Events2Join

The Food and Drug Administration's Orphan Drug Program


Gene Therapy – FDA Takes Steps Toward Clarifying Scope of ...

In addition to the seven years of exclusivity, products receiving an orphan drug designation are eligible for certain financial incentives, including tax ...

Tracing the rise of orphan drug designations over almost 40 years

The Orphan Drug Act was established in 1983 to promote the development of drugs for rare diseases with limited or no available treatment options ...

Orphan Drugs WG | Pharmaceuticals and Medical Devices Agency

Based on ToR, exchanged opinions and strengthened mutual understanding concerning orphan drug designation systems. ... FDA: US Food and Drug Administration ...

ReAlta Granted FDA Orphan Drug Designation and Fast Track ...

The FDA's Orphan Drug Designation is granted to investigational therapies intended for the treatment of rare diseases or conditions that affect ...

Four Considerations to Shape Your Orphan Drug Designation ...

Orphan drug designation (ODD) provides several compelling incentives and benefits for pharmaceutical organizations developing drugs to treat rare diseases.

Novartis receives US Food and Drug Administration (FDA) Orphan ...

An Orphan Drug Designation grants special status to a drug that treats a rare disease or condition, and provides companies certain benefits to encourage the ...

High-expenditure Medicare drugs often qualified for Orphan Drug ...

Over 7,000 rare diseases affect more than 30 million Americans. The Orphan Drug Act (ODA) provides financial incentives—such as tax ...

A2 Bio Receives FDA Orphan Drug Designation for Novel Cell ...

FDA Orphan Drug Designation incentivizes the development of innovative drugs and biologics for the safe and effective treatment of rare diseases ...

The Orphan Drug Act at 40: Legislative Triumph and the Challenges ...

Orphan drugs are those intended to treat rare diseases or conditions that affect fewer than 200,000 Americans or more than 200,000 patients, or for which “there ...

AND017 Receives FDA Orphan Drug Designation for Sickle Cell ...

On October 25, 2024, Kind Pharmaceutical announced receipt of FDA Orphan Drug Designation for AND017 in the treatment of sickle cell disease.

Grants and contracts for development of drugs for rare diseases and ...

Despite the urgent health need for these medicines, they came to be known as 'orphan drugs' because no companies would commercialize them. "(3) During the 1970s ...

Follow-On Indications for Orphan Drugs Related to the Inflation ...

We identified novel orphan drug approvals (2003 through 2022) from FDA's New Molecular Entity (NME) Drug and New Biologic Approvals database.

FDA grants orphan drug designation to indotecan for the treatment ...

Orphan drug designation is given to therapies that treat, prevent or diagnose rare diseases. Although this designation does not necessarily mean ...

Olezarsen receives Orphan Drug designation from U.S. FDA for ...

Under the FDA's Orphan Drug Act, orphan drug status provides incentives, including tax credits, grants and waiver of certain administrative fees for clinical ...

Tezepelumab granted Orphan Drug Designation in the US for ...

The FDA grants ODD status to medicines and potential new medicines intended for the treatment, diagnosis or prevention of rare diseases or ...

FDA, Rare Diseases, Orphan Drug Act - Bio.News

This program has not had increases in funding commensurate with inflation for many years. BIO urges increased funding for the Orphan Drug Grant Program. We also ...

Neurocrine Biosciences Receives Orphan Drug Designation for ...

(Nasdaq: NBIX) today announced that it has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for valbenazine as a treatment for ...

Panel on Financial Incentives FDA Orphan Drugs Program and ...

Roberta Szydlo from FDA's Office of Orphan Products Development and Peter Chen from CDER's Division of User Fee Management & Budget ...

GAO To Launch Investigation Of FDA's Orphan Drug Program

The Government Accountability Office said it will investigate potential abuses of the orphan drug program, which offers incentives to ...

Orphan Drug Credit: What It Is, How It Works - Investopedia

The orphan drug credit is a federal tax credit that gives pharmaceutical companies incentives to develop medications and treatments for rare diseases that ...