BioPharm International

 Merck’s blockbuster cancer drug Keytruda (pembrolizumab) for the treatment of cancers with a specific biomarker. This new approval marks the first-time FDA has approved a treatment based on a common biomarker, as opposed to the location in the body where the tumor originated.

	“This is an important first for the cancer community,” said Richard Pazdur, MD, acting director of the Office of Hematology and Oncology Products in FDA’s Center for Drug Evaluation and Research and director of the FDA’s Oncology Center of Excellence. “Until now, the FDA has approved cancer treatments based on where in the body the cancer started-for example, lung or breast cancers. We have now approved a drug based on a tumor’s biomarker without regard to the tumor’s original location.”

	In clinical trials, of the 149 patients who received the drug, 39.6% had a complete or partial response. For 78% of these patients, the response lasted for six months or more, FDA wrote.

	Keytruda is approved for other indications including melanoma and non-small cell lung cancer, among others. The drug was listed as a topic selling drug in Merck’s 

Articles

FDA Approves Keytruda for Cancers with Specific Biomarker

	On Dec. 16, 2016, the European Medicines Agency (EMA) 

 the launch of a pilot project, scheduled to start February 2017, that will test the “value and feasibility of tailored scientific advice for the development path of biosimilar medicines.” During the project, the agency will provide biosimilar developers with advice on the conduction of studies and tests on the “basis of the quality, analytical, and functional data they have already available for the medicine.”

	To obtain market authorization of biosimilars in the European Union, developers must establish similarity to the reference product. EU guidelines recommend a stepwise development of biosimilars in which required studies and tests are based on already accumulated data. The EMA pilot project will include an in-depth review of available quality, analytical, and functional data. Advice based on these data will be given to developers so they may tailor their studies and tests during development. The agency hopes this advice will be beneficial to market authorization applicants when making development strategies, but according to EMA, this advice will not be a formal pre-assessment of the data submitted.

	The pilot project, which is open to any company seeking biosimilar development advice, is expected to run until six scientific advice requests have been completed. A maximum number of one request will be accepted per month. Companies interested will have a pre-submission meeting. An additional month will be added to normal scientific advice timelines so that EMA’s Scientific Advice Working Party can review the requests. 

EMA Launches Biosimilars Project

	On Nov. 10, 2016, the Broad Institute and IBM Watson Health announced the start of a research initiative aimed at discovering the basis of cancer drug resistance. The research will last for five years and will cost $50 million. The project will study drug-resistant tumors in order to better understand how cancers become resistant to therapies. After the project is completed, the data will be available to the scientific community and researchers worldwide, the organizations noted in a statement.

	Cancers become drug resistant because of mutations, and the cause of this resistance is not fully understood. To help better understand this resistance, the Broad Institute will generate tumor genome sequence data from patients who initially responded to treatment but then became drug resistant. Broad will use genome-editing methods to conduct large-scale cancer drug resistance studies to identify tumor vulnerabilities. Scientists at IBM will use Watson, IBM’s cloud-based data analytics system, to identify genomic patterns that may help researchers predict drug sensitivity and resistance.

	“Currently, cancer researchers have access to genomic information from only a few hundred drug-resistant cancers samples. In addition to the goals of this specific study, IBM and Broad are committed to advancing cancer research by sharing the data from thousands of tumor samples with the scientific community to accelerate progress everywhere against cancer,” said Todd Golub, MD, chief scientific officer and founder of the cancer program at the Broad Institute. “What we and many others will learn with this information will have important implications for the future of cancer precision medicine and cancer diagnostics.”

Broad Institute and IBM Begin Cancer Drug Resistance Research

	On Sept. 7, 2016, the US Department of Health and Human Services’ Office of the Assistant Secretary for Preparedness and Response (ASPR) announced an $8.2-million contract award to Moderna Therapeutics of Cambridge, Massachusetts, to help speed the development of a novel vaccine to prevent the spread of the Zika virus. ASPR’s Biomedical Advanced Research and Development Authority (BARDA) will provide technical assistance, oversight, and funding to advance the Zika vaccine’s clinical development.

	Under the initial four-year agreement, BARDA will support a Phase I clinical trial, toxicology studies, vaccine formulation, and manufacturing. If additional funding is identified, the agreement could be extended up to a total of five years and a total of $125.5 million to support Phase II and III clinical trials, as well as large-scale manufacturing.

	Moderna’s vaccine uses messenger RNA (mRNA) vaccine technology. mRNA is a molecule that carries specific genetic codes to parts of the cell. This type of vaccine uses mRNA containing the genetic sequence of the Zika virus to generate an immune response in people. Producing vaccine from this type of technology is rapid compared to other vaccine technologies that require the growth and purification of an attenuated or inactivated virus.

	This new mRNA Zika vaccine development project is part of ASPR/BARDA’s comprehensive integrated portfolio approach for advanced research and development, innovation, acquisition, and manufacturing of vaccines, drugs, diagnostic tools, and non-pharmaceutical products for public health emergency threats. In addition to emerging infectious diseases, these threats include pandemic influenza, chemical, biological, radiological, and nuclear agents, and antimicrobial resistant pathogens.

Author | BioPharm International

Articles

FDA Approves Keytruda for Cancers with Specific Biomarker

EMA Launches Biosimilars Project

Broad Institute and IBM Begin Cancer Drug Resistance Research

HHS Awards $8.2 Million to Moderna for Zika Vaccine Development