News & Analysis as of

Food and Drug Administration (FDA) Orphan Drugs Life Sciences

Knobbe Martens

Rare Disease Therapies Gain Momentum With Regulatory Support and Continued Market Growth

Knobbe Martens on

The landscape for rare disease treatments appears to be evolving rapidly, thanks to a confluence of regulatory momentum and a booming market forecast. Statements from key federal figures in recent months combined with market...more

Brownstein Hyatt Farber Schreck

FDA Nominee Makary on the Orphan Drug Act

President-elect Trump’s nominee for commissioner of Food and Drugs, Martin A. Makary, MD, MPH, argued in 2016 that Congress should reform the Orphan Drug Act (ODA) because drug companies “are gaming the system to use the law...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

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

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

Hogan Lovells on

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 - The Patent Playbook

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

MoFo Life Sciences

Rare Disease: Sustained Progress In Development Regardless Of The Pandemic

MoFo Life Sciences on

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

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

Sterne, Kessler, Goldstein & Fox P.L.L.C.

FDA Issues Biosimilar Guidance for Label Carve-Outs and Supplements

On February 6, 2020, the U.S. Food and Drug Administration (“FDA”) issued a draft guidance (“Guidance”) that addresses the licensure of a biosimilar or biosimilar interchangeable product for fewer than all of the conditions...more

Hogan Lovells

FDA’s Bendeka decision reverses approvals of Treanda generics

Hogan Lovells on

Wednesday, FDA released a letter stating how the agency intends to implement the scope of orphan drug exclusivity awarded to Eagle Pharmaceuticals’ Bendeka (bendamustine HCl) by the D.C. Circuit Court in Eagle...more

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