Date: 2017-11-06
Type of information: Granting of a Market Authorisation in the US
Product name: Zelboraf®
Compound: vemurafenib
Therapeutic area: Cancer - Oncology - Rare diseases
Action mechanism:
- kinase inhibitor. Vemurafenib is a BRAF-inhibitor and it is designed to selectively target and inhibit a mutated form of the BRAF protein.
Company: Roche (Switzerland)
Disease:
Latest news:
- • On November 6, 2017, the FDA has expanded the approval of Zelboraf® (vemurafenib) to include the treatment of certain adult patients with Erdheim-Chester Disease , 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 Erdheim-Chester Disease.
- 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.
- The approval is based on data from the Phase II VE-BASKET study. Basket studies use an innovative clinical trial design that helps collect data faster and may accelerate the development of medicines for diseases with high unmet need. Instead of enrolling people based primarily on their disease or its location, basket studies match a disease’s underlying genetic profile to the mechanism of action of the medicine. Final results of VE-BASKET for the 22 people with ECD showed a best overall response rate of 54.5 percent. The most common Grade 3 or higher adverse events were new skin cancers, high blood pressure, rash and joint pain. The most common adverse events were joint pain, rash, hair loss, fatigue, change in heart rhythm and skin tags.
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: 2017-11-06
UE authorization:
Favourable opinion UE:
Favourable opinion USA:
Orphan status USA: 2016-08-02
Orphan status UE:
Pediatric exclusivit _USA:
Pediatric exclusivity UE:
OTC status:
Other news:
Is general: Yes
