Weekly Roundup 10.27.17

The leaves are falling around us and still no first frost. And we are just a few short days from the knocks at the door by young and sometimes not so young seeking sugar handouts while dressed in costume. If you don’t have your candy in hand, better run to the store. Before you do – a few things that happened this week:

  • FDA Issues Draft Guidance for “Breakthrough Devices – This week the agency published in the Federal Register a draft guidance to enhance the regulatory approval for breakthrough devices. As with breakthrough status designation for drugs already in existence, FDA is seeking to enhance the approval of devices, however, the status for devices differs from that of drugs in important respects. The particulars of the program as described in the draft guidance are detailed by the Regulatory Affairs Professional Society.  In short, while allowing for an enhanced pathway to approval, it is not a guarantee of faster approval. In addition, Commissioner Gottlieb released a statement respecting the development of a new medical device evaluation tool for use in cardiovascular devices that ties patient output with device development as part of the FDA’s Medical Innovation Access Plan
  • Duchenne Treatment Candidate Gets Complete Response Letter – Last year the agency gave accelerated approval to a drug for use to treat a subset of patients with Duchenne muscular dystrophy and earlier this year another that could be more broadly applied. However, this week in issuing a complete response letter for another candidate treatment – one that would treat patients with Duchenne due to a nonsense mutation in the dystrophin gene, FDA said that the compound needed further evidence of efficacy which reflected some of the input from the FDA Advisory Committee held earlier this year. In a statement, the sponsor has said that an appeal to the decision will be filed. The webcast and meeting minutes from the advisory committee meeting can be found here, though the transcript is not yet posted. 
  • Opioid Public Health Emergency Declared – A long-anticipated announcement by the White House declaring the opioid epidemic a public health emergency was announced this week. The measure opens up the way for government agencies to work to take measures to address the spectrum of complex issues that make up the epidemic, but fell short of the expectations of many to provide new funding for states. Last week in a panel discussion at the Washington Post called Addiction in America, Senators Portman, Hassan and Manchin expressed the hope that the declaration would bring with it additional funding. In addition, Commissioner Gottlieb issued a statement from FDA outlining the steps the agency has been taking on opioids. 

Upcoming Events to Keep an Eye on This Week

Regulatory Developments in Pharma/Biotech/Devices

Photograph by Lynne Bertram, Woodstock, Vermont

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Weekly Roundup 10.20.17

The first frost on the pumpkin is not far away. The garden is beyond tired and leaves are filling the gutters. I find myself craving comfort foods and am anxious for that first fire in the fireplace. Time marches on, and so do we. Here is a bit of what happened this week:

  • Second CAR-T Approval – During what has most certainly been an important year in new approvals in oncology, this week FDA announced another CAR-T therapy approval (chimeric antigen receptor T-Cell therapy). CAR-T treatments take human cells and re-train them to fight cancer. The new treatment – Yescarta (axicabtagene ciloeucel) – is indicated to treat adults with certain types of large B-cell lympohoma, the most common form of non-Hodgkin lymphoma, accounting for 3 out of 5 cases. In the FDA press release, Commissioner Gottlieb noted that the approval continues “the momentum of this promising new area of medicine” and re-stated the commitment of the agency to expedite such therapies and signaled that a new and comprehensive policy will be forthcoming to outline support for cell-based regenerative medicine. As noted in the posting on Wednesday, it also continues the momentum of new cancer therapies approved this year. Administration of the therapy will take place through hospitals that have undergone certification and training. Yescarta was designated by FDA as having Breakthrough Therapy status, had Priority Review and is an Orphan Drug. You can find the company release, which among other things contains information about a Patient Assistance Program, here
  • FDA, REMS and Opioids – In 2013, the HHS Office of the Inspector General (OIG) issued a report that criticized FDA’s oversight of REMS programs (Risk Evaluation and Mitigation Strategies) that are put into place when drugs are approved in order to shore up safe use. Now the OIG has scheduled a new investigation that is specific to the REMS programs of FDA approved opioids. The analysis will focus on how FDA determined the need for REMS programs for products as well as examine the extent to which the agency has monitored the implementation of the programs by companies to mitigate abuse. The fact that such an investigation is underway may pave the way for enhanced attention to these programs by FDA prior to the report coming out. 

That’s it for me this week folks, Have a great weekend!

Upcoming Events to Keep an Eye on This Week

Regulatory Developments in Pharma/Biotech/Devices


Photograph by Lynne Bertram, Woodstock, Vermont


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Is FDA Set for Another Banner Year of Approvals?

In 2015, FDA experienced a banner year with respect to the number of novel drugs and biologics that were approved by the agency – 45 all told. Since then, the 21st Century Cures Act was passed with the goal, among other things, of facilitating the approval of new medicines. That piece of legislation is still in the process of implementation, but even so, it appears that FDA could be poised for another banner year of new approvals. 

Comparing the first three quarters of the last three years, one can see that 2017 is on track to be an important year for approvals and if it keeps up at the current pace, could overtake 2015 in bringing new medicines out of the pipeline.

As one can readily see, by the end of the third quarter, new approvals were outstripping 2015 – 34 in 2017 compared to 27 in 2016 and twice the number of 17 just last year. 

There are a lot of factors that go into this in terms of timing, and one of them is the way the NDA’s stack up. Looking at the last quarter of 2016 there were 12 breakthrough therapy requests submitted to FDA, more than any other quarter since inception of the program, and another 9 during the first two quarters. As breakthrough therapy status means FDA will expedite review of the drug candidate, this could be one of the factors in the increased number of approvals for this year.

And that also means that the approvals were not for “me-too” drugs either. As you can see below, the therapeutic categories for approvals were highly concentrated in cardio and oncology – two of the biggest causes of mortality as well as diseases of the central nervous system or mental health – Multiple Sclerosis, bipolar disorder and schizophrenia. 

There still need to be eleven approvals during the last quarter to tie 2015. At the end of the fourth quarter, we’ll take another look at year end to see how we ended up. But the signs right now are pointing to not only a record year for approvals, one one in which the approvals are noteworthy and demonstrative of the policy drive the past few years to improve the process. 






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Weekly Roundup 10.13.17

It is Friday the 13th – one of two this year (the first was in January) and we sit a few weeks away from Halloween. Pumpkins on porches this month, in pies next month. 

A few weeks ago you may have noticed I have added a regular feature to the Weekly Roundup posting by listing upcoming interesting events of note – Congressional Hearings, Advisory Committee meetings and conferences.

Beginning this week, I am also adding a section below to note announcements either through press releases or through media reports that might interest you as well. 

In the meantime, here is a little of what happened this week: 

  • Commissioner Gottlieb Testimony on Opioids – Last week the Senate HELP Committee held a hearing on the federal response to the opioid epidemic. The Committee site has a video of the hearing which included Commissioner Gottlieb along with representatives from the Substance Abuse and Mental Health Services Administration, the Centers for Disease Control and Prevention and the National Institutes of Health – and the official written testimony for all of them was posted this week on the FDA site. The testimony sites two primary reasons for the growth of the opioid epidemic – (1) the significant rise in opioid analgesic prescriptions, particularly to treat pain that we now know to be high-risk with high doses for long durations, and (2) the lack of the healthcare system to identify and engage people with addiction with treatment, particularly medication assisted treatment. FDA’s portion of the testimony centered on four key areas:
    • Supporting the development of Abuse-Deterrent Formulations;
    • Development of alternatives to opioids for the treatment of pain;
    • Working to influence changes in prescribing patterns to limit exposure through number of pills and duration of exposure
    • Use of REMS to expand education among prescribers 
  • FDA AdComm Recommends Gene Therapy – On Thursday the FDA Cellular, Tissue and Gene Therapy Advisory Committee unanimously recommended approval of an investigational gene therapy for the treatment of confirmed balletic RPE65-mediated inherited retinal disease (IRD). If approved Luxturna™ (voretigene neparvovec) would be the first gene therapy for a genetic disease in the U.S. The data used for approval included four year efficacy data and the first Phase 3 clinical trial data ever conducted for a genetic disease, according to the company press release. There are not existing pharmacologic treatments for this IRD and the BLA is under Priority Review with the FDA, having a PDUFA date of January 12, 2018. 
  • NIH Partners with Biopharma on Cancer – NIH announced this week that it would be launching a new public-private partnership called PACT – the Partnership for Accelerating Cancer Therapies in conjunction with eleven biopharmaceutical companies in support of the Cancer Moonshot effort. The effort will aim to identify and validate biomarkers for use in cancer research to advance immunotherapy treatments and will be managed by the Foundation for NIH with the FDA serving in an advisory capacity. Identification and testing of the biomarkers for use in immunotherapy research will help define a consistent generation of data to support analysis and will facilitate progress by acting jointly rather than leaving it up to individual entities to perform the research. Both NIH and each company will be contributing funding over a period of five years. You can see the full press release from NIH, with the names of the companies involved, here

Upcoming Items to Keep an Eye on This Week

  • Senate HELP Committee Hearing – October 17 – The Cost of Prescription Drugs – How the Drug Delivery System Affects What Patients Pay – More information can be found here
  • Endocrinologic and Metabolic Drugs Advisory Committee Meeting – October 18 – EMDAC will be meeting to consider NDA for semaglutide injection to improve glycemic control in adults with Type 2 Diabetes. More information can be found here

Regulatory Developments in Pharma/Biotech/Devices – Links to news either reported through company press releases or media reports

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Weekly Roundup 10.6.17

We are already arriving at Columbus Day Weekend. The year has gone by breathtakingly fast from my perspective. We are only a month away from changing the clocks, but it will be here before you know it. I am already craving the comfort foods of winter. In the meantime, here is a bit of what happened during our speedy week – and a quick reminder that Monday is a federal holiday.

  • No Love for FDA – In what has become a widely reported transmission from FDA, the agency this week posted a warning to a food manufacturer that included, among other things, an admonishment about an unusual ingredient listed in the label.  Specifically, FDA concerned itself that included in a label for granola, the manufacturer had included the word “love” to which FDA registered disapproval. “Ingredients required to be declared on the label or labeling of food must be listed by their common or usual name…Love is not a common or usual name of an ingredient, and is considered to be intervening material because it is not part of the common or usual name of the ingredient.” Golly FDA reviewer, I don’t know about your household, but in mine love is quite a common ingredient. This quite literal exercise in enforcement led to a lot of coverage respecting the letter, more than would be usual. While the letter addressed many items, the issue of the ingredient of love was the part that was widely reported which makes an interesting learning lesson in and of itself. 
  • First Zika Test Approved for Blood System – The agency announced the approval of the first assay to screen donated blood and blood components for the Zika virus – and not meant for use in testing or screening individuals. Up to this point, blood screening was being done utilizing a screening test under investigative review in order to comply with FDA’s guidance for screening issued in August 2016 in response to the widening exposure of Zika in the U.S. 
  • Access to Investigational Drugs Testimony – Recalling his own experience as a cancer patient and taking off-label treatment, Commissioner Gottlieb spoke before a House Energy and Commerce, Subcommittee on Health hearing this week on early access to investigational drugs. He stated that in recent years the agency had received over 1000 applications for early access to investigational drugs and that 99 percent of those applications were granted and that the processing was very quick and had in fact been streamlined this past year. He also said that FDA was working with NIH to provide patient advocacy groups with more information about expanded access and had launched a tool for patients to get more information online as well as addressing concerns raised in a recent GAO report regarding adverse events associated with expanded access. Finally he addressed issues associated with proposed “right to try” legislation before the Senate. 

That’s it for me this long weekend. Have a good one folks and be well. 

Upcoming Items to Keep an Eye on This Week

  • Patient Engagement Advisory Committee Meeting – The meeting will be October 11 and 12 and the topic will be the subject of patient input into the clinical trials for devices. More information can be found here
  • Cellular, Tissue and Gene Therapy Advisory Committee Meeting – The committee will meet to consider a BLA for a new therapy for a type of retinal dystrophy on October 13. More information can be found here
  • Dermatologic and Ophthalmic Drugs Advisory Committee Meeting – The committee will meet to consider the NDA for a new therapy for reducing intraocular pressure in patients with glaucoma or ocular hypertension. More information can be found here
  • International Conference on Viral Hepatitis – Chicago, Illinois October 9-10 – for more information, visit here
  • Senate HELP Committee Hearing – October 17, Part II on The Cost of Prescription Drugs: How the Drug Delivery System Affects What Patients Pay – check it out here


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