Nirogacestat: Uses, Side Effects, Dosage - Drugvu

ℹ️ Compiled from public regulatory records · Last regulator revision: May 6, 2026🚩 Report this page

Nirogacestat

Other Antineoplastic Agents

Sold as Ogsiveo

GB MHRAEU EMA

Drug class: Other Antineoplastic Agents
Availability: See label
Markets covered: 2
Products on record: 5

Overview

Nirogacestat is an active pharmaceutical ingredient in the Other Antineoplastic Agents group (L01XX). The information below is compiled per regulator from the product labels on record, with direct links to the original documents.

Regulatory status by market

Market	Regulator	Products	Last revision
GB United Kingdom	MHRA	4	March 13, 2026
EU European Union	EMA	1	May 6, 2026

GBUnited Kingdom· MHRA

4 products

Uses

GBOfficial regulatory label· revised March 13, 2026[1]

Ogsiveo as monotherapy is indicated for the treatment of adult patients with progressing desmoid tumours who require systemic treatment.

How to take

EUEuropean Union· EMA

1 product

Uses

EUOfficial regulatory label· revised May 6, 2026[2]

Ogsiveo as monotherapy is indicated for the treatment of adult patients with progressing desmoid tumours who require systemic treatment.

How to take

Sources & citations

[1]MHRA (UK) · PL116480298 · revised March 13, 2026
[2]European Medicines Agency · EMEA/H/C/006071 · revised May 6, 2026

Information on this page is compiled from public regulatory records. Drugvu is not affiliated with any regulator or pharmaceutical manufacturer. This is not medical advice. Always consult a qualified healthcare professional.

Ogsiveo should be initiated and monitored by a physician experienced in the use of anticancer therapies. Posology The recommended dose is 150 mg Ogsiveo twice daily, one dose in the morning and one dose in the evening. This dose should not be exceeded.
Duration of treatment Ogsiveo should be continued until disease progression or unacceptable toxicity. Missed dose If a dose of Ogsiveo is missed, patients should not take an additional dose. Patients should take the next prescribed dose.
Dose adjustments for adverse reactions The recommended dose modifications for selected adverse reactions are provided in Table 1. For other severe adverse reactions, or in the event of life-threatening adverse reactions, Ogsiveo should be withheld until the reaction is resolved to Grade ≤ 1 or baseline.
Ogsiveo should only be restarted at a dose of 100 mg twice daily and only after carefully considering the potential benefit and likelihood of recurrence of the adverse reaction. Ogsiveo should be permanently discontinued for recurrence of severe or life-threatening adverse reaction upon rechallenge at the reduced dose.
Dose modifications should be made if patients experience the following adverse reactions (grades refer to Common Terminology Criteria for Adverse Events): Table 1: Recommended dose modifications for adverse reactions in patients treated with Ogsiveo Adverse reaction Recommended action Diarrhoea Grade 3 diarrhoea persisting for ≥ 3 days despite maximal medical therapy Ogsiveo should be withheld until reaction is resolved to Grade ≤ 1 or baseline, then it should be restarted at a dose of 100 mg twice daily.
Skin reactions Grade 3 folliculitis Ogsiveo should be withheld until reaction is resolved to Grade ≤ 1 or baseline, then it should be restarted at a dose of 100 mg twice daily. Grade 3 maculopapular rash Ogsiveo should be withheld until reaction is resolved to Grade ≤ 1 or baseline, then it should be restarted at a dose of 100 mg twice daily.
Grade 3 hidradenitis Ogsiveo should be withheld until reaction is resolved to Grade ≤ 1 or baseline, then it should be restarted at a dose of 100 mg twice daily. Electrolyte abnormalities Grade 3 hypophosphataemia persisting for ≥ 7 days despite maximal replacement therapy Ogsiveo should be withheld until reaction is resolved to Grade ≤ 1 or baseline, then it should be restarted at a dose of 100 mg twice daily.
Grade 3 hypokalaemia despite maximal replacement therapy Ogsiveo should be withheld until reaction is resolved to Grade ≤ 1 or baseline, then it should be restarted at a dose of 100 mg twice daily. Hepatic abnormalities Alanine transaminase (ALT) or Aspartate transaminase (AST) ≥ 3 to 5 Ogsiveo should be withheld until ALT, AST, or both are resolved to < 3 x ULN x ULN or baseline, then it should be restarted at a dose of 100 mg twice daily.
ALT or AST > 5 x ULN Ogsiveo should be permanently discontinued. Other adverse reactions Anaphylaxis or other severe hypersensitivity reaction Ogsiveo should be permanently discontinued. Special populations Elderly population No dose adjustment is recommended for patients who are aged 65 years or over.
Clinical data in patients aged 65 years or over is limited. Renal impairment No dose adjustment is recommended in patients with mild or moderate renal impairment. 2). Hepatic impairment No dose adjustment is recommended in patients with mild or moderate hepatic impairment.
2). Paediatric population The safety and efficacy of Ogsiveo in children from 2 to 18 years of age have not been established. Ogsiveo should not be used in children from birth to less than 2 years of age because of potential safety concerns related to structural and functional growth.
1, but no recommendation on a posology can be made. Method of administration Ogsiveo is for oral use. The tablets may be taken with or without food. Tablets should not be broken, chewed or crushed because there are no data currently available to support other methods of administration.
5).

Side effects & warnings

GBOfficial regulatory label· Adverse reactions· revised March 13, 2026[1]

Summary of the safety profile The most common adverse reactions are: diarrhoea (85%), rash (65%), ovarian toxicity in women of childbearing potential (60%), nausea (59%), fatigue (50%), hypophosphataemia (50%), headache (40%), and stomatitis (40%).
The most frequently reported serious adverse reaction was ovarian toxicity (premature menopause, 3%). The most common severe adverse reactions were diarrhoea (16%) and hypophosphataemia (13%). Permanent discontinuation of nirogacestat due to an adverse event occurred in 19% of patients.
The most common adverse reactions leading to discontinuation were diarrhoea (5%), ovarian toxicity (5%), and increased ALT (3%). The frequency of dose interruption of nirogacestat due to adverse reactions was 59%. The most common adverse reactions leading to dose interruption were diarrhoea (11%), rash maculo-papular (10%), hypophosphatemia (6%) and nausea (5%).
The frequency of dose reduction of nirogacestat due to adverse reactions was 44%. The most common adverse reactions leading to dose reduction were diarrhoea (9%), rash maculo-papular (6%), stomatitis (3%), and hypophosphatemia (3%).
5 months in clinical studies. The adverse reactions are ranked under heading of frequency using the following convention: very common (≥ 1/10), common (≥ 1/100 to < 1/10), uncommon (≥ 1/1 000 to < 1/100), rare (≥ 1/10 000 to < 1/1 000), very rare (< 1/10 000) and not known (cannot be estimated from the available data).
Within each frequency grouping, adverse reactions are presented in order of decreasing seriousness.
Table 2:
Adverse reactions reported System organ class Adverse reaction All grades Grades 3-4 Diarrhoea Very common Very common Nausea Very common Common Stomatitisa Very common Common Gastrointestinal disorders Dry mouth Very common -- Rashb Very common Common Alopecia Very common -- Folliculitis Very common Common Hidradenitis Common Common Dry skin Very common -- Skin and subcutaneous disorders Pruritis Very common -- Basal cell carcinoma Common --Neoplasms benign, malignant and unspecified Squamous cellc carcinoma Common -- Hypophosphataemia Very common Very commonMetabolism and nutrition disorders Hypokalaemia Very common Common Headache Very common --Nervous system disorders Dizziness Very common -- Proteinuria Very common --Investigation Glycosuria Very common -- System organ class Adverse reaction All grades Grades 3-4 Blood and lymphatic system disorders Eosinophilia Very common -- Renal and urinary disorders Renal tubular disorder Common -- Injury, poisoning and procedural complications Bone fractured Common -- ALT increased Very common CommonHepatobiliary disorders AST increased Very common Common Reproductive system and breast disorders Ovarian toxicitye Very common -- Cough Very common -- Upper respiratory tract infectionf Very common -- Dyspnoea Very common -- Respiratory, thoracic and mediastinal disorders Epistaxis Very common -- Fatigue Very common CommonGeneral disorders and administration site conditions Influenza-like illness Very common -- a Stomatitis includes stomatitis, mouth ulceration, oral pain, and oropharyngeal pain.
b Rash includes rash maculo-papular, dermatitis acneiform, rash, rash erythematous, rash pruritic, and rash papular. c Squamous cell carcinoma included squamous cell carcinoma of skin and squamous cell carcinoma. d Bone fracture includes fracture, foot fracture, hand fracture, radius fracture, hip fracture and rib fracture.
e Ovarian toxicity includes ovarian failure, premature menopause, amenorrhoea, oligomenorrhoea, menstruation irregular, dysmenorrhoea, heavy menstrual bleeding, vulvovaginal dryness, hot flush, decreased anti-Müllerian hormone (AMH) and increased follicle-stimulating hormone (FSH).
f Upper respiratory tract infection (URTI) includes URTI, viral URTI, acute sinusitis, and sinusitis -- Represents no cases were reported. Description of selected adverse reactions The data described below reflect results of the randomised, double-blind, Phase 3 DeFi study in patients with desmoid tumours treated with 150 mg BID nirogacestat (N=69) or placebo (N=72) twice daily.
Diarrhoea In the double-blind phase of the DeFi study, diarrhoea was reported in 84% of patients receiving nirogacestat compared to 35% in patients receiving placebo. 4). Grade ≤ 2 diarrhoea resolved in 74% of patients who continued on nirogacestat treatment.
The median time to first onset of diarrhoea in patients receiving nirogacestat was 9 days (range 2 to 234 days). Diarrhoea led to dose reduction in 10% of patients and treatment discontinuation in 7% receiving nirogacestat. 4). The median time to rash events was 22 days (range 2 to 603 days).
Skin and subcutaneous disorders led to dose reduction in 9% of patients receiving nirogacestat, including maculo-papular rash in 4% and hidradenitis in 3%. Maculo-papular rash led to treatment discontinuation in 1%. Ovarian toxicity In the double-blind phase of the DeFi study, 75% of women of childbearing potential receiving nirogacestat reported ovarian toxicity (defined as ovarian failure, premature menopause, amenorrhea, oligomenorrhea, and menopause) compared to no patients receiving placebo.
There were three serious adverse reactions of ovarian toxicity, all premature menopause, representing 11% of all participants reporting ovarian toxicity. The […]

GBOfficial regulatory label· Warnings and precautions· revised March 13, 2026[1]

8). Patients who experience diarrhoea during treatment with nirogacestat should be monitored and managed using anti-diarrhoeal medicinal products. 2). 8). Patients should be monitored for dermatologic reactions throughout the course of treatment and managed as clinically indicated.
2). 8). Ovarian toxicity, identified based on abnormal reproductive hormone levels or peri-menopausal symptoms, was reported in 75% of women of childbearing potential receiving nirogacestat in the DeFi study. Ovarian toxicity has been reported to resolve in 79% of women of childbearing potential during treatment.
8). Effects of nirogacestat on human fertility are unknown. Based on findings from animal studies, female fertility may be impaired. Women of childbearing potential should be advised about the risk of ovarian toxicity before initiating treatment with nirogacestat.
Patients should be monitored for changes in menstrual cycle regularity or the development of symptoms of oestrogen deficiency, including hot flashes, night sweats, and vaginal dryness. 8). Phosphate and potassium levels should be monitored regularly and supplemented as necessary.
2). 2). 8). Liver function tests should be monitored regularly. For ALT or AST ≥ 3 to 5 x ULN, nirogacestat should be withheld until ALT, AST, or both are resolved to < 3 x ULN or baseline, then it should be restarted at a dose of 100 mg twice daily.
2). 8). Skin examinations should be performed prior to initiation of nirogacestat and routinely during treatment with nirogacestat. Cases should be managed according to clinical practices and patients may continue with nirogacestat treatment without dose adjustment.
3). Patients should be advised of the potential risk to a foetus. Women of childbearing potential must have a negative pregnancy test prior to initiating nirogacestat treatment. Pregnancy testing during treatment with nirogacestat should be considered for women of childbearing potential experiencing amenorrhoea.
6). Women of childbearing potential should be advised to inform their healthcare provider immediately of a known or suspected pregnancy, and they must stop taking nirogacestat if they become pregnant. 6). 1). Patients with rare hereditary problems of galactose intolerance, total lactase deficiency or glucose- galactose malabsorption should not take this medicinal product.

This is not medical advice. Consult a qualified healthcare professional.

Ogsiveo should be initiated and monitored by a physician experienced in the use of anticancer therapies. Posology The recommended dose is 150 mg Ogsiveo twice daily, one dose in the morning and one dose in the evening. This dose should not be exceeded.
Duration of treatment Ogsiveo should be continued until disease progression or unacceptable toxicity. Missed dose If a dose of Ogsiveo is missed, patients should not take an additional dose. Patients should take the next prescribed dose.
Dose adjustments for adverse reactions The recommended dose modifications for selected adverse reactions are provided in Table 1. For other severe adverse reactions, or in the event of life‑threatening adverse reactions, Ogsiveo should be withheld until the reaction is resolved to Grade ≤ 1 or baseline.
Ogsiveo should only be restarted at a dose of 100 mg twice daily and only after carefully considering the potential benefit and likelihood of recurrence of the adverse reaction. Ogsiveo should be permanently discontinued for recurrence of severe or life-threatening adverse reaction upon rechallenge at the reduced dose.
Dose modifications should be made if patients experience the following adverse reactions (grades refer to Common Terminology Criteria for Adverse Events): Table 1: Recommended dose modifications for adverse reactions in patients treated with Ogsiveo Adverse reaction Recommended action Diarrhoea Grade 3 diarrhoea persisting for ≥ 3 days despite maximal medical therapy Ogsiveo should be withheld until reaction is resolved to Grade ≤ 1 or baseline, then it should be restarted at a dose of 100 mg twice daily.
Skin reactions Grade 3 folliculitis Ogsiveo should be withheld until reaction is resolved to Grade ≤ 1 or baseline, then it should be restarted at a dose of 100 mg twice daily. Grade 3 maculopapular rash Ogsiveo should be withheld until reaction is resolved to Grade ≤ 1 or baseline, then it should be restarted at a dose of 100 mg twice daily.
Grade 3 hidradenitis Ogsiveo should be withheld until reaction is resolved to Grade ≤ 1 or baseline, then it should be restarted at a dose of 100 mg twice daily. 4 Adverse reaction Recommended action Electrolyte abnormalities Grade 3 hypophosphataemia persisting for ≥ 7 days despite maximal replacement therapy Ogsiveo should be withheld until reaction is resolved to Grade ≤ 1 or baseline, then it should be restarted at a dose of 100 mg twice daily.
Grade 3 hypokalaemia despite maximal replacement therapy Ogsiveo should be withheld until reaction is resolved to Grade ≤ 1 or baseline, then it should be restarted at a dose of 100 mg twice daily. Hepatic abnormalities Alanine transaminase (ALT) or Aspartate transaminase (AST) ≥ 3 to 5 x ULN Ogsiveo should be withheld until ALT, AST, or both are resolved to < 3 x ULN or baseline, then it should be restarted at a dose of 100 mg twice daily.
ALT or AST > 5 x ULN Ogsiveo should be permanently discontinued. Other adverse reactions Anaphylaxis or other severe hypersensitivity reaction Ogsiveo should be permanently discontinued. Special populations Elderly population No dose adjustment is recommended for patients who are aged 65 years or over.
Clinical data in patients aged 65 years or over is limited. Renal impairment No dose adjustment is recommended in patients with mild or moderate renal impairment. 2). Hepatic impairment No dose adjustment is recommended in patients with mild or moderate hepatic impairment.
2). Paediatric population The safety and efficacy of Ogsiveo in children from 2 to 18 years of age have not been established. Ogsiveo should not be used in children from birth to less than 2 years of age because of potential safety concerns related to structural and functional growth.
1, but no recommendation on a posology can be made. Method of administration Ogsiveo is for oral use. The tablets may be taken with or without food. Tablets should not be broken, chewed or crushed because there are no data currently available to support other methods of administration.
5).