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Pharmacist's Applications to Practice
HOPA, through the Publications Committee, will review new drug updates and provide analysis and research on the application of these new drugs or indications.

The letters “PAP” after drugs listed below indicates that they include this additional analysis.  


November 6, 2017

https://www.accessdata.fda.gov/drugsatfda_docs/label/2017/202429s016lbl.pdf

On November 6, 2017, the Food and Drug Administration granted regular approval to vemurafenib (ZELBORAF®, Hoffmann-La Roche Inc.) for the treatment of patients with Erdheim-Chester Disease (ECD) with BRAF V600 mutation.

Approval was based on an open-label, multicenter, single-arm, multiple cohort clinical trial in patients aged 16 years of age and older. The trial included 22 patients with BRAF V600 mutation- positive ECD. The best overall response rate, as assessed by the investigator using RECIST 1.1, was 54.5% (n=12) (95% CI: 32.2, 75.6). Eleven partial responses (50%) and 1 complete response (4.5%) were observed. The median duration of follow- up was 26.6 months (range: 3.0 to 44.3 months). The median time to response was 11 months (95% CI: 3.7, 14.6). The median response duration was not estimable.

In the trial, the most commonly reported adverse reactions (>50%) in patients with BRAF V600 ECD were arthralgia, rash maculo papular, alopecia, fatigue, electrocardiogram QT prolonged, and skin papilloma. The most common (≥ 10%) Grade ³ 3 adverse reactions were squamous cell carcinoma of the skin, hypertension, rash maculo-papular, and arthralgia. The incidence of adverse reactions resulting in permanent discontinuation was 32%.

The recommended dose of vemurafenib is 960 mg orally twice daily taken approximately 12 hours apart with or without a meal.

Full prescribing information is available at
https://www.accessdata.fda.gov/drugsatfda_docs/label/2017/202429s016lbl.pdf

FDA granted this application priority review. Breakthrough Therapy and Orphan Drug designations for this indication were also granted. A description of FDA expedited programs is in the Guidance for Industry: Expedited Programs for Serious Conditions-Drugs and Biologics, available at:
http://www.fda.gov/downloads/drugs/guidancecomplianceregulatoryinformation/guidances/ucm358301.pdf

Healthcare professionals should report all serious adverse events suspected to be associated with the use of any medicine and device to FDA’s MedWatch Reporting System by completing a form online at http://www.fda.gov/medwatch/report.htm, by faxing (1-800-FDA-0178) or mailing the postage-paid address form provided online, or by telephone (1-800-FDA-1088).

 

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