Biotechnology fund iShares NASDAQ Biotechnology Index (IBB) was up 0.1%. Sarepta Therapeutics (SRPT) rose nearly 5% after it said it intends to complete a rolling submission of its therapeutic candidate, golodirsen, after it received the final minutes from a February 2018 Type C meeting held with …
Regeneron Pharmaceuticals and partner Sanofi report new data this morning from a large, highly-anticipated trial suggesting that their drug alirocumab (Praluent) may reduce the risk of death for people with high cholesterol who have recently suffered heart attacks or strokes and are at risk of having another one or dying.
And in an unusual move, Regeneron-based on input from the nonprofit drug-pricing watchdog, the Institute for Clinical and Economic Review (ICER)-vows to cut the cost of the drug if it can get certain concessions from insurers. Regeneron and Amgen, which makes a competing drug, have struggled mightily to get payers to cover… Read more »
UNDERWRITERS AND PARTNERS
This post is part of BIO’s yearlong, bi-weekly series called Flashback Friday, highlighting newsletter stories from BIO’s past. To learn more about BIO’s history and our 25th Anniversary visit our interactive historical timeline.
Reprinted from BIONews, February/March 1996
HOW FDA IS CHANGING ITS REGULATION OF BIOTECHNOLOGY
By David A. Kessler, M.D., Commissioner, Food and Drug Administration
For the last several years, the Food and Drug Administration (FDA) has been re-examining and reforming some of the ways it regulates biologics, including medicines made through biotechnology. In the past few months, there has been considerable change in the way FDA deals with these products, and the result will be faster and more targeted reviews that save biotech companies time and money as they bring needed therapies to the public.
Many of the ideas came from productive discussion between FDA and the biotechnology community that led FDA to promise certain changes. Today, I can report that we have delivered on those promises. For the changes that have yet to be fully implemented, we are still on target for completion in the time-frames promised.
It is important to remember that everything we do as we modernize our biologic approval process builds on this agency’s fundamental mission: to protect consumers from products under our jurisdiction that are not safe or do not do what they are supposed to do and, at the same time, to promote the public health by getting therapies to consumers as quickly as possible.
This commitment to high standards for safety and efficacy has served consumers well and it has served industry well, too. FDA-approved products are the gold standard of the world. Our second responsibility ─ to get products to patients expeditiously ─ requires that we strip away unnecessary regulatory and procedural hurdles. This not only allows industry to move promising products from the bench to the bedside efficiently but also enables FDA to use its limited resources well.
So, what have we done to achieve this? The changes started early last year when the Center for Biologics Evaluation and Research (CBER) addressed two of industry’s concerns. First was the licensure of small-scale or pilot plants. For some biologic products, indust1y had to actually build a large-scale production plant before it could receive marketing approval. That led to a few costly mistakes when construction started before the drug was shown to be safe and effective. Now, for well-characterized biotech medicines, the pilot plant can be sufficient to win marketing approval.
CBER’s second decision was to separate the filing of a product license application and an establishment license application-PLAs and ELAs. In the past, the FDA required that PLAs and ELAs always be submitted together from the same company, a policy that prevented indust1y from submitting one or the other application when it was ready. We modified that policy so that the two applications could come in at separate times. This could allow a company to receive earlier feedback from FDA on an application than it would have previously.
After additional study, we announced several new measures in November that represent the most significant overhaul of the regulation of biotech drugs that the FDA has ever attempted. As biotechnology became more sophisticated, it became possible to characterize some biotech drugs as accurately as a traditional chemicalbased medicine, suggesting that the two types of drugs could be regulated in the same way. As a result, the agency decided that it could drop the ELA requirements for well-characterized biologics while not compromising the quality of these products. The proposed rule dropping that requirement was published on January 29 of this year. It must still go through a comment period and final rulemaking, but the process is well on its way.
In addition, FDA promised to consolidate the 21 different product and establishment license application forms that were used for biological products into a single form that could be used by both CBER and the Center for Drug Evaluation and Research (CDER). The draft form is now available for use for well-characterized biotech drugs. It is now available through CBER’s fax-ondemand system. It, too, will be going through clearance for publication in the Federal Register.
At this point, if a company has a well-characterized biologic for which it is ready to seek a PLA, and the agency agrees that the compound is well characterized, then the company only has to file the new single form. CBER is working with companies to make this transition period as smooth as possible. It is even likely that companies just entering the review process may never need to think about ELAs because they will have been eliminated by the time the product is approved. Of course, if some problem shows up in the rulemaking, ELAs may last longer than any of us expects.
We made several other announcements in November including the decision to no longer require biotech companies to seek approval to release each lot of a well-characterized biotech product, and the elimination of the need for companies to submit all of their promotional labeling to FDA for pre-approval before they launched a new product. Final rules need to be written, but the process is well along.
Lastly, we promised in November that we would respond to new information submitted by companies in response to a clinical hold within 30 days. We are now doing that. Both CBER and CDER have committed to that response time and are now working on needed internal procedures.
As the discussion between the agency and industry continues, we need to think beyond terms like ELAs and PLAs and focus on the science and the medicine, and then ask what we need to know to determine whether it is safe and effective, and what is the most cost-effective way for the company to submit its information so we can make good, fast decisions.
The notion that one regulatory structure fits all biologics is outmoded. We simply cannot assume that the ways we have regulated biologics in the past will work for the future. And we no longer are assuming that. I think you will find that this agency recognizes that we need to be flexible enough to, as much as possible, tailor our policies to specific product lines so that safe and efficacious medicines reach consumers as quickly and efficiently as possible.
Outspoken former pharmaceutical executive Martin Shkreli will serve seven years in prison, a sentence handed down Friday following securities fraud convictions related to two hedge funds and a drug company that he once ran.
The judge in the case said Shkreli will receive credit for time served since September, when his bail was revoked, Bloomberg reported. The average prison sentence for those convicted of securities and investment fraud in fiscal 2016, the most recent year available, was four years, according to the U.S. Sentencing Commission.
Shkreli is widely known as the former CEO of Turing Pharmaceuticals, the company that,… Read more »
UNDERWRITERS AND PARTNERS
[Source: http://ec.europa.eu/health/ageing/innovation/index_en.htm] Identifier: JUST-AG-2018Pillar: Justice programme 2014-2020Planned Opening Date: Deadline: Wed, 19 Sep 2018 17:00:00 (Brussels local time)Modification Date: Fri, 9 Mar 2018Latest information: The submission session is now available for: JUST-JCOO-EJN-AG-2018(JUST-AG)
European Biotechnology – first and foremost in European biotech · RSS · CONTACT US · EDITORIAL TEAM · ADVERTISING · ASSOCIATIONS · BIOCOM AG · UP TO DATE · LATEST NEWS · BACKGROUNDS & STORIES · APPOINTMENTS · OPINION · EVENTS · HIGHLIGHTS · THE MAG · ISSUES.
In honor of today being International Women’s Day, and March representing National Women’s Month, I’d like to profile a few of the influential women working at the intersection of science and food and agriculture.
Allison Van Eenennaam
Allison Van Eenennaam is a specialist in animal genomics and biotechnology working in the Department of Animal Science at the University of California, Davis. She is a vocal proponent of using biotechnology in agricultural to drive innovation and frequently showcases her expertise and passion on the issue 
through education and public events. She was the recipient of the 2014 Borlaug CAST Communication Award and was prominently featured in the film Food Evolution. Allison is so adamant about raising public awareness around agriculture issues, such as research funding and consumer acceptance of technology, that her and her team of scientists have created some very entertaining videos to educate the public. Fast forward to 2:33 in her team’s Gene Shop video, a parody of Macklemore’s Thrift Shop, to see and hear Allison’s solo performance.
Emma Naluyima Mugerwa
Emma Naluyima Mugerwa is a smallholder farmer and private veterinarian from Uganda who has advised other local farmers, as well as Uganda’s President, on improving livestock through genetics. She may be best known for her role in the film Food Evolution, where she worked with scientists to educate African communities on the humanitarian benefits of biotechnology in agriculture, specifically gene modification. Emma’s approach to enhancing food using biotechnology boils down to the idea that “everyone needs to eat”. During her formative years as a student, Emma was taught how to farm for both food and income by her teachers and grandmother. In honor of her work, Emma was asked to give the keynote address at the 2014 World Food Prize, where she discussed her work in Uganda and how we can continue to feed the growing world by leveraging biotechnology.
Mary-Dell Chilton
We profiled Mary-Dell Chilton in our 25th anniversary piece looking back at her work with agrobacterium. The discovery of this natural method of gene modification has led to countless innovations in the field of agriculture, contributing to the 4.58 million acres of GM crops sustainably grown around the world today. In 2013, Chilton was honored with the World Food Prize for her groundbreaking work. Like Emma Naluyima Mugerwa, Chilton views the benefits of agricultural biotechnology through a humanitarian lens. In response to winning the Award, Chilton noted “With biotechnology, we are working with nature on a higher level to more precisely determine the outcome of crops. Through ongoing research, we can continually improve their quality and productivity and do this in a way that will allow future generations to provide for their needs as well.”
Nina Fedoroff 
A molecular biologist by training, Fedoroff has long been involved in regulatory issues surrounding genetic modification of organisms (GMOs), serving on countless scientific advisory bodies including the National Science Foundation and the National Academy of Sciences (NAS). Dr. Fedoroff was an author of a 1987 NAS Council White Paper which first articulated the principle that GMOs should be regulated based on their properties, not on the method by which they were derived. In 2007, President George W. Bush awarded her the National Medal of Science. She has contributed to education and public policy pertaining to recombinant DNA and genetic modification of plants.” She lectures all over the world and is a frequent media contributor on the history and science of genetically modified organisms, science diplomacy and the future of food production.
Tamar Haspel
While Tamar Haspel is not a scientist or researcher by trade, she is an influential figure in the food and agriculture space because of her extensive reporting on food and science for the Washington Post’s Unearthed blog. Her blog addresses many of the questions, and subsequently much of the confusion, around science and food. As a journalist she is careful to consider both sides of the debate when writing about biotechnology, or using GMOs, in food production. And, Tamar doesn’t stick to her day job. According to her website, when she’s not “knee-deep in the public food conversation” she gets “dirty” raising chickens and oysters, growing her own vegetables and trying “to stay connected to the idea that food has to come from somewhere.”
Cigna, one of America’s largest health insurance providers, said Thursday it had agreed to acquire Express Scripts in a deal valued at $52 billion.
St. Louis-based Express Scripts (NASDAQ: ESRX) is the nation’s largest pharmacy benefit manager, or PBM. Organizations in that category manage prescription drug programs for commercial and government-provided health plans. Express Scripts said it’s responsible for the drug plans of more than 80 million patients in the U.S.
Cigna (NYSE: CI), based in Bloomfield, CT, said in its announcement that it was purchasing Express Scripts in a deal aimed at “making healthcare simpler” for its members,… Read more »
UNDERWRITERS AND PARTNERS
[Source: http://ec.europa.eu/health/ageing/innovation/index_en.htm] Identifier: REC-AG-2018Pillar: REC Programme 2014-2020Opening Date: Deadline: Thu, 31 May 2018 17:00:00 (Brussels local time)Modification Date: Thu, 8 Mar 2018Latest information: The submission session is now available for: REC-RGEN-WWLB-AG-2018(REC-AG)
The use of fearmongering around genetically modified (GM) crops by the organic industry has led to ballot initiatives in several states and counties looking to ban the technology. Arguably, the most famous example was highlighted in Food Evolution when Hawaii outlawed GM crops only to reverse course and rely on the technology to save one of the state’s most popular exports, the papaya, from being wiped out by disease.
In a recent piece for the Daily Camera, Mara Abbott looks at a similar case currently taking place on the U.S. mainland in Boulder County, Colorado. Abbott writes about Boulder County’s Cropland Policy, which was passed in 2016 and bans the use of GM crops on county-owned open land. As a result, many farmers that lease land from the county are prohibited from growing GM crops.
Spearheaded by county commissioners Elise Jones and Deb Gardner, the underlying goal of the ban was to decrease the use of glyphosate, a pesticide that is commonly used on GM crops. As part of the ban, county officials were to conduct research on alternatives to GM crops to help farmers who leased county land and were now on the wrong side of the law. However, as Abbott points out, the county looked to uneven the playing field in their favor during the bidding process for the research project:
The original research project vision, the Sustainable Agriculture Research and Innovation Initiative – SARII – was scrapped last fall due to accusations of a fixed process in the first bid attempt and a lack of interest from viable tenants in the second.
Fast forward more than a year later and inaction by the county allowed field bindweed to start taking over the land that was to be researched. And, as a result:
Acting on a desire to reduce the use of pesticides, the county commissioners held a piece of land out of production for over a year, hoping to use it for a research project that failed before it even started, during which time enough weeds grew on the property that the county staff had to treat them with the very chemical Jones and Gardner wished to avoid.
Pretty ironic, right? But, what’s even more ironic? While glyphosate is continuing to be used by county officials, farmers looking to lease county land to grow GM crops – which have “allowed the local farmers to be more targeted and effective with pesticides, reducing [glyphosate] use compared to previously grown conventional crops by 80 percent” – are restricted from doing so.
As Abbott writes in closing:
The GMO ban overly simplified the challenges of a complex industry.
Read Mara Abbott’s full piece here.
