Group Calls for Support as FDA Orphan Drug Act Turns 40

AADC deficiency, other rare diseases, still need treatments, companies say

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by Mary Chapman |

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This illustration shows the U.S. Capitol building, seat of the legislative branch of the country's federal government.

In marking the 40th year of the Orphan Drug Act (ODA) — used by the U.S. Food and Drug Administration (FDA) to spur treatment development for rare diseases — a life sciences industry coalition is calling on legislators and other policymakers to make a new commitment to finding therapies for rare disorders.

The ODA was passed in 1983 to incentivize investment in research and development for rare disorders, such as aromatic l-amino acid decarboxylase (AADC) deficiency, which had been significantly lacking. Since that enactment, the prospect of an FDA orphan drug designation — with its tax credits and marketing exclusivity for approved new treatments for rare diseases — has prompted biopharmaceutical companies to pursue therapies for small patient populations that would otherwise be too risky financially.

Now, the Rare Disease Company Coalition (RDCC) is recognizing the ODA’s milestone anniversary by asking U.S. lawmakers and other stakeholders to create and support policies that further spur treatment investment and innovation.

The RDCC also is acknowledging advancements in therapies for people with rare diseases.

“Over the past 40 years, the ODA has established a strong foundation for the biotech community to invest in and develop new medicines for rare diseases,” Amanda Malakoff, executive director of the RDCC, said in a press release. “We need to build on that strong foundation so we can ensure continued innovation for the 95% of rare diseases that currently have no approved treatments.”

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Too few treatments despite FDA Orphan Drug Act

To date, the FDA Orphan Drug Act has resulted in an increase in the number of federally approved orphan drugs — medicines for rare diseases or conditions — from 38 to more than 600 for more than 1,000 rare disorders, according to the RDCC.

Still, of the approximately 7,000 known rare diseases thought to affect one in 10 Americans, the vast majority has no treatment. There is no approved therapy for AADC deficiency.

This underscores the need to protect and build upon ODA policies, the RDCC contends. The coalition was founded in 2021 to represent life science companies that seek to discover and develop rare disease therapies.

According to the RDCC, its members have more than 200 promising treatments in development pipelines.

“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,” said Nevan Charles Elam, JD, CEO and founder of Rezolute Bio, an RDCC member. “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.”

The coalition members contend that recent policy changes threaten ODA-established incentives such as the Orphan Drug Tax Credit. The RDCC also says new, all-inclusive policy proposals don’t fully address the subtleties of rare disease R&D.

To continue and safeguard progress driven by the Orphan Drug Act, the RDCC is calling on lawmakers to:

  • increase the Orphan Drug Tax Credit to its original 50% of eligible research and development expenses.
  • support exemptions in the Inflation Reduction Act for rare disease therapies and future treatment pricing policies so that future rare-disease investigations are not at risk.
  • incentivize biopharmaceutical companies to complete promising clinical studies that were put on hold due to the COVID-19 pandemic.
  • make permanent U.S. Food and Drug Administration’s priority review vouchers which are set to expire between 2024 and 2026 and consider other kinds of incentives that patterned after the vouchers.

“In recognizing the 40th anniversary of the ODA, it is more important than ever for policymakers, patients, patient advocacy groups, providers, researchers, and manufacturers to come together and reaffirm their commitment to the rare disease community by showing steadfast support for upholding and strengthening the ODA,” Malakoff said.