Eli Lilly to refile RA remedy to FDA
INDIANAPOLIS — Eli Lilly and Incyte Corporation on Wednesday announced that, after discussions with the U.S. Food and Drug Administration in late August, Lilly will resubmit the New Drug Application for baricitinib before the end of January 2018. The resubmission package will include new safety and efficacy data.
The companies anticipate the FDA will classify the application as a Class II resubmission, which will start a new six-month review cycle. Baricitinib is a once-daily oral investigational medication for the treatment of patients with moderate-to-severe rheumatoid arthritis.
"We are committed to making life better for people living with RA. There is a significant unmet need for Americans suffering from this debilitating disease in spite of available therapies," stated Christi Shaw, president of Lilly Bio-Medicines. "We are pleased with the opportunity to provide our resubmission package for baricitinib sooner than anticipated and look forward to continuing to work with the FDA as we seek to bring baricitinib to people with RA in the U.S."
FDA approves Chemo Research’s treatment for Chagas disease
WASHINGTON — Earlier this week the U.S. Food and Drug Administration approved Chemo Research's New Drug Application for benznidazole. This is the first drug ever approved by the FDA to treat Chagas disease.
Benznidazole is an essential medicine for Chagas disease, a parasitic disease that affects an estimated 6 million to 8 million people worldwide. In the United States, an estimated 300,000 people are living with Chagas disease. Previously, it was available through the Centers for Disease Control and Prevention, but was not FDA approved.
"The approval of benznidazole represents a major milestone in the U.S. and global response to address Chagas disease," stated Nick Haggar, CEO of Chemo Group. "We are excited to have the opportunity to fulfill this medical need and make a meaningful difference in the lives of Chagas patients. The FDA's decision approves benznidazole for children ages 2-12 years old. We look forward to continuing our strong collaboration with the FDA to expand indication."
Chemo Group played a central role in registering benznidazole with the FDA, in close collaboration with its U.S.-based pharmaceutical division Exeltis, corporate social responsibility partner Mundo Sano, with the support of the Drugs for Neglected Diseases initiative, a non-profit drug development organization. DNDi supported U.S. registration through provision of technical expertise and sharing of data from DNDi-led clinical trials. The approval of benznidazole will facilitate the delivery of life-saving medical treatment to people with Chagas disease.
"I am thrilled that we are taking a giant step forward in our journey to overcome the many barriers to Chagas treatment," said Silvia Gold, president of Mundo Sano. "I would like to express my heartfelt thanks to the caring and dedicated doctors who fight for Chagas patients each day. I also send a warm thank you to all the researchers and experts diligently working to expand knowledge and understanding of this complex disease. It is a privilege to celebrate this milestone together with a passionate, dedicated community. Our work does not stop here, and we will work hand in hand to transform the lives of people suffering from Chagas disease."
Looking ahead, Chemo Group will continue working in collaboration with Exeltis, Mundo Sano and Drugs for Neglected Diseases initiative (DNDi) to overcome barriers to treatment of Chagas disease. Exeltis will be responsible for the product distribution in the US market. In parallel, Mundo Sano and DNDi will pursue efforts to boost access and increase patient awareness.
FDA approves first-ever gene therapy treatment for acute lymphoblastic leukemia
SILVER SPRING, Md. — The U.S. Food and Drug Administration issued a historic action today making the first gene therapy available in the United States, ushering in a new approach to the treatment of cancer and other serious and life-threatening diseases.
The FDA approved Novartis' Kymriah (tisagenlecleucel) for certain pediatric and young adult patients with a form of acute lymphoblastic leukemia. Kymriah is the first therapy based on gene transfer approved by the FDA.
“At Novartis, we have a long history of being at the forefront of transformative cancer treatment,” said Joseph Jimenez, CEO of Novartis. “Five years ago, we began collaborating with the University of Pennsylvania and invested in further developing and bringing what we believed would be a paradigm-changing immunocellular therapy to cancer patients in dire need. With the approval of Kymriah, we are once again delivering on our commitment to change the course of cancer care.”
“We are so proud to be part of this historic moment in cancer treatment and are deeply grateful to our researchers, collaborators, and the patients and families who participated in the Kymriah clinical program,” added Bruno Strigini, CEO of Novartis Oncology. “As a breakthrough immunocellular therapy for children and young adults who desperately need new options, Kymriah truly embodies our mission to discover new ways to improve patient outcomes and the way cancer is treated.”
"We're entering a new frontier in medical innovation with the ability to reprogram a patient's own cells to attack a deadly cancer," stated FDA Commissioner Scott Gottlieb. "New technologies such as gene and cell therapies hold out the potential to transform medicine and create an inflection point in our ability to treat and even cure many intractable illnesses. At the FDA, we're committed to helping expedite the development and review of groundbreaking treatments that have the potential to be life-saving."
"Kymriah is a first-of-its-kind treatment approach that fills an important unmet need for children and young adults with this serious disease," said Peter Marks, director of the FDA's Center for Biologics Evaluation and Research. "Not only does Kymriah provide these patients with a new treatment option where very limited options existed, but a treatment option that has shown promising remission and survival rates in clinical trials."
Kymriah, a cell-based gene therapy, is approved in the United States for the treatment of patients up to 25 years of age with B-cell precursor ALL that is refractory or in second or later relapse.
Kymriah is a genetically-modified autologous T-cell immunotherapy. Each dose of Kymriah is a customized treatment created using an individual patient's own T-cells, a type of white blood cell known as a lymphocyte. The patient's T-cells are collected and sent to a manufacturing center where they are genetically modified to include a new gene that contains a specific protein (a chimeric antigen receptor or CAR) that directs the T-cells to target and kill leukemia cells that have a specific antigen (CD19) on the surface. Once the cells are modified, they are infused back into the patient to kill the cancer cells.
Novartis plans additional filings for Kymriah in the U.S. and EU later this year, including applications with the FDA and European Medicines Agency, for the treatment of adult patients with r/r diffuse large B-cell lymphoma (DLBCL). Additional filings beyond the U.S. and EU are anticipated in 2018.