Megestrol Acetate

Evidence Level: L5

Predicted Indications: 7

Megestrol Acetate

Pharmacist Assessment Report

Megestrol Acetate: Hereditary breast ovarian cancer syndrome

One-Sentence Summary

Megestrol acetate is a synthetic progestin originally used to treat endometrial carcinoma. Evidence for this top prediction is 1 clinical trial (a completed randomized Phase 2 study) and no directly linked publications.

Quick Overview

Item	Content
Original Indication	Not recorded in the local data
Predicted New Indication	Hereditary breast ovarian cancer syndrome
TxGNN Prediction Score	0.9989
Evidence Level	L5
India Market Status	Listed in a compiled Indian medicine price dataset (at least 20) – CDSCO approval status is not included in this data
Number of Listed Products	at least 20
Recommended Decision	Proceed with Guardrails

Why is This Prediction Reasonable?

Currently, detailed mechanism of action data is not available. Based on known information, megestrol acetate is a synthetic progestin. Progestins act through progesterone receptor signaling to induce differentiation and suppress proliferation in hormone-sensitive endometrial tumors.

The predicted indication is the same disease family as the original one. The high score therefore reflects consistency with known use rather than a new therapeutic direction. A randomized Phase 2 trial in advanced or recurrent endometrial carcinoma includes a megestrol acetate (with tamoxifen) hormonal arm, which supports the clinical relevance of this use.

Clinical Trial Evidence

Trial Number	Phase	Status	Enrollment	Key Findings
NCT00729586	Phase 2	Completed	73	Randomized trial of temsirolimus alone or combined with hormonal therapy (megestrol acetate plus tamoxifen) in advanced, persistent or recurrent endometrial cancer. The main question concerns temsirolimus, with the hormonal regimen as a comparator/combination arm.

Literature Evidence

Currently no related literature available for this top prediction.

India Market Information

Twenty valid single-ingredient registrations are on record. The main ones are listed below; no approved-indication text was available in the data compiled for this report.

Authorization Number	Product Name	Dosage Form	Approved Indication
136498	Mhega 160mg Tablet	Strip of 10 tablets	Not available
134685	Megeetron 160mg Tablet	Strip of 10 tablets	Not available
133810	Megesta 160 Tablet	Strip of 10 tablets	Not available
37822	Cachexiam 160 Tablet	Strip of 10 tablets	Not available
72436	Endace 40 Tablet	Strip of 10 tablets	Not available

All products are oral tablets (40, 80 or 160 mg strengths).

Cytotoxicity

Megestrol acetate is a hormonal (endocrine) antineoplastic agent, not a conventional cytotoxic drug.

Item	Content
Cytotoxicity Classification	Hormonal therapy (progestin); not a conventional cytotoxic
Myelosuppression Risk	Please refer to the package insert warnings and precautions
Emetogenicity Classification	Please refer to the package insert warnings and precautions
Monitoring Items	Please refer to the package insert; blood glucose, blood pressure, and liver and renal function are reasonable given the associated conditions below
Handling Protection	Please refer to the package insert warnings and precautions

Safety Considerations

Contraindication-related conditions (from the knowledge graph, not label text): hypertensive disorder, kidney disease, liver disease, gallbladder disease, hyperglycemia, diabetes mellitus, diabetic ketoacidosis, adrenocortical insufficiency, Addison disease, male hypogonadism, premature menopause, androgen insensitivity syndrome.
Adrenal suppression: A published study (PMID 10491532) examined secondary adrenal suppression in patients on megestrol acetate for advanced cancer.
Drug Interactions: 134 interactions were identified, mostly Moderate. The listed ones are largely antidiabetic agents, such as metformin, pioglitazone, glimepiride, repaglinide, sitagliptin, semaglutide, empagliflozin, dapagliflozin, canagliflozin and insulins. Others include aprepitant and clarithromycin. Glucose monitoring is advisable when combined with antidiabetic drugs.

Package insert warnings and contraindications were not available, so please refer to the package insert for complete safety information.

Conclusion and Next Steps

Decision: Proceed with Guardrails

Rationale: The predicted indication is effectively the drug’s original use. A completed randomized Phase 2 trial includes a megestrol acetate arm in endometrial cancer. However, the trial’s main question concerned temsirolimus, and no directly linked publications were supplied. Important safety information is also missing.

To proceed, the following is needed:

Package insert warnings and contraindications for the marketed Indian products (currently a blocking gap)
Detailed mechanism of action data
Approved indication text for the Indian registrations
Efficacy data focused on megestrol acetate in endometrial carcinoma
A glucose, blood pressure and adrenal function monitoring plan, given the knowledge-graph contraindications and interactions

Other predictions (for reference):

Ovarian cancer (score 0.9971, L1 by trial count): this is a research question rather than a go. The Phase 3 trial (NCT00005975) studied megestrol acetate for hot flashes after breast cancer treatment, not as anti-tumor therapy. Anti-tumor support is limited to older Phase 2 studies of high-dose megestrol acetate in platinum-refractory disease (PMIDs 11593052, 9856656, 3099393).
Uterine carcinosarcoma: only indirect literature (a hormone receptor expression study and a case report), so it remains a research question.
Other predictions (endometrial transitional cell carcinoma, hereditary breast ovarian cancer syndrome, borderline ovarian tumor, placenta cancer, ovarian clear cell adenocarcinoma, ovarian dysgerminoma, low-grade uterine neuroendocrine tumor): model prediction only with no clinical evidence, so Hold.
Disclaimer

This content is for research purposes only and does not constitute medical advice. Clinical validation is required before any clinical application.