The Alliance for Regenerative Medicine Issues Statement in Response to the FDA Advisory Committee's Unanimous Endorsement of Spark Therapeutics' Gene Therapy Product Candidate Luxturna for Inherited Retinal Dystrophy

WASHINGTON, D.C. – October 13, 2017

In response to today’s FDA Advisory Committee’s 16-0 favorable review of Spark Therapeutics’ Luxturna, the Alliance for Regenerative Medicine has issued the following statement, attributable to Janet Lynch Lambert, CEO, ARM:

The Alliance for Regenerative Medicine (ARM) is extremely pleased and encouraged by today’s U.S. FDA Advisory Committee’s unanimous endorsement of Spark Therapeutics’ AAV gene therapy product candidate, voretigene neparvovec (Luxturna), to treat blindness-causing inherited retinal dystrophy. This marks significant progress in the effort to provide thousands of patients access to improved vision, and a much higher quality of life.

Today’s recognition of this technology’s impressive capabilities moves the entire field forward, to the benefit of patients and their families worldwide.

With more than 70 regenerative medicine and advanced therapy products in Phase 3 clinical trials, and hundreds more in earlier stage, ARM will continue to work closely alongside therapeutic developers and other stakeholders to ensure our healthcare system is supportive of broad patient access to these transformative products.

About the Alliance for Regenerative Medicine
The Alliance for Regenerative Medicine (ARM) is an international multi-stakeholder advocacy organization that promotes legislative, regulatory and reimbursement initiatives necessary to facilitate access to life-giving advances in regenerative medicine worldwide. ARM also works to increase public understanding of the field and its potential to transform human healthcare, providing business development and investor outreach services to support the growth of its member companies and research organizations. Prior to the formation of ARM in 2009, there was no advocacy organization operating in Washington, D.C. to specifically represent the interests of the companies, research institutions, investors and patient groups that comprise the entire regenerative medicine community. Today, ARM has more than 270 members and is the leading global advocacy organization in this field. To learn more about ARM or to become a member, visit