Date: 2015-07-14
Type of information: Granting of the orphan status in the US
Product name: Kevetrin® - thioureidobutyronitrile
Compound: thioureidobutyronitrile
Therapeutic area: Cancer - Oncology - Rare diseases
Action mechanism: Kevetrin™’s primary mechanism of action has been shown to induce activation of p53.
Company: Cellceutix (USA - MA)
Disease: ovarian cancer
Latest news:
- • On July 14, 2015, the FDA has granted orphan drug designation for thioureidobutyronitrile (Kevetrin®) for the treatment of ovarian cancer. Cellceutix has recently reported the near complete disappearance of a metastatic lesion in the spleen of a Stage 4 ovarian cancer patient who was enrolled in the company’s Phase 1 clinical trial of Kevetrin™ being conducted at Harvard Cancer Center’s Dana-Farber Cancer Institute and Beth Israel Deaconess Medical Center. According to information supplied by the hospital, the patient, who successfully completed three Kevetrin 3-dose cycles before discontinuing the trial, experienced increased energy, while scans showed a reduction in the amount of peritoneal fluid (ascites) during treatment with Kevetrin. Subsequent to the second and third Kevetrin cycles, scans showed the spleen lesion to be essentially undetectable and the patient’s disease to be clinically stable. With the completion of the ninth cohort, and commencement of tenth cohort at 450 mg/m2, the hospital has continued research to determine the effect of Kevetrin on p21, the key biomarker tightly controlled by the tumor suppressor protein p53.
- Research conducted by the hospital evaluating p21 expression in earlier cohorts, for which the level of exposure to Kevetrin was substantially lower, showed that about 50 percent of the patients demonstrated at least a 10% increase in p21. The data supported the hypothesis that enhanced p21 is dose related, meaning that as the amount of Kevetrin increased, so did the p21 expression. Hospital scientists are now running samples examining the effect of Kevetrin on p21 at higher dosing levels in more recent cohorts and Cellceutix hopes to disclose the results shortly. Additionally, the principal investigators for the Kevetrin trial have requested that the Company start preparing a presentation to be used for an article on the safety and pharmacological effect of Kevetrin on multiple cancer lines as demonstrated in the clinical trial. Cellceutix interprets this recommendation as a very optimistic sign regarding anticipated outcomes for the trial and clinical advancement of Kevetrin.
Patents:
Submission of marketing authorization application USA :
Submission of marketing authorization application UE:
Withdrawal of marketing authorization application USA:
Withdrawal of marketing authorization application UE:
US authorization:
UE authorization:
Favourable opinion UE:
Favourable opinion USA:
Orphan status USA: 2015-07-14
Orphan status UE:
Pediatric exclusivit _USA:
Pediatric exclusivity UE:
OTC status:
Other news:
Is general: Yes
