FDA Approves Vemurafenib for Erdheim-Chester Disease

Share this content:
This is the first-ever drug approval for the blood cancer, which, although rare, carries a poor prognosis.
This is the first-ever drug approval for the blood cancer, which, although rare, carries a poor prognosis.

The US Food and Drug Administration (FDA) approved vemurafenib, a BRAFV600 inhibitor, for Erdheim-Chester Disease (ECD), according to a press release.1 This is the first-ever drug approval for the blood cancer, which, although rare, carries a poor prognosis.

Approval was based on results from the non-randomized phase 2 VE-BASKET study (ClinicalTrials.gov Identifier: NCT01524978), for which patients with BRAFV600-positive cancers were enrolled and assigned to receive vemurafenib.

Of the 22 patients with BRAFV600-positive ECD, the best overall response rate was 54.5%: 11 patients had a partial response and 1 patient had a complete response. At the median follow-up of 26.6 months, the median duration of response was not estimable.

Severe adverse events noted with vemurafenib include second primary malignancies, anaphylaxis, QT prolongation, and liver damage.

About one-half of patients with ECD have BRAFV600 disease.

The FDA granted vemurafenib Priority Review as well as Breakthrough Therapy and Orphan Drug statuses. In 2011, vemurafenib was approved to treat BRAFV600-mutated metastatic melanoma.

Reference

  1. FDA approves first treatment for certain patients with Erdheim-Chester Disease, a rare blood cancer [news release]. US Food and Drug Administration; November 6, 2017. https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm583931.htm?utm_campaign=11062017_PR_FDA%20approves%20treatment%20Erdheim-Chester&utm_medium=email&utm_source=Eloqua. Accessed November 6, 2016.

Related Resources

You must be a registered member of Cancer Therapy Advisor to post a comment.

Regimen and Drug Listings

GET FULL LISTINGS OF TREATMENT Regimens and Drug INFORMATION

Bone Cancer Regimens Drugs
Brain Cancer Regimens Drugs
Breast Cancer Regimens Drugs
Endocrine Cancer Regimens Drugs
Gastrointestinal Cancer Regimens Drugs
Gynecologic Cancer Regimens Drugs
Head and Neck Cancer Regimens Drugs
Hematologic Cancer Regimens Drugs
Lung Cancer Regimens Drugs
Other Cancers Regimens
Prostate Cancer Regimens Drugs
Rare Cancers Regimens
Renal Cell Carcinoma Regimens Drugs
Skin Cancer Regimens Drugs
Urologic Cancers Regimens Drugs

Sign Up for Free e-newsletters