on where, specifically, the cancer develops. For example, some forms of pancreatic cancer include Islet cell tumors and pancreatic adenocarcinoma. Risk factors include having a family history of pancreatic cancer or certain genetic syndromes, pancreatitis, diabetes, smoking cigarettes, being older in age (65+), or being male.
Unfortunately, pancreatic cancer is mostly asymptomatic in earlier stages. Because of this, many patients do not get diagnosed until the cancer has progressed. When symptoms appear, these can (but do not always) include:
- Newly onset or worsening diabetes
- Abdominal pain that may radiate to the back
- Unintended weight loss
- Jaundice (yellowing of the skin, eyes, and mucous membranes)
- Dark urine and light stools
- Fatigue
- Appetite loss
- Itchy skin
- Bowel obstruction
- Depression and/or anxiety
- Blood clots
