OPEN Act Introduced to Repurpose Drugs for Rare Diseases

EveryLife Foundation Applauds Congress for Support of Bipartisan Legislation

News provided by

EveryLife Foundation for Rare Diseases

Nov 24, 2014, 08:00 ET

WASHINGTON, Nov. 24, 2014 /PRNewswire-USNewswire/ -- Last week, Representatives Gus Bilirakis (R-FL) and G.K. Butterfield (D-NC) took a bold bipartisan step on behalf of patients with rare diseases by introducing the OPEN Act. Original co-sponsors of the legislation include Representatives McCaul (R-TX) and Hastings (D-FL). The Act will incentivize drug makers and innovators to "repurpose" major market drugs for life-threatening rare diseases and pediatric cancers, which opens the door to the development of hundreds of safe, effective, and affordable treatments for rare disease patients.

"The OPEN Act has the potential to result in hundreds of new drugs and treatments for individuals with rare diseases, as well as a new surge in biotechnology jobs and investments. This bill can help millions of people by ensuring medications are safe and effective for rare conditions, and can be reimbursed through insurance coverage," said Representative Bilirakis.

"The OPEN Act is landmark legislation for patients in the rare disease community. This bill provides hope to the millions of Americans who are suffering from rare diseases and are desperately awaiting treatments. We are proud and thankful to see Congress taking a bipartisan approach to solving this challenge on behalf of patients and their families," said Emil Kakkis, MD, PhD, President of the EveryLife Foundation for Rare Diseases.

Modeled on the incentive programs in the Best Pharmaceuticals for Children Act (BPCA), the OPEN Act would make available to drug companies an "Orphan Product Exclusivity Extension," which would provide an additional six months of exclusivity to the patent life of the major market drug being repurposed for rare disease treatment so long as the sponsor company establishes that the therapy: (1) is designated to treat a rare disease and (2) obtains a rare disease indication from FDA on the drug label.

There are over 7,000 rare diseases, impacting 30 million Americans, yet 95% of these diseases have no treatments.

The EveryLife Foundation for Rare Diseases, headquartered in Novato, CA, is dedicated to accelerating biotech innovation for rare disease treatments through science-driven public policy. We can do more with the science we already have and bring life saving treatments to millions of people suffering from rare diseases.

Contact: Julia Jenkins
[email protected]
415-884-0223

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SOURCE EveryLife Foundation for Rare Diseases