The Food and Drug Administration's Orphan Drug Program

Sage Therapeutics Announces U.S. Food and Drug Administration ...

Orphan drug designation is granted by the FDA Office of Orphan Products Development to assist and encourage companies to develop safe and ...

Corvus Pharmaceuticals Announces Orphan Drug Designation ...

FDA Orphan Drug Designation is granted to investigational therapies addressing rare medical diseases or conditions that affect fewer than ...

Shattuck Labs Announces Orphan Drug Designation Granted by the ...

The FDA's Office of Orphan Products Development grants orphan status to drugs being developed to treat, diagnose, or prevent a rare disease or ...

FDA's orphan drug program under GAO scrutiny after senators ...

Just weeks after senators raised questions about the FDA's orphan drug program—and after Marathon Pharmaceuticals caught fire for an $89000 ...

Canada's regulatory approach to drugs for rare diseases: orphan ...

effective treatment, prevention or diagnosis of a disease or condition for which no drug is presently marketed in Canada or; a significant ...

Terns Pharmaceuticals Announces Orphan Drug Designation ...

FDA Orphan Drug Designation is granted to investigational therapies addressing rare medical diseases or conditions that affect fewer than ...

Agios Announces FDA Orphan Drug Designation Granted to ...

Agios Announces FDA Orphan Drug Designation Granted to Mitapivat for Treatment of Sickle Cell Disease ... CAMBRIDGE, Mass. , Nov. 12, 2020 (GLOBE ...

The Orphan Drug Act: Legal Overview and Policy Considerations

second drug is clinically superior to the first. populations. ... for the same disease or condition. ... orphan drug field. ... unless the second drug ...

Ionis announces eplontersen receives orphan drug designation from ...

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

FDA Grants Orphan Drug Designation to Avutometinib in Ovarian ...

The orphan drug designation is granted to agents that prevent, diagnose, or treat a rare disease. Drug sponsors are eligible for incentives ...

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

Mirroring the draft guidance published in January 2020, the final guidance affirms FDA's position that two gene therapy products that express ...

How FDA's orphan drug program leads to high prescription drug prices

When a drugmaker wins approval for an orphan drug, the company is entitled to seven years of exclusive rights to the marketplace, which means ...

C4 Therapeutics Announces FDA Orphan Drug Designation for ...

“We are pleased to receive FDA's orphan drug designation for CFT7455 in multiple myeloma and believe this designation highlights the potential ...

Orphan Drugs Archives - KFF Health News

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

Apellis Pharmaceuticals' APL-2 Receives Orphan Drug Designation ...

The FDA Orphan Drug program provides orphan designation to novel drugs that are intended for the treatment of rare diseases (those affecting ...

Government investigation finds flaws in the FDA's orphan drug ...

The FDA's rare-disease program began after Congress overwhelmingly passed the 1983 Orphan Drug Act to motivate pharmaceutical companies to ...

Orphan designation: Overview | European Medicines Agency (EMA)

The European Medicines Agency (EMA) plays a central role in facilitating the development and authorisation of medicines for rare diseases, which are termed ' ...

FDA's Office of Orphan Products Development Grants Programs

Patients Matter: FDA's Office of Orphan Products Development Grants Programs · U.S. Food and Drug Administration · How FDA Investigates Foodborne ...

340B Drug Pricing Program Orphan Drugs Program Requirements

It is based upon specific fields captured from the list of orphan drug designations provided by the U.S. Food and Drug Administration (FDA) ...

Sapience Therapeutics Receives FDA Orphan Drug Designation for ...

Orphan Drug Designation is granted to drugs or biological products for the treatment of rare diseases or conditions that impact fewer than ...