Today (October 12, 2017) FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee unanimously recommended approval of Spark Therapeutics, Inc.’s Biologics License Application (BLA 125610) for Voretigene Neparvovec (also called LUXTURNA), a gene therapy for the treatment of patients with vision loss due to confirmed biallelic RPE65 …
If you read our blog, you already know that the 21st Century Cures Act included a provision that created the Regenerative Advanced Therapy designation program, or “RAT” (see our previous coverage of the Act here). You will also already know that the Center for Biologics …
On August 29, 2017, FDA approved Chemo Research’s NDA for benznidazole for treating children with Chagas disease and granted the sponsor a Tropical Disease Priority Review Voucher (PRV) (see FDA Press Release here). This is the first drug to treat Chagas disease, a life-threatening parasitic disease that affects …
On August 18, 2017, President Trump signed into law the Food and Drug Administration Reauthorization Act of 2017 (“FDARA”). In addition to reauthorizing and amending several drug and medical device provisions that were scheduled to sunset, FDARA also makes several changes to the law concerning …
FDA’s Center for Devices and Radiological Health (CDRH) recently announced in the Federal Register and the FDA Voice blog that it has scheduled the first-ever meeting of the Patient Engagement Advisory Committee (PEAC), which will provide recommendations to FDA regarding the regulation of medical devices …
On July 27, 2017, the Drug Information Association (DIA) journal, Therapeutic Innovation & Regulatory Science, published recommendations from the Clinical Trials Transformation Initiative (CTTI) Patient Groups & Clinical Trials (PGCT) Project team, of which HP&M Attorney James E. Valentine is a member, on the best …
On July 25, 2017, FDA announced the availability of a guidance on “IRB Waiver or Alteration of Informed Consent for Clinical Investigations Involving No More Than Minimal Risk to Human Subjects.” FDA issued this guidance for immediate implementation, without initially seeking prior comment, based on …
On July 11, 2017, the Government Accountability Office (GAO) provided Congress with an assessment report on FDA’s Expanded Access (“EA”) program. EA programs allow patients with serious or life-threatening diseases and no satisfactory therapy available to access investigational drugs outside of a clinical trial (report …
In a new paper, titled “The Drug Innovation Paradox,” Professor Erika Lietzan of the University of Missouri School of Law (and a friend and fellow Hatch-Waxman addict) examines the relationship between the incentives offered under the law to develop drug products and the research and …
The latest issue of FDLI’s Update Magazine features an interview conducted by Hyman, Phelps & McNamara, P.C.’s James E. Valentine. In the interview, James Valentine talks with rare disease patient advocate Christine McSherry about patient engagement in the drug development and approval process, focusing on …
On April 14, 2017, Congress released a discussion draft of a bill to reauthorize the FDA’s various user fee programs, commonly referred to as “the UFAs.” The FDA Reauthorization Act (FDARA) would renew the Agency’s authority to collect user fees in Fiscal Years 2018 through …
On March 15-17, 2017, the American Society for Experimental Neurotherapeutics (ASENT) held its 19th Annual Meeting in Rockville, MD. The meeting brought together clinical investigators, pharmaceutical industry sponsors, officials from drug regulatory agencies, and patient advocacy organizations to address and advance the science of neurotherapeutics. On …
On January 18, 2017, the U.S. Department of Health and Human Services along with 15 other federal agencies issued the Final Rule to revise the Federal Policy for the Protection of Human Subjects, known as the “Common Rule.” This 1991 set of regulations created a …
FDA recently put forth its first interpretation of the Regenerative Advanced Therapy (“RAT”) designation program, which was established by section 3033 of the 21st Century Cures Act. Although it consists of just a few short paragraphs on FDA’s website (here), the guidance confirms that FDA …
The 21st Century Cures Act was signed into law on December 13, 2016. The Act addresses acceleration of medical product discovery, development, and delivery. Hyman, Phelps & McNamara, P.C. ("HP&M") will hold complimentary webinars on Thursday, January 12, 2017 from 12:00-1:30 PM (Eastern) and Wednesday, …
