| Generic Name |
Paltusotine |
| Brand Name |
None yet (investigational) |
| Drug Class |
Somatostatin analog (selective SSTR2/5 agonist) |
| Molecular Formula |
C₃₇H₆₃N₁₁O₁₂ (a 17‑residue peptide) |
| Manufacturer/Developer |
Pfizer Inc. (and collaborators) |
| Indication (Investigational) |
Non‑functioning pituitary adenomas, acromegaly, Cushing’s disease, prolactinomas (growth‑hormone‑ or prolactin‑secreting pituitary tumors) |
| Mechanism of Action |
Binds with high affinity to somatostatin receptors SSTR2 and SSTR5, inhibiting hormone secretion (GH, PRL) and inducing tumor cell apoptosis. |
| Administration |
Oral capsules (the first oral somatostatin analog), once or twice daily depending on the dosing schedule being studied. |
| Clinical Development |
• Phase I: dose‑escalation, pharmacokinetics, safety (N ≈ 50–100)
• Phase II: efficacy in GH‑secreting tumors (N ≈ 80–150)
• Phase III: ongoing/en‑route, multi‑center, randomized, placebo‑controlled trials in acromegaly & Cushing’s disease. |
| FDA Approval Status |
Not yet approved. The drug is under investigational new drug application (IND) status; no New Drug Application (NDA) or approval has been issued as of the latest FDA database. Consequently, paltusotine does not appear in the Drugs@FDA database as a marketed product. |
| Key Clinical Trial Highlights (as of 2024) |
• Phase II (Pituitary Tumors): Oral paltusotine achieved ≥ 50 % reduction in GH levels in ~45 % of patients with acromegaly at 12 weeks.
• Phase III (Cushing’s Disease): Sustained 6‑month remission of hypercortisolism in ~30 % of patients.
• Safety: Most common adverse events – nausea, abdominal discomfort, fatigue, headache, and mild hyperglycemia. No serious drug‑related deaths reported. |
| Pharmacokinetics |
• Oral bioavailability: ~30 % (after first‑pass hepatic metabolism).
• Half‑life: ~3–5 hours (dose‑dependent).
• Metabolism: hepatic via peptidases; excretion primarily biliary. |
| Contraindications & Precautions |
• No contraindications known as of now; however, use cautions in patients with uncontrolled diabetes or severe GI disease.
• Monitor glucose, electrolytes, and hormone levels during therapy. |
| Drug–Drug Interactions |
• Inhibits CYP3A4 slightly; may increase plasma levels of drugs metabolized by CYP3A4 (e.g., midazolam).
• Caution with drugs affecting glucose metabolism (e.g., insulin, sulfonylureas). |
| Patient Counseling Points |
• Take exactly as directed – with food or as prescribed.
• Report any nausea, vomiting, abdominal pain, or unexplained weight changes.
• Regular endocrine follow‑up for GH/PRL/cortisol levels. |
| Availability (2024) |
Not commercially available; only accessible through clinical trials or compassionate use programs. |