Designating an Orphan Product: Drugs and Biological Products
Aug 12, 2024 · The FDA has authority to grant orphan drug designation to a drug or biological product to prevent, diagnose or treat a rare disease or condition.
Search Orphan Drug Designations and Approvals - Food and Drug …
This page searches the Orphan Drug Product designation database. Searches may be run by entering the product name, orphan designation, and dates. Results can be displayed as a condensed...
Frequently Asked Questions (FAQ) About Designating an Orphan …
In order to designate a product as an orphan drug, the scientific rationale portion of the designation application must include enough information to establish a medically plausible basis for...
Orphan Drug Designation: Disease Considerations | FDA
For a complete list of orphan drug designations and approvals see the searchable Orphan Products Designation Database. FDA considers ovarian cancer, fallopian tube cancer, and primary...
Orphan drug - Wikipedia
According to the US Food and Drug Administration (FDA), an orphan drug is defined as one "intended for the treatment, prevention or diagnosis of a rare disease or condition, which is one that affects less than 200,000 persons in the US" (which equates to approximately 6 cases per 10,000 population) "or meets cost recovery provisions of the act"....
eCFR :: 21 CFR Part 316 -- Orphan Drugs
(10) Orphan drug means a drug intended for use in a rare disease or condition as defined in section 526 of the act. (11) Orphan-drug designation means FDA's act of granting a request for designation under section 526 of the act.
Where is OOPD within FDA? The mission of OOPD is to advance the evaluation and development of products that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions. OOPD programs include 3 designation programs and 3 grants programs.
Orphanet : Search for an orphan drug
Orphan designation is a legal procedure that allows for the designation of a medicinal substance with therapeutic potential for a rare disease, before its first administration in humans or during its clinical development.
Orphan designation - European Medicines Agency (EMA)
To qualify for orphan designation, a medicine must meet a number of criteria: no satisfactory method of diagnosis, prevention or treatment of the condition concerned can be authorised, or, if such a method exists, the medicine must be of significant …
What is an orphan drug? - Johnson & Johnson
Feb 28, 2024 · Under the ODA, a company can request orphan drug designation from the FDA. If granted, the company qualifies for research grants, tax credits for qualified clinical trials and potential seven-year market exclusivity after the drug’s approval, among other benefits.
Special FDA designations for drug development: orphan, fast …
Over the past decades, US Congress enabled the US Food and Drug Administration (FDA) to facilitate and expedite drug development for serious conditions filling unmet medical needs with five special designations and review pathways: orphan, fast track, accelerated approval, priority review, and breakthrough therapy.
Special FDA designations for drug development: orphan, fast …
Background: Over the past decades, US Congress enabled the US Food and Drug Administration (FDA) to facilitate and expedite drug development for serious conditions filling unmet medical needs with five special designations and review pathways: orphan, fast track, accelerated approval, priority review, and breakthrough therapy.
Drugs and biologics receiving FDA orphan drug designation: an …
We found 5,099 orphan drug designations representing 3,269 unique products, of which 508 had an orphan-designated approval from FDA. Unique products with only a single designation represented 2,448 (75%) of the total products and 26 (1%) products had 10 or more designations.
Instructions for Searchable Designation Database | FDA
Searches can return all designations or be restricted to products that are FDA-approved for the Orphan Designation. The default is all designations.
What is an Orphan Drug? A Guide to Rare Disease Therapies
1 day ago · Since the act was introduced, over 880 orphan drugs have been approved for use by the FDA. 4 Similarly, in the EU, over 200 orphan drugs have been authorized due to legislation.
Rigel Announces R289 Granted Orphan Drug Designation by the …
Jan 9, 2025 · This press release contains forward-looking statements relating to, among other things, the possible advantages of Orphan Drug and Fast Track designations, the potential benefits of R289 as a therapeutic for MDS and LR-MDS, the existence of patients with an unmet medical need for such therapy, certain potential benefits associated with orphan ...
21 CFR Part 316 Subpart C -- Designation of an Orphan Drug
More than one sponsor may receive orphan-drug designation of the same drug for the same rare disease or condition, but each sponsor seeking orphan-drug designation must file a complete request for designation as provided in paragraph (b) of this section.
FDA Grants Orphan Drug Designation to RAG-21 for Treatment …
Nov 22, 2024 · The FDA granted an orphan drug designation to RAG-21 (Ractigen Therapeutics) for the treatment of amyotrophic lateral sclerosis (ALS). Preclinical data showcases RAG-21’s potential to mitigate motor neuron degeneration and improve disease outcomes for patients. 1
FDA Grants Orphan Drug Designation for CAN-3110 for the …
May 30, 2024 · Orphan Drug Designation is granted by the FDA to drugs or biologics intended to treat a rare disease or condition, defined as one that affects fewer than 200,000 people in the United States. Orphan Drug Designation provides certain financial incentives to support clinical development, and the potential for up to seven years of marketing ...
Zai Lab Receives Orphan Drug Designation from the U.S. FDA for …
8 hours ago · "Receiving an Orphan Drug Designation for ZL-1310 recognizes its potential to treat patients with SCLC. These patients have an urgent need for innovative treatment options with improved efficacy ...
ALS treatment: Zydus receives USFDA Orphan Drug Designation …
22 hours ago · The USFDA has earlier granted Zydus an ‘Orphan Drug Designation’ for Usnoflast to treat patients with Cryopyrin Associated Periodic Syndrome (CAPS), a rare auto-inflammatory disease. Zydus has previously completed a Phase 2(a) randomized, double-blind, placebo controlled clinical trial in 24 ALS patients across 7 clinical trial sites in India.
Rare Diseases at FDA | FDA - U.S. Food and Drug Administration
Nov 21, 2024 · What is the Orphan Drug Act? The Orphan Drug Act is a law passed by Congress in 1983 that incentivizes the development of drugs to treat rare diseases. Companies and other drug...
Orphan Drug Designations in the US and EU - ProPharma Group
Jul 8, 2022 · What is an Orphan Drug Designation? The Orphan Drug Designation (ODD) program in both the United States (U.S.) and European Union (E.U.) qualifies sponsors to receive potential incentives to develop therapies for the diagnosis, prevention, or treatment of rare diseases or conditions.
Orphan designation | European Medicines Agency (EMA)
A status assigned to a medicine intended for use against a rare condition. The medicine must fulfil certain criteria for designation as an orphan medicine so that it can benefit from incentives such as protection from competition once on the market. More information can be found under 'Orphan designation: Overview'.
• Orphan drug: – Drugs (includes biologics) for the prevention, diagnosis, or treatment of diseases or conditions affecting fewer than 200,000 persons in the US
Zai Lab Receives Orphan Drug Designation from the U.S. FDA for …
8 hours ago · “Receiving an Orphan Drug Designation for ZL-1310 recognizes its potential to treat patients with SCLC. These patients have an urgent need for innovative treatment options with improved efficacy, safety and ready access in tertiary care and community settings,” said Rafael G. Amado, M.D., President, Head of Global Research and Development ...