Blueprint Medicines Submits New Drug Application to U.S. Food and Drug Administration for Avapritinib for the Treatment of PDGFRA Exon 18 Mutant GIST and Fourth-Line GIST
CAMBRIDGE, Mass., June 14, 2019 /PRNewswire/ -- Blueprint Medicines Corporation (NASDAQ: BPMC), a precision therapy company focused on genomically defined cancers, rare diseases and cancer immunotherapy, today announced it has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for avapritinib for the treatment of adult patients with PDGFRA Exon 18 mutant gastrointestinal stromal tumors (GIST), regardless of prior therapy, and fourth-line GIST. Currently, no effective therapy exists for either population. Avapritinib is an investigational, potent and highly selective KIT and PDGFRA inhibitor for patients with advanced GIST.
Avapritinib has received Breakthrough Therapy Designation from the FDA for the treatment of patients with unresectable or metastatic GIST harboring the PDGFRα D842V mutation, as well as Fast Track Designations for the treatment of patients with GIST harboring a PDGFRα D842V mutation regardless of prior therapy, and patients with GIST who have progressed following treatment with imatinib and a second tyrosine kinase inhibitor.
Blueprint Medicines has requested priority review for the application, which, if granted, could result in a six-month review process. The FDA has a 60-day filing review period to determine whether the NDA is complete and acceptable for filing.
"There is an important need for new treatment options that offer durable responses for PDGFRA Exon 18 mutant and fourth-line GIST patients," said Andy Boral, M.D., Ph.D., Chief Medical Officer at Blueprint Medicines. "By targeting the underlying molecular drivers of GIST, avapritinib has the potential to help these patients realize the benefits of precision therapy. We plan to work closely with the FDA to bring avapritinib to appropriate GIST patients as quickly as possible."
About GIST
GIST is a sarcoma, or tumor of bone or connective tissue, of the gastrointestinal (GI) tract. Tumors arise from cells in the wall of the GI tract and occur most often in the stomach or small intestine. Most patients are diagnosed between the ages of 50 to 80, and diagnosis is typically triggered by GI bleeding, incidental findings during surgery or imaging and, in rare cases, tumor rupture or GI obstruction.
Most GIST cases are caused by a spectrum of clinically relevant mutations that force the KIT or PDGFRA protein kinases into an increasingly active state. Because currently available therapies primarily bind to the inactive protein conformations, certain primary and secondary mutations typically lead to treatment resistance and disease progression.
In unresectable or metastatic GIST, clinical benefits from existing treatments can vary by mutation type. Mutational testing is critical to tailor therapy to the underlying disease driver and is recommended in expert guidelines. Currently, there are no approved therapies for patients with KIT-driven GIST whose disease progresses beyond imatinib, sunitinib and regorafenib. In patients with metastatic PDGFRα D842V-driven GIST, progression occurs in a median of approximately three to four months with available therapy.
About Avapritinib
Avapritinib is an investigational, oral precision therapy that selectively and potently inhibits KIT and PDGFRA mutant kinases. It is a type 1 inhibitor designed to target the active kinase conformation; all oncogenic kinases signal via this conformation. Avapritinib has demonstrated broad inhibition of KIT and PDGFRA mutations associated with GIST, including potent activity against activation loop mutations that are associated with resistance to currently approved therapies.
Blueprint Medicines is initially developing avapritinib for the treatment of advanced GIST, advanced systemic mastocytosis (SM), and indolent and smoldering SM. The FDA has granted Breakthrough Therapy Designation to avapritinib for two indications: one for the treatment of unresectable or metastatic GIST harboring the PDGFRα D842V mutation and one for the treatment of advanced SM, including the subtypes of aggressive SM, SM with an associated hematologic neoplasm and mast cell leukemia.
Blueprint Medicines has an exclusive collaboration and license agreement with CStone Pharmaceuticals for the development and commercialization of avapritinib and certain other drug candidates in Mainland China, Hong Kong, Macau and Taiwan. Blueprint Medicines retains development and commercial rights for avapritinib in the rest of the world.
About Blueprint Medicines
Blueprint Medicines is a precision therapy company striving to improve human health. With a focus on genomically defined cancers, rare diseases and cancer immunotherapy, we are developing transformational medicines rooted in our leading expertise in protein kinases, which are proven drivers of disease. Our uniquely targeted, scalable approach empowers the rapid design and development of new treatments and increases the likelihood of clinical success. We are currently advancing four investigational medicines in clinical development, along with multiple research programs. For
CAMBRIDGE, Mass., June 14, 2019 /PRNewswire/ -- Blueprint Medicines Corporation (NASDAQ: BPMC), a precision therapy company focused on genomically defined cancers, rare diseases and cancer immunotherapy, today announced it has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for avapritinib for the treatment of adult patients with PDGFRA Exon 18 mutant gastrointestinal stromal tumors (GIST), regardless of prior therapy, and fourth-line GIST. Currently, no effective therapy exists for either population. Avapritinib is an investigational, potent and highly selective KIT and PDGFRA inhibitor for patients with advanced GIST.
Avapritinib has received Breakthrough Therapy Designation from the FDA for the treatment of patients with unresectable or metastatic GIST harboring the PDGFRα D842V mutation, as well as Fast Track Designations for the treatment of patients with GIST harboring a PDGFRα D842V mutation regardless of prior therapy, and patients with GIST who have progressed following treatment with imatinib and a second tyrosine kinase inhibitor.
Blueprint Medicines has requested priority review for the application, which, if granted, could result in a six-month review process. The FDA has a 60-day filing review period to determine whether the NDA is complete and acceptable for filing.
"There is an important need for new treatment options that offer durable responses for PDGFRA Exon 18 mutant and fourth-line GIST patients," said Andy Boral, M.D., Ph.D., Chief Medical Officer at Blueprint Medicines. "By targeting the underlying molecular drivers of GIST, avapritinib has the potential to help these patients realize the benefits of precision therapy. We plan to work closely with the FDA to bring avapritinib to appropriate GIST patients as quickly as possible."
About GIST
GIST is a sarcoma, or tumor of bone or connective tissue, of the gastrointestinal (GI) tract. Tumors arise from cells in the wall of the GI tract and occur most often in the stomach or small intestine. Most patients are diagnosed between the ages of 50 to 80, and diagnosis is typically triggered by GI bleeding, incidental findings during surgery or imaging and, in rare cases, tumor rupture or GI obstruction.
Most GIST cases are caused by a spectrum of clinically relevant mutations that force the KIT or PDGFRA protein kinases into an increasingly active state. Because currently available therapies primarily bind to the inactive protein conformations, certain primary and secondary mutations typically lead to treatment resistance and disease progression.
In unresectable or metastatic GIST, clinical benefits from existing treatments can vary by mutation type. Mutational testing is critical to tailor therapy to the underlying disease driver and is recommended in expert guidelines. Currently, there are no approved therapies for patients with KIT-driven GIST whose disease progresses beyond imatinib, sunitinib and regorafenib. In patients with metastatic PDGFRα D842V-driven GIST, progression occurs in a median of approximately three to four months with available therapy.
About Avapritinib
Avapritinib is an investigational, oral precision therapy that selectively and potently inhibits KIT and PDGFRA mutant kinases. It is a type 1 inhibitor designed to target the active kinase conformation; all oncogenic kinases signal via this conformation. Avapritinib has demonstrated broad inhibition of KIT and PDGFRA mutations associated with GIST, including potent activity against activation loop mutations that are associated with resistance to currently approved therapies.
Blueprint Medicines is initially developing avapritinib for the treatment of advanced GIST, advanced systemic mastocytosis (SM), and indolent and smoldering SM. The FDA has granted Breakthrough Therapy Designation to avapritinib for two indications: one for the treatment of unresectable or metastatic GIST harboring the PDGFRα D842V mutation and one for the treatment of advanced SM, including the subtypes of aggressive SM, SM with an associated hematologic neoplasm and mast cell leukemia.
Blueprint Medicines has an exclusive collaboration and license agreement with CStone Pharmaceuticals for the development and commercialization of avapritinib and certain other drug candidates in Mainland China, Hong Kong, Macau and Taiwan. Blueprint Medicines retains development and commercial rights for avapritinib in the rest of the world.
About Blueprint Medicines
Blueprint Medicines is a precision therapy company striving to improve human health. With a focus on genomically defined cancers, rare diseases and cancer immunotherapy, we are developing transformational medicines rooted in our leading expertise in protein kinases, which are proven drivers of disease. Our uniquely targeted, scalable approach empowers the rapid design and development of new treatments and increases the likelihood of clinical success. We are currently advancing four investigational medicines in clinical development, along with multiple research programs. For
Cigarette use fell in most countries over the past three decades, but increased in some nations, notably China, a new global study says.
ReplyDeleteResearchers analyzed data from 71 countries that represent 85% of the world's population and account for more than 95% of global cigarette use.
While overall cigarette use declined, there were significant differences between countries.
About 2.5 million metric tons (MMT) of cigarettes were smoked in China in 2013, more than Russia (0.36 MMT), the United States (0.28 MMT), Indonesia (0.28 MMT), Japan (0.20 MMT), and the next 35 highest consuming countries combined.
The United States and Japan had reductions of more than 0.1 MMT over a decade, while Russian consumption plateaued, and Chinese and Indonesian consumption rose by 0.75 MMT and 0.1 MMT, respectively.
The study was published June 19 in The BMJ.
A second study in the same issue of the journal looked at global cigarette use after adoption of the World Health Organization's Framework Convention on Tobacco Control, a 2003 international treaty meant to reduce tobacco use.
It found that wealthy and European countries had a decrease in annual consumption of more than 1,000 cigarettes per adult, but low- and middle-income and Asian countries had an annual increase of more than 500 cigarettes per adult.
The findings "should motivate greater implementation of proven tobacco control policies" and "encourage more assertive responses to tobacco industry activities," the study authors wrote. Both studies were led by Steven Hoffman, from York University in Toronto.
A third study in the same issue of the journal examined data from the United States, Canada and England and found that e-cigarette rose use among teens ages 16-19 in the United States and Canada in 2017 and 2018, and that smoking increased among Canadian teens in that age group.
The study, led by David Hammond, of the University of Waterloo in Ontario, also found that use of Juul brand e-cigarettes rose in the United States, Canada and England.
"Taken together, these new studies emphasize the value of comparative research for tobacco control across different countries," Linda Bauld wrote in an accompanying editorial.
"They also warn against complacency in our attempts to address smoking, now and in the future," said Bauld, of the University of Edinburgh in Scotland.
Continued investment in international tobacco control is more important than ever, particularly in low- and middle-income countries with limited resources to fight tobacco industry attempts to undermine anti-tobacco policies, she concluded.
FDA Approves Zeposia (ozanimod) for Relapsing Forms of Multiple Sclerosis
ReplyDelete(BUSINESS WIRE)--Bristol-Myers Squibb Company (NYSE: BMY) today announced that the U.S. Food and Drug Administration (FDA) approved Zeposia (ozanimod) 0.92 mg for the treatment of adults with relapsing forms of multiple sclerosis (RMS), including clinically isolated syndrome, relapsing-remitting disease, and active secondary progressive disease.1 Zeposia,...
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