— 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, CA, USA I November 6, 2017 I 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.
About Zelboraf
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 Indication
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.
About Genentech
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.
SOURCE: Genentech
Post Views: 24
— 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, CA, USA I November 6, 2017 I 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.
About Zelboraf
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 Indication
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.
About Genentech
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.
SOURCE: Genentech
Post Views: 24
