Yesterday, Kymriah, a gene therapy made by Novartis that treats an aggressive type of leukemia was approved for children and young adults. The new commissioner of the FDA, Dr. Scott Gottlieb, remarked on the FDA's approval of the first gene therapy in the United States.

For our Duchenne community on the brink of starting gene therapy clinical trials, this news is another piece of evidence showing the tremendous strides this technology has made since the 1990s and its early days of research.
  

This news also reinforces PPMD's decision to heavily invest, with your help, in gene therapy through our Gene Transfer Initiative announced in January of this year -- an investment that began with a $2.2 million grant to Dr. Jerry Mendell, Dr. Louise Rodino-Klapac (co-PIs), and Nationwide Children’s Hospital. This trial is making tremendous strides towards entering the clinic in the last quarter of 2017. We hope you will join us for our upcoming webinar with Dr. Mendell on Wednesday, September 6 at 2 PM ET to learn more.

Yesterday's approval means that there are regulatory and commercial pathways for cell and gene based therapy. It means that  you can put living DNA into a human and it can do its job. And it means that years of scientists making seemingly incremental advances can all come together and result into a giant step forward. A step forward that we believe will move this technology in a direction that will eventually benefit our community, our children.

PPMD is pleased for the patients that can benefit from Kymriah and very optimistic about what gene therapy can do to help end Duchenne.

Read the statement from Dr. Scott Gottlieb, FDA: 

Remarks by Scott Gottlieb, M.D
Commissioner of Food and Drug Administration
August 30, 2017
White Oak, MD

Today is an important milestone in a long journey we’ve been on to transform clinical medicine by using modern advances in genomics.

As one part of that journey, researchers spent many decades working on technologies that could modify human cells and tissue using the tools of gene therapy. This was always held out as a way to alter the course of many vexing diseases, and maybe even deliver the ability to cure some deadly disorders.

There have been many challenges on that path. We’ve witnessed hardships and even tragedy along the way. We’ve also realized certain triumphs. Today a pivotal leg in that journey is complete. The science has reached a point of superiority where enough of the components of these endeavors are worked out. We can deliver effective therapies to patients. We can deliver on the original promise.

FDA is announcing the approval of the first gene therapy product in the United States. FDA is approving the drug Kymriah for treatment of certain pediatric and young adult patients with a devastating and deadly form of leukemia.

Many people have contributed to the science that made today possible. There are many patients who committed themselves to clinical trials that have enabled these opportunities to advance. There are many people at the company that developed this product – the drug maker Novartis -- who remained steadfast to a challenging and risky scientific vision. There are many people at FDA who worked for decades on defining a framework for properly developing and evaluating these platforms, while this field underwent intense scrutiny and setbacks.

And there are many researchers across the world who made incremental advances that all consolidated to give us this scientific moment -- the opportunity to have this effective new therapy that will help young cancer patients; and to change the face of modern drug development in the process.

Gene therapy products are now being studied in many diseases and conditions, including genetic disorders, autoimmune diseases, heart disease, cancer, diabetes, and HIV/AIDS. At FDA, we look forward to working with the research and development community to advance potential new therapies in these serious disease areas—which affect millions of Americans and their families.

Today is just the first approval in this promising new class of medical products. Other similar medical opportunities lie just behind this milestone. To give you just one example related to the agency’s current action; FDA has granted more than 550 active investigational new drug applications related to gene therapy products, and has 76 active investigational new drug applications related to CAR-T cell products.

Providing patients with timely access to advances in medical products is a critical component of FDA’s work. We’re committed to expediting the development of new and ground-breaking therapies that have the potential to be life-saving.

Toward these ends, today’s review and approval of Kymriah happened quickly. It’s being approved seven months after FDA received the application. The approval also involved a cross-agency approach in which the FDA’s top oncologists and gene therapy experts worked together to help ensure Kymriah would be available as quickly as possible. This program alignment is an organizational approach that we intend to pursue more widely at FDA, across other therapeutic areas, as a way to improve our efficiency, and deepen our scientific collaboration.

I’d like to personally thank the FDA staff and leaders in the Center for Biologics Evaluation and Research and the Oncology Center of Excellence for their collective efforts to make today possible. They worked to pioneer and implement this more collaborative scientific model for drug review at FDA.

Today’s announcement was many years in the making. At FDA, we’re committed to doing our part to help advance breakthrough science that has the potential to deliver outsized benefits to certain patients who aren’t served well by conventional therapies. In the coming months, FDA will be advancing new policies to help make the development of very novel technologies more efficient. I will also continue to advocate for policies that improve access to these treatments. Today, my colleague at the Centers for Medicare and Medicaid Services, Seema Verma, will take steps to facilitate this access, by pursuing more novel approaches to coverage of these kinds of highly targeted, highly specialized therapies.

I want to thank you for joining us today and as we move forward together to unlock the full potential of gene therapy for patients. I’d like to introduce Dr. Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research--the FDA center that is tasked with regulating cellular and gene therapies.

Learn more

Join us for the second part of PPMD's new Gene Therapy Webinar Series on Wednesday, September 6 at 2 PM ET. Dr. Jerry Mendell of Nationwide Children’s Hospital will discuss his upcoming gene therapy trial, including trial design, inclusion/exclusion criteria, and timelines.