News & Analysis as of

Food and Drug Administration (FDA) Orphan Drugs

Holland & Hart LLP

Alternative Means for FDA Approval of Pet Care Drugs – Part 3: Differences Between Major and Minor Species

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This update is the final in a multi-part series exploring FDA approval of pet care drugs. In part one of our series we discussed that before an animal drug product can be legally marketed for use in animals, a New Animal Drug...more

MoFo Life Sciences

Medicare Drug Price Negotiation Program Final Guidance

MoFo Life Sciences on

On June 30, 2023, the Centers for Medicare and Medicaid Services (CMS) released its much-anticipated final guidance (“Final Guidance”) for the Medicare Drug Price Negotiation Program (“Negotiation Program”) through the...more

Arnall Golden Gregory LLP

The Sun Will Come Up Tomorrow for Another Company: An Interesting Orphan Drug Exclusivity Decision

Last month, the Food and Drug Administration’s Office of Orphan Products Development (“OOPD”) found that one prescription drug company was able to, essentially, breach another’s orphan drug exclusivity (and have its new drug...more

Morgan Lewis - As Prescribed

When Words of Encouragement May Indicate More: Congress Focuses on Rare Disease Approval Process

While many provisions of the FY 2023 Consolidated Appropriations Act (Omnibus) have received much attention, one has flown under the radar. In its explanatory statement on the Omnibus, the US Congress indicated that it is...more

Arnall Golden Gregory LLP

The Sun Will Come Up Tomorrow (or Will It)? FDA Issues Notification About Orphan Drug Exclusivity in Light of Recent Court...

Recently, the Food and Drug Administration published a Notification to clarify its policy about orphan drug exclusivity. The agency issued the announcement because the U.S. Court of Appeals for the 11th Circuit found that FDA...more

Epstein Becker & Green

FDA Issues Orphan Drug Exclusivity Policy That Could be a Catalyst for Future Litigation

Epstein Becker & Green on

On January 24, 2023, FDA published a notice in the Federal Register entitled, “Clarification of Orphan-Drug Exclusivity Following Catalyst Pharms., Inc. v. Becerra.” In brief, the Catalyst decision by the 11th Circuit Court...more

Manatt, Phelps & Phillips, LLP

FDA Stands By Long-standing Interpretation of the Orphan Drug Act…For Now

On January 24, the Food and Drug Administration (FDA) made a long-awaited announcement with major implications for how the agency awards exclusivity for rare disease drugs under the Orphan Drug Act (ODA) (P.L. 97-414)....more

Cooley LLP

FDA Doubles Down on Its Pre-Catalyst Stance on Orphan Drug Exclusivity

Cooley LLP on

On January 24, 2023, the Food and Drug Administration published a notice in the Federal Register to “address the uncertainty” created by the US Court of Appeals for the Eleventh Circuit’s September 30, 2021, decision in...more

King & Spalding

Not Quite the Titanic: The Food and Drug Omnibus Reform Act Rescues Some FDA Policy Initiatives

King & Spalding on

The Food and Drug Administration (“FDA” or the “Agency”) user fee negotiations may have hit an iceberg, but it did not sink all the legislative riders that accompanied this summer’s House and Senate bills. Buried within the...more

Manatt, Phelps & Phillips, LLP

A Focal Point for FDA’s Rare Diseases Efforts: CDER’s New ARC Program

Last week, the Center for Drug Evaluation and Research (CDER) at the Food and Drug Administration (FDA) launched a new program to coordinate and centralize CDER’s rare disease activities. The mission of the Accelerating Rare...more

Goodwin

FDA Accepts Regeneron and Sanofi’s DUPIXENT (Dupilumab) sBLA for Priority Review

Goodwin on

Last week, Regeneron Pharmaceuticals, Inc. and Sanofi announced that the FDA has accepted a Priority Review of the supplemental Biologics License Application (sBLA) for DUPIXENT (dupilumab) 300 mg weekly to treat patients...more

Haug Partners LLP

A Tale of Two Orphans: The Potential Ramifications of an Orphan Drug “Cutting in Line”

Haug Partners LLP on

Orphan drugs are the pharmaceutical industry’s way of helping those who suffer from rare conditions. Thanks to the Orphan Drug Act, such patients can get the medications they need, and pharmaceutical companies can be duly...more

Hogan Lovells

Eleventh Circuit decision could significantly expand scope of orphan exclusivity

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With the issuance of its mandate on January 28, 2022, in Catalyst Pharmaceuticals, Inc. v. Becerra, the U.S. Court of Appeals for the Eleventh Circuit has upheld the orphan exclusivity for Catalyst Pharmaceuticals, Inc....more

MoFo Life Sciences

FDA Finalizes Guidance On Interpretation Of Orphan Drug “Sameness” For Gene Therapy Products

MoFo Life Sciences on

On September 30, 2021, the U.S. Food and Drug Administration (FDA) announced final guidance titled “Interpreting Sameness of Gene Therapy Products under the Orphan Drug Regulations.” The guidance finalizes the January 2020...more

Morgan Lewis - As Prescribed

FDA Finalizes Guidance on Gene Therapy Orphan Sameness, but Questions Remain

While it may feel like FDA’s attention has been focused on COVID-19 for nearly two years, as we have written about in a previous blog post, FDA is looking to the future, and particularly, one that signals exciting prospects...more

Proskauer - Life Sciences

In the Orphan Drug Approval Race, Winner Takes All? Ramifications of Catalyst Pharms. v. Becerra

How is orphan drug exclusivity affected when the FDA-approved use for an orphan drug is arguably narrower than the treatment of the rare disease it was designated for? By way of background, a sponsor can obtain orphan drug...more

Hogan Lovells

FDA finalizes guidance on Orphan Drug “sameness” of gene therapies

Hogan Lovells on

Recently, the U.S. Food and Drug Administration’s (FDA’s) Office of Orphan Product Development published the four-page final guidance “Interpreting Sameness of Gene Therapy Products Under the Orphan Drug Regulations,” which...more

McGuireWoods Consulting

Washington Healthcare Update - May 2021 #4

This week in Washington: Talks continue on infrastructure, Senate committees moving forward on health legislation, President’s budget due the end of the week....more

Locke Lord LLP

Ensuring Innovation Act Alters Eligibility for New Chemical Exclusivity

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On April 23, 2021, President Biden signed into law The Ensuring Innovation Act (S. 415), which amends the requirements for New Chemical Entity (NCE) and Orphan Drug (ODE) exclusivities under the Federal Food, Drug, and...more

MoFo Life Sciences

Rare Disease: Sustained Progress In Development Regardless Of The Pandemic

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Sunday, February 28, 2021, was Rare Disease Day. With so much focus on COVID-19 throughout 2020, it’s important to recognize the continued work done in rare disease drug development by sponsors and FDA throughout 2020. In...more

Kilpatrick

Orphan Drug Act Reform: Will the Loophole be Closed During the Biden Administration?

Kilpatrick on

The U.S. House of Representatives generated buzz in November 2020 when it passed a bill aimed at removing a loophole in the Orphan Drug Act. Although the bill did not make it far in the Senate, the bipartisan legislative push...more

Goodwin

Congress Enacts Amendments Affecting The Regulation Of Generic Drugs And Biosimilars

Goodwin on

On December 27, 2020, the President signed into law the “Consolidated Appropriations Act, 2021” (the “Act”). Included within this omnibus legislation are several provisions (in Division BB, Title III, Subtitle C) that affect...more

Proskauer - Minding Your Business

H.R. 4712: A “Narrowly Tailored” Obligation Under the Orphan Drug Act?

The Orphan Drug Act provides two mechanisms by which a drug can receive an orphan drug designation for a “rare” disease: (1) if it affects less than 200,000 persons in the United States, or (2) if it “affects more than...more

Proskauer - Minding Your Business

Orphan Drug Exclusivity for CRISPR/Cas-Based Therapeutics

The prospect of genetic engineering using CRISPR (clustered regularly interspaced short palindromic repeats) and CRISPR-associated nucleases (Cas) has long been hailed as a “revolutionary” development in medicine....more

King & Spalding

EUROPE – Are Revisions of the Orphan Drug Regulation and Adoption of a Regulation on “Unmet Medical Needs” on the Horizon

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The European Commission (Commission) has released a Staff Working Document concerning its joint evaluation of the Orphan Regulation and the Paediatric Regulation (collectively, the Regulations) (Joint Evaluation) and an...more

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