Kimozo (temozolomide oral suspension) / Orphelia Pharma - LARVOL DELTA

Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 24-27 February 2025 (European Medicines Agency) - "After re-examining its initial opinion, the European Medicines Agency has confirmed its recommendation to refuse marketing authorisation for the medicine Kizfizo. The medicine was intended for the treatment of children with neuroblastoma, a cancer of nerve cells. The Agency issued its opinion after re-examination on 27 February 2025."

European regulatory • Neuroblastoma

Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 11-14 November 2024 (European Medicines Agency) - "The European Medicines Agency has recommended the refusal of the marketing authorisation for Kizfizo, a medicine intended for the treatment of children with neuroblastoma, a cancer of nerve cells. The Agency issued its opinion on 14 November 2024. The company that applied for authorisation, Orphelia Pharma, may ask for re-examination of the opinion within 15 days of receiving the opinion."

European regulatory • Neuroblastoma

Dispensing Oral Temozolomide in Children: Precision and Stability of a Novel and Ready to Use Liquid Formulation in Comparison with Capsule Derived Mixtures. (PubMed, Pharmaceutics) - "Only 1/72 of preparations from capsules met the predefined acceptance criteria, whereas Ped-TMZ showed no TMZ loss, and the AIC remained within specifications. In conclusion, mixing TMZ capsule content with food may result in significant underexposure, possibly even greater in routine practice, as complete food intake by the child is unlikely."

Journal • Oncology • Oral Cancer • Pediatrics

A Multicenter Randomized Bioequivalence Study of a Novel Ready-to-Use Temozolomide Oral Suspension vs. Temozolomide Capsules. (PubMed, Pharmaceutics) - P1 | "This study showed that Ped-TMZ oral suspension and TMZ oral capsule treatment are immediate release and bioequivalent medicines. There were also no unexpected safety signals or local toxicity (funded by ORPHELIA Pharma; ClinicalTrials.gov number, NCT04467346)."

Journal • Brain Cancer • CNS Tumor • Glioblastoma • Glioma • Neuroblastoma • Oncology • Pediatrics • Solid Tumor

Orphelia Pharma files for EU marketing authorization of Kizfizo (The Pharma Letter) - "Privately-held French company Orphelia Pharma...has filed a centralized Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for Kizfizo, the first oral liquid formulation of temozolomide, the active ingredient of Merck & Co’s...Temodar."

European regulatory • Oncology

"ORPHELIA Pharma Selects Tanner Pharma Group to Initiate Named Patient Program for KIMOZO® (temozolomide) in European Countries https://t.co/MHuiEUXxPp" (@NewsFromBW)

Clinical

ORPHELIA Pharma Selects Tanner Pharma Group to Initiate Named Patient Program for KIMOZO (temozolomide) in European Countries (Businesswire) - "ORPHELIA Pharma...and Tanner Pharma Group...have signed an exclusive agreement to manage the supply and distribution of Ped-TMZ, also known as KIMOZO® (temozolomide 40 mg/ml, oral suspension), to meet the special demands which may come from physicians for this unapproved pediatric form of temozolomide....Under the terms of the agreement, Tanner Pharma will provide access to KIMOZO® via a named patient program in European countries, in particular countries where KIMOZO® has already been used as an investigational medication in the context of ORPHELIA Pharma's now fully enrolled TEMOkids clinical trial."

Licensing / partnership • Brain Cancer • CNS Tumor • Glioblastoma • Glioma • Neuroblastoma • Oncology • Solid Tumor

A bioequivalence study of a novel liquid and ready-to-use temozolomide oral suspension and temozolomide capsules in patients with primary tumors central nervous system malignancies. (ASCO 2022) - P1 | "The oral suspension of temozolomide (Kimozo) and capsule of temozolomide (Temodal) are bioequivalent under fasting conditions in patients with CNS primary malignancies, supporting that they are therapeutic equivalent."

Clinical • Brain Cancer • CNS Tumor • Glioblastoma • Glioma • Mucositis • Neuroblastoma • Oncology • Pediatrics

TEMOkids Study : A Phase I Pediatric Study for KIMOZO, Oral Suspension of Temozolomide (clinicaltrials.gov) - P1 | N=49 | Active, not recruiting | Sponsor: Orphelia Pharma | Recruiting ➔ Active, not recruiting

Enrollment closed • Oncology • Pediatrics

TEMOkids Study : A Phase I Pediatric Study for KIMOZO, Oral Suspension of Temozolomide (clinicaltrials.gov) - P1 | N=40 | Recruiting | Sponsor: Orphelia Pharma | Trial completion date: Aug 2022 ➔ Jun 2023 | Trial primary completion date: Aug 2022 ➔ Dec 2022

Trial completion date • Trial primary completion date • Oncology • Pediatrics

Capecitabine Plus Temozolomide vs Temozolomide Alone in Advanced Pancreatic Neuroendocrine Tumors (THE ASCO POST) - "In the updated final analysis of the phase II ECOG-ACRIN E2211 trial, patients with advanced pancreatic neuroendocrine tumors experienced a significant improvement in progression-free survival with capecitabine plus temozolomide over temozolomide alone.1 Although the 5-month difference in overall survival was not statistically significant, it was 'clinically meaningful,' according to Pamela L. Kunz, MD..."

Media quote

Melanoma and Skin Cancer Highlights From ASCO 2022 (Medscape) - "Key studies on melanoma and skin cancers presented at ASCO 2022 included refinements to neoaduvant therapies for melanoma and two promising agents in advanced disease, one in melanoma and one in Merkel cell carcinoma....In advanced disease, Dr Weber reviews data presented on two new therapies. He discusses a phase 2 trial of camrelizumab, an IgG4 antibody against PD-1, combined with apatinib and temozolomide for patients with advanced acral melanoma. He closes with a report examining navtemadlin, an oral small-molecule inhibitor of MDM2 in metastatic Merkel cell carcinoma."

Video

Development of a Hospital Compounded, Taste-Masked, Temozolomide Oral Suspension and 5-Year Real-Life Experience in Treating Paediatric Patients. (PubMed, Pharmaceuticals (Basel)) - "Thirty-eight patients participated in the palatability study and choose cola flavour, and 104 patients were treated in Gustave Roussy with the developed suspension; no unexpected event was reported. To conclude, we propose here a new TMZ liquid formulation which is stable for at least 6 weeks and well-tolerated with extensive feedback."

Journal • Brain Cancer • Glioblastoma • Medulloblastoma • Neuroblastoma • Oncology • Pediatrics • Solid Tumor

TEMOkids Study : A Phase I Pediatric Study for KIMOZO, Oral Suspension of Temozolomide (clinicaltrials.gov) - P1 | N=40 | Recruiting | Sponsor: Orphelia Pharma | Trial primary completion date: Feb 2022 ➔ Aug 2022

Trial primary completion date • Oncology • Pediatrics

Bioequivalence Study Between Temozolomide Oral Suspension (Ped-TMZ) and Temodal® Capsules (clinicaltrials.gov) - P1 | N=36 | Completed | Sponsor: Orphelia Pharma | Recruiting ➔ Completed

Trial completion

Bioequivalence Study Between Temozolomide Oral Suspension (Ped-TMZ) and Temodal® Capsules (clinicaltrials.gov) - P1; N=30; Recruiting; Sponsor: Orphelia Pharma; Trial completion date: Mar 2021 ➔ Oct 2021; Trial primary completion date: Mar 2021 ➔ Oct 2021

Trial completion date • Trial primary completion date