Breaking News
November 19, 2017 - Don’t Fret Delays in Treating Colon Cancer, Study Suggests
November 19, 2017 - Yeast Infection Test: MedlinePlus Lab Test Information
November 19, 2017 - Video of blood clot contraction reveals how platelets naturally form unobtrusive clots
November 19, 2017 - UNN researchers explore modeling of cognitive dissonance phenomenon
November 19, 2017 - Verseon presents second anticoagulation candidate for clinical trials at AHA 2017
November 19, 2017 - Pregnant Asian women who develop high blood pressure at highest risk for heart failure hospitalization
November 19, 2017 - Medicare seeks comment on ways to cut costs of Part D drugs
November 19, 2017 - Tree cover linked to fewer asthma cases in polluted urban neighborhoods
November 19, 2017 - Make the Diagnosis: Hair Care Conundrum
November 19, 2017 - New techniques give blood biopsies greater promise
November 19, 2017 - Researchers identify possible genetic basis for coronary artery disease
November 19, 2017 - Stress experienced by emergency call handlers has negative impact on psychological health
November 18, 2017 - New cancer cell screening is improving childhood leukaemia treatment
November 18, 2017 - Groundbreaking study identifies protein as potential factor in cancer metastasis
November 18, 2017 - New model to test effectiveness of existing and potential CF therapies
November 18, 2017 - Staying Active May Lower Odds for Glaucoma
November 18, 2017 - Potential new autism drug shows promise in mice
November 18, 2017 - Some states roll back ‘retroactive Medicaid,’ a buffer for the poor — and for hospitals
November 18, 2017 - Selectively deleting stem cell factor promotes recovery after TBI in mice
November 18, 2017 - Breakthrough research brings new procedure closer to helping patients with blood cancer
November 18, 2017 - Dr Peter Simpson Appointed to SLAS Board of Directors
November 18, 2017 - Friendships between young children can protect against ADHD
November 18, 2017 - Old World monkeys could hold key to stop progression of rheumatoid arthritis
November 18, 2017 - Harris Health System RNs named among 20 Outstanding Nurses for 2017
November 18, 2017 - Old World monkeys could be key to a new, powerful rheumatoid arthritis therapy
November 18, 2017 - Mount Sinai researchers identify new therapeutic target for ER+ breast cancer
November 18, 2017 - Age, CRP levels predict success in tapering of biologics in rheumatoid arthritis patients
November 18, 2017 - New dye could be used to observe electrical activity of neurons in the brain
November 18, 2017 - New study further validates use of vaginal progesterone to decrease risk of preterm birth
November 18, 2017 - Russian researcher determined range of reference values for boron in the human body
November 18, 2017 - ‘What the Health?’ Tax bill or health bill?
November 18, 2017 - Could Your Cat Give You ‘Bird Flu?’
November 18, 2017 - Vitamin D Linked to Fertility Outcomes in ART
November 18, 2017 - Neuroscientists identify genetic changes in microglia in a mouse model of neurodegeneration and Alzheimer’s disease
November 18, 2017 - Tax reform proposal could impact care for older Americans
November 18, 2017 - PCSK9 inhibitor offers clinical benefit to patients with peripheral artery disease
November 18, 2017 - Researchers receive £1.3 million to develop sight-saving imaging technology
November 18, 2017 - Novel buckypaper sensor could pave way for high-performance, affordable wearable technology
November 18, 2017 - Despite ACA cost protections, most adolescents skip regular checkups
November 18, 2017 - Stem cell treatment allows paraplegic rats to walk and regain sensory perception
November 18, 2017 - HTC analytical conference comes to the UK
November 18, 2017 - Face It: Drinking, Smoking Takes Toll on Looks: MedlinePlus Health News
November 18, 2017 - New research shows where in the brain the earliest signs of Alzheimer’s occur
November 18, 2017 - Philips announces launch of global movement to raise awareness for COPD
November 18, 2017 - University of Bristol awarded grant to reduce antibacterial drug resistance in Thailand
November 18, 2017 - New oxytocin chemical sensor could be first step towards early diagnosis of autism
November 18, 2017 - Study shows how naïve T-cells may affect tumor immunity and immunotherapy
November 18, 2017 - New studies highlight importance of cardiorespiratory fitness to reduce CVD risk
November 18, 2017 - Study finds medical cannabis is effective at reducing opioid addiction
November 18, 2017 - Specially tailored, ultrafast light pulses can trigger neurons to fire in different patterns
November 18, 2017 - Decrease in sunshine linked to rising incidence of Rickets
November 18, 2017 - Harnessing social media big data to fight against prescription drug crisis
November 18, 2017 - Researchers find way to switch tumor cells between 2D and 3D morphology
November 18, 2017 - Leaf-eating ability of beetle largely due to bacteria inside the insect
November 18, 2017 - FDA Approves Hemlibra (emicizumab-kxwh) for Hemophilia A with Inhibitors
November 18, 2017 - Adolescents underreport amphetamine use, likely unaware that adderall is amphetamine
November 18, 2017 - Study reveals a reduced risk of teenage eczema in breastfed babies
November 18, 2017 - Separating side effects could pave way for safe, effective pain medications
November 18, 2017 - Gut bacteria at young age can contribute to MS disease onset and progression, study suggests
November 18, 2017 - Environmental triggers may play role in development of Lupus
November 18, 2017 - Review looks into conventional versus new treatment modalities in orthodontic pain management
November 17, 2017 - FDA Alert: Diphenoxylate Hydrochloride and Atropine Sulfate Tablets by Greenstone: Recall
November 17, 2017 - For older women, every movement matters
November 17, 2017 - Talking-based therapy could transform aftercare for cancer survivors
November 17, 2017 - Olympus IXplore SpinSR10 imaging system enables researchers to observe fine details in live cells
November 17, 2017 - Study explores reasons for underrepresentation of minorities in genetic cancer research
November 17, 2017 - California firm running physician practices is closing down as scrutiny ramps up
November 17, 2017 - BMI not valid measure of obesity in postmenopausal women, study shows
November 17, 2017 - Vaginal progesterone decreases the risk of premature birth in women with short cervix
November 17, 2017 - Pricey ER Tests for Chest Pain Often Unnecessary
November 17, 2017 - ‘Old’ Lungs May Be Good Transplant Options: MedlinePlus Health News
November 17, 2017 - How not to gain weight over the holidays
November 17, 2017 - Researchers map first-ever proteome of healthy human heart
November 17, 2017 - Drug used to prevent and treat malaria may also be effective for Zika virus
November 17, 2017 - One in 20 children still receiving codeine to treat pain despite warning from federal regulators
November 17, 2017 - Improving clinical trials with machine learning
November 17, 2017 - Experts identify mental exercise program that can reduce risk of dementia
November 17, 2017 - Just-in-time 3-D implants set to transform tumor surgery
November 17, 2017 - Skin patch offers hope for people with peanut allergy
November 17, 2017 - Scientists identify biomarkers that predict risk of death in Ebola patients
FDA Approves Zelboraf (vemurafenib) for Erdheim-Chester Disease with BRAF V600 Mutation

FDA Approves Zelboraf (vemurafenib) for Erdheim-Chester Disease with BRAF V600 Mutation

image_pdfDownload PDFimage_print

November 6, 2017 – The U.S. Food and Drug Administration today expanded the approval of Zelboraf (vemurafenib) to include the treatment of certain adult patients with Erdheim-Chester Disease (ECD), a rare cancer of the blood. Zelboraf is indicated to treat patients whose cancer cells have a specific genetic mutation known as BRAF V600. This is the first FDA-approved treatment for ECD.

“Today’s approval of Zelboraf for patients with ECD demonstrates how we can apply knowledge of the underlying genetic characteristics of certain malignancies to other cancers,” said Richard Pazdur, M.D., director of the FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research. “This product was first approved in 2011 to treat certain patients with melanoma that harbor the BRAF V600E mutation, and we are now bringing the therapy to patients with a rare cancer with no approved therapies.”

ECD is a slow-growing blood cancer that originates in the bone marrow. ECD causes an increased production of histiocytes, a type of white blood cell. Excess histiocytes can result in tumors infiltrating many organs and tissues throughout the body, including the heart, lungs, brain and others. ECD is estimated to affect 600 to 700 patients worldwide. Approximately 54 percent of patients with ECD have the BRAF V600 mutation. Patients with ECD have very limited life expectancies.

Zelboraf is a kinase inhibitor that works by blocking certain enzymes that promote cell growth.

The efficacy of Zelboraf for the treatment of ECD was studied in 22 patients with BRAF-V600-mutation positive ECD. The trial measured the percent of patients who experienced a complete or partial reduction in tumor size (overall response rate). In the trial, 11 patients (50 percent) experienced a partial response and 1 patient (4.5 percent) experienced a complete response.

Common side effects of Zelboraf in patients with ECD include joint pain (arthralgia); small, raised bumps (maculo-papular rash); hair loss (alopecia); fatigue; change in the heart’s electrical activity (prolonged QT interval) and skin growths (papilloma).

Severe side effects of Zelboraf include the development of new cancers (skin cancer, squamous cell carcinoma or other cancers), growth of tumors in patients with BRAF wild-type melanoma, hypersensitivity reactions (anaphylaxis and DRESS syndrome), severe skin reactions (Stevens-Johnson Syndrome and toxic epidermal necrolysis), heart abnormalities (QT prolongation), liver damage (hepatotoxicity), photosensitivity, severe reactions in the eye (uveitis), immune reactions after receiving radiation treatment (radiation sensitization and radiation recall), kidney failure and thickening of tissue in the hands and feet (Dupuytren’s contracture and plantar fascial fibromatosis). Zelboraf can cause harm to a developing fetus; women should be advised of the potential risk to the fetus and to use effective contraception.

The FDA granted this application Priority Review and Breakthrough Therapy designations for this indication. Zelboraf also received Orphan Drug designation for this indication, which provides incentives to assist and encourage the development of drugs for rare diseases.

The FDA granted the approval of Zelboraf to Hoffman-LaRoche, Inc.

Source: FDA

Posted: November 2017

Related Articles:

Zelboraf (vemurafenib) FDA Approval History

Tagged with:

About author

Related Articles