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