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Learn more about how the Orphan Drug Act enables researchers to discover breakthrough cures for rare diseases
RDCC member companies are commemorating the 40th anniversary of the Orphan Drug Act (ODA) by sharing how its policies have enabled their therapeutics to reach rare disease patients around the world who have had limited or no treatment options. For 40 years, the ODA has empowered biotech companies to invest in rare disease research and innovation that would otherwise be impractical or impossible to pursue.
Read how biotech companies have relied on incentives established by the ODA, like the Orphan Drug Tax Credit, to deliver new treatments for patients living with rare diseases like hyperoxaluria type 1 and acute hepatic porphyria, facioscapulohumeral muscular dystrophy, Batten disease, enzyme deficiency diseases, tumor-induced osteomalacia (TIO), X-linked hypophosphatemia (XLH), rare blood disorders including pediatric PK deficiency, thalassemia and sickle cell disease, and more. With 95% of rare diseases still lacking a treatment option today, policymakers should renew their commitment to protecting and building on the ODA to ensure continued innovation and investment.
Pushkal Garg, MD, Chief Medical Officer and EVP, Development & Medical Affairs of Alnylam Pharmaceuticals
“Alnylam has obtained 5 drug approvals in the past 5 years, of which 4 were for medicines which have been granted an Orphan Drug Designation. We believe the speed and agility with which we were able to bring these transformative medicines to patients who are living with some of the world’s rarest diseases, such as primary hyperoxaluria type 1 and acute hepatic porphyria, would not have been possible without the provisions of the Orphan Drug Act. The provisions of the Act, importantly those that offer financial incentives and marketing exclusivity are vital for innovative Companies like Alnylam who have made sizeable investments in rare disease drug development, thereby bringing novel therapeutics and hope to patients around the world who have had limited or no treatment options, while also allowing us to continue to invest in our R&D engine to fuel the discovery and development of future breakthroughs in medicine.”
Giacomo Chiesi, Head of Chiesi Global Rare Diseases
“The Orphan Drug Act created a framework whereby industry can focus on long-term investments in R&D, but there is much work to be done. Policymakers must understand that rare disease patients and families have unique needs. Working together for the unified purpose of delivering treatments for rare disease patients is a public health imperative.”
Santiago Arroyo, M.D., Ph.D. Chief Medical Officer, Fulcrum Therapeutics
“Time is precious for patients and families affected by rare diseases. Fulcrum is proud to have launched the first Phase 3 clinical trial for facioscapulohumeral muscular dystrophy (FSHD) – a devastating, progressive neuromuscular disease for which there are no approved treatments – less than three years after becoming a public company. The incentives provided by the Orphan Drug Tax Credit are essential for enabling young companies like ours to rapidly research and develop promising new therapies for the communities that are waiting.”
Nevan Charles Elam, JD, Chief Executive Officer & Founder, Rezolute Bio
“When you look beyond rare diseases that are currently being treated or have the potential for new therapies, you see millions of people who now have hope thanks to the ODA. While the disease may be rare, the people are not, and without the ODA, the incentive to research and develop new therapies for patients in need would simply not be there.”
Jean-Jacques Bienaime, Chairman and Chief Executive Officer at BioMarin
“The Orphan Drug Act (ODA) makes it possible for us to invest in the research and development necessary to address the unmet needs of rare disease patients. Since the passage of the ODA, all of BioMarin’s FDA-approved therapies have received orphan designation. As such, BioMarin has been able to transform the lives of children across the globe who have rare diseases such as CLN2 (a form of Batten disease) and enzyme deficiency diseases, among others.” By protecting the ODA, we are protecting the future of innovation and sustaining hope for children and adults alike with rare diseases.”
Camille Bedrosian, Chief Medical Officer, Ultragenyx
“The incentive provided by the Orphan Drug Tax Credit allowed Ultragenyx to study Crysvita® for tumor-induced osteomalacia (TIO), an ultra-rare disease that most companies would never have studied, after Ultragenyx previously obtained initial FDA approval of Crysvita® for the treatment of X-linked hypophosphatemia (XLH). We support the continued existence of the Orphan Drug Tax Credit as a critical incentive for rare disease companies.”
Del Lebel, Head, U.S. Government Affairs and Policy, Alexion, AstraZeneca Rare Disease
“The Orphan Drug Act spurred tremendous progress in the development of rare diseases treatments not only in the United States, but worldwide. The law has served as a catalyst for rare disease innovation and regulatory approval pathways in many other countries. Now is the time to build on that progress and promote sound policies that will improve patients’ lives.”
Ernesto Aycardi, Chief Development Officer and Head of Global Drug Development, Kyowa Kirin
“Kyowa Kirin is focused on advancing science and providing medicines for patients with unmet needs, and we consider Orphan Diseases an opportunity to accomplish our goals of serving the neediest. The ODA incentive programs have been crucial to our industry’s work – and we celebrate the profound impact they have had for patients living with rare diseases. Given the number of patients still in search of effective treatment options, we need collective action and appropriate programs to continue making a difference and foster innovation.”
Brian Goff, Chief Executive Officer at Agios Pharmaceuticals
On the 40th anniversary of the Orphan Drug Act, we celebrate the progress that has been made for hundreds of rare disease communities who previously had no treatment options. Since the legislation’s enactment in 1983, the number of FDA-approved medicines has grown from 38 to more than 650 – and that’s no coincidence. We also recognize that more progress is needed for the 95% of rare diseases that still lack treatment. Upholding and strengthening the provisions of the Orphan Drug Act fuel ongoing innovation on behalf of underserved communities, such as Agios’ clinical programs in rare blood disorders including pediatric PK deficiency, thalassemia and sickle cell disease.”
Jeffrey M. Dayno, M.D., Interim Chief Executive Officer, Harmony Biosciences
“The Orphan Drug Act catalyzed new hope and possibilities for the millions of people living with a rare disease who had not yet had their unmet medical needs addressed by an approved new treatment option. Thanks to the tireless efforts of patients and their advocates, we can now build upon 40 years of industry rare disease research, innovation, and therapeutic breakthroughs. Harmony Biosciences is privileged to continue this mission as we strive to bring hope to people living with rare neurological diseases – ensuring that patients remain at the heart of everything we do.”
Steven Pfanstiel, Chief Financial Officer, Marinus Pharmaceuticals
“As a company committed to the development of innovative treatments for patients with rare forms of epilepsy, the Orphan Drug Act (ODA) helped enable us to deliver a first-of-its-kind treatment option to the CDKL5 deficiency disorder community for an indication with no prior approved therapies. With four additional clinical programs underway or expected to begin in 2023 utilizing the same compound, the ODA is critical to ensuring we can continue to drive innovation for our underserved patient communities in need of new treatment options.”
Chris Morabito, M.D., Chief Medical Officer, Astria Therapeutics
“Astria’s mission to provide life-changing therapies for people affected by rare diseases is directly aligned with the goals of the Orphan Drug Act and the Orphan Drug Tax Credit (ODTC). The ODTC enables companies like ours to bring important medicines to patients more efficiently, and we are supportive of the preservation of this critical incentive for the drug development process.”
