– Zelboraf is the first FDA-approved treatment for Erdheim-Chester disease (ECD), a rare blood disease –
– Approval based on data from a basket study, which enrolls participants across multiple diseases based predominantly on genetic profile –
SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)-- Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced that the U.S. Food and Drug Administration (FDA) has approved Zelboraf® (vemurafenib) for Erdheim-Chester disease (ECD) with BRAF V600 mutation. ECD is a rare, serious blood disease characterized by the abnormal multiplication of certain white blood cells called histiocytes, which can invade normal tissues and organs in the body.
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.
“This FDA decision means people living with Erdheim-Chester disease will now, for the first time, have an FDA-approved treatment option,” said Sandra Horning, M.D., chief medical officer and head of Global Product Development. “We are committed to finding new ways to bring medicines to patients with high unmet need, and we are pleased that this innovative clinical trial helped identify Zelboraf for treatment of this rare disease.”
“The Erdheim-Chester disease community is very encouraged by this first FDA-approved treatment for ECD, bringing new hope to patients and their families," said Kathleen Brewer, president of ECD Global Alliance. "This new treatment option shows that meaningful breakthroughs can occur rapidly when patients, families, research physicians, industry and the FDA work together to help patients.”
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.
Zelboraf monotherapy was approved for the treatment of people with unresectable or metastatic melanoma with BRAF V600E mutation in 2011. The FDA previously granted Priority Review and Breakthrough Therapy Designation to Zelboraf for ECD with BRAF V600 mutation.
For those who qualify, Genentech offers patient assistance programs for people prescribed Zelboraf by their doctor through Genentech Access Solutions. Doctors can contact Genentech Access Solutions at (888) 249-4918. More information is also available at http://www.Genentech-Access.com.
About the VE-BASKET Study
VE-BASKET is an open-label, Phase II, non-randomized, basket study investigating the use of Zelboraf for people with BRAF V600 mutation-positive cancers and other diseases, including ECD. Final results for the 22 people with ECD showed a best overall response rate of 54.5 percent by RECIST v1.1. The median duration of response was not estimable at a median follow-up time of 26.6 months. The most common Grade 3 or higher adverse events (≥10 percent) were new skin cancers, high blood pressure, rash and joint pain. The most common adverse events (>50 percent) were joint pain, rash, hair loss, fatigue, change in heart rhythm and skin tags. Initial study results were published in the New England Journal of Medicine in August 2015.
About Erdheim-Chester Disease
Erdheim-Chester disease (ECD) is an extremely rare non-Langerhans cell histiocytosis. The exact prevalence and incidence of ECD are difficult to ascertain given the disease is so rare. Based on available published data, it’s estimated there are fewer than 500 cases of ECD in the United States. More than 50 percent of people with ECD have BRAF V600 mutation-positive disease.
About Genentech Access Solutions
Access Solutions is part of Genentech’s commitment to helping people access the Genentech medicines they are prescribed, regardless of their ability to pay. The team of in-house specialists at Access Solutions is dedicated to helping people navigate the access and reimbursement process, and to providing assistance to eligible patients in the United States who are uninsured or cannot afford the out-of-pocket costs for their medicine. To date, the team has helped more than 1.4 million patients access the medicines they need. Please contact Access Solutions (866) 4ACCESS/(866) 422-2377 or visit http://www.Genentech-Access.com for more information.
Zelboraf is designed to inhibit some mutated forms of BRAF, which cause abnormal signaling inside cancer cells leading to tumor growth. BRAF is a protein in a cell signaling pathway that helps control cell growth and survival. Zelboraf was the first approved product in its class. Zelboraf was co-developed under a 2006 license and collaboration agreement between Roche and Plexxikon, now a member of the Daiichi Sankyo Group.
Zelboraf is a prescription medicine used to treat a type of skin cancer called melanoma that has spread to other parts of the body or cannot be removed by surgery, and that has a certain type of abnormal “BRAF” gene.
Zelboraf is not used to treat melanoma with a normal BRAF gene. A patient’s healthcare provider will perform a test to make sure that Zelboraf is right for them.
Zelboraf is a prescription medicine used to treat a type of blood cell cancer called Erdheim-Chester disease (ECD), that can affect body tissues and organs, and that has a certain type of abnormal “BRAF” gene.
It is not known if Zelboraf is safe and effective in children under 18 years of age.
Important Safety Information
What is the most important information patients should know about Zelboraf?
Zelboraf can cause serious side effects, including:
- Risk of new cancers. Zelboraf may cause certain types of skin cancer called cutaneous squamous cell carcinoma (cuSCC) and keratoacanthoma. New melanoma lesions have occurred in people who take Zelboraf. Zelboraf may also cause another type of cancer called non-cutaneous squamous cell carcinoma (non-cuSCC). Patients should talk with their healthcare provider about their risk for these cancers.
Patients should check their skin and tell their healthcare provider right away about any skin changes, including:
- A new wart
- A skin sore or reddish bump that bleeds or does not heal
- A change in size or color of a mole
A patient’s healthcare provider should check their skin before they start taking Zelboraf, and every 2 months during treatment with Zelboraf, to look for any new skin cancers. A patient’s healthcare provider may continue to check their skin for 6 months after they stop taking Zelboraf.
A patient’s healthcare provider should also check for cancers that may not occur on the skin. Patients should tell their healthcare provider about any new symptoms that they get while taking Zelboraf.
Bone marrow blood cancer has happened in some people with Erdheim-Chester disease (ECD) including those who take Zelboraf. A patient’s healthcare provider will monitor them for blood cancer through routine blood tests.
Before patients take Zelboraf, they should tell their healthcare provider about all of their medical conditions, including if they:
- Have any heart problems, including a condition called long QT syndrome
- Have liver or kidney problems
- Have had or are planning to receive radiation therapy
- Have been told that they have low blood levels of potassium, calcium, or magnesium
- Are pregnant or plan to become pregnant. Zelboraf can harm an unborn baby.
- Females who are able to become pregnant should use effective birth control during treatment with Zelboraf and for 2 weeks after the final dose of Zelboraf
- Patients should talk to their healthcare provider about birth control methods that may be right for them
- Patients should tell their healthcare provider right away if they become pregnant during treatment with Zelboraf
- Are breastfeeding or plan to breastfeed. It is not known if Zelboraf passes into breast milk. Patients should not breastfeed during treatment with Zelboraf and for 2 weeks after the final dose of Zelboraf. Patients should talk to their healthcare provider about the best way to feed their baby during this time.
Patients should tell their healthcare provider about all of the medicines they take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.
What should patients avoid while taking Zelboraf?
Patients should avoid sunlight during treatment with Zelboraf. Zelboraf can make a patient’s skin sensitive to sunlight. Patients may burn more easily and get severe sunburns. To help protect against sunburn:
- When patients go outside, they should wear clothes that protect their skin, including their head, face, hands, arms, and legs.
- Patients should use lip balm and a broad-spectrum sunscreen with SPF 30 or higher.
What are the possible side effects of Zelboraf?
- Allergic reactions can happen while taking Zelboraf, and can be severe. Patients should stop taking Zelboraf and get medical help right away if they get any of these symptoms of an allergic reaction:
- Rash or redness all over their body
- Trouble breathing or swallowing
- Swelling of the face, lips, or tongue
- Throat tightness or hoarseness
- Feel faint
- Fast heartbeat
- Severe skin reactions. Patients should stop taking Zelboraf and call their healthcare provider right away if they get a skin rash with any of the following symptoms, because they may have a severe skin reaction:
- Blisters on their skin
- Blisters or sores in their mouth
- Peeling of their skin
- Redness or swelling of their face, hands, or soles of their feet
- Changes in the electrical activity of the heart called QT prolongation. QT prolongation can cause irregular heartbeats that can be life-threatening. A patient’s healthcare provider should do tests before they start taking Zelboraf and during treatment with Zelboraf to check the electrical activity of their heart and their body salts (electrolytes). Patients should tell their healthcare provider right away if they feel faint, lightheaded, dizzy, or feel their heart beating irregularly or fast while taking Zelboraf. These may be symptoms related to QT prolongation.
- Liver injury. A patient’s healthcare provider should do blood tests to check their liver function before they start taking Zelboraf and during treatment. Patients should tell their healthcare provider right away if they get any of these symptoms of a liver problem during treatment:
- Yellowing of their skin or the white part of their eyes
- Dark or brown (tea color) urine
- Nausea or vomiting
- Loss of appetite
- Pain on the right side of their stomach
- Eye problems. Patients should tell their healthcare provider right away if they get any of these symptoms during treatment with Zelboraf:
- Eye pain, swelling, or redness
- Blurred vision or other vision changes
- Worsening side effects from radiation treatment that can sometimes be severe or lead to death. Patients should tell their healthcare provider if they have had or are planning to receive radiation therapy.
- Kidney injury. A patient’s healthcare provider should do blood tests to check their kidney function before they start taking Zelboraf and during treatment.
- Connective tissue disorders. Patients should tell their healthcare provider if they develop an unusual thickening of the palms of their hands along with tightening of the fingers inward or any unusual thickening of the soles of their feet which may be painful.
The most common side effects of Zelboraf in melanoma include:
- Joint pain
- Hair loss
- Sunburn or sun sensitivity
The most common side effects of Zelboraf in Erdheim-Chester disease include:
- Joint pain
- Hair loss
- QT prolongation
These are not all of the possible side effects of Zelboraf. Patients should call their doctor for medical advice about side effects.Patients may report side effects to the FDA at (800) FDA-1088 or http://www.fda.gov/medwatch. Patients may also report side effects to Genentech at (888) 835-2555.
Please see Full Prescribing Information and Medication Guide for additional Important Safety Information at http://www.zelboraf.com.
Founded more than 40 years ago, Genentech is a leading biotechnology company that discovers, develops, manufactures and commercializes medicines to treat patients with serious or life-threatening medical conditions. The company, a member of the Roche Group, has headquarters in South San Francisco, California. For additional information about the company, please visit http://www.gene.com.
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