MDM2-p53 antagonist Medicine 04 June 2024 Extended indication Bij volwassen patiënten met advanced metastatic gedifferentieerd liposarcoom (DDLPS). Therapeutic value No estimate possible yet Total cost Registration phase No registration expected Product Active substance MDM2-p53 antagonist Domain Oncology Reason of inclusion New medicine (specialité) Main indication Tumour agnostic medication Extended indication Bij volwassen patiënten met advanced metastatic gedifferentieerd liposarcoom (DDLPS). Current proprietary name Already available biosimilars / generics Proprietary name Brigimadlin Manufacturer Boehringer Ingelheim Portfolio holder Boehringer Ingelheim Mechanism of action Receptor antagonist Route of administration Oral Therapeutical formulation Tablet Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks adjuvante therapie Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity ATMP No Submission date October 2024 Expected Registration October 2025 Orphan drug Yes Registration phase No registration expected Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Doxorubicin Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Fase 1a data laten zien dat oraal MDM2-P53 antagonist Brigimadlin een grotere efficacy laat zien in patiënten met DDLPS ten opzichte van SoC. Verdere resultaten worden verwacht in 2026. Duration of treatment Median 8 month / months Frequency of administration 1 times a day Dosage per administration 45mg References LoRusso et al., The MDM2-p53 Antagonist Brigimadlin (BI 907828) in Patients with Advanced or Metastatic Solid Tumors: Results of a Phase Ia, First-in-Human, Dose-Escalation Study. Cancer Discov. 2023 Aug 4;13(8):1802-1813. doi: 10.1158/2159-8290.CD-23-0153. PMID: 37269344; PMCID: PMC10401071. Additional remarks Expected patient volume per year Patient volume 25 - 40 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Orphanet Expert opinions: (Van het totaal aantal patiënten met DDLPS heeft ongeveer de helft van de patiënten Metastatic/advanced DDLPS). Additional remarks Expected cost per patient per year Cost References Additional remarks Primair eindpunt van fase III studie niet behaald. Potential total cost per year Total cost Total cost for sluice Additional remarks De totale kosten zijn op dit moment nog niet bekend. Off label use Off label use Indications off label use References Additional remarks Indication extension Indication extensions Indication extensions References Additional remarks Other information Additional remarks Positieve eindpunt klinische studie niet gehaald. Geen registratie verwacht.