Fda orphan drug list. Searches may be run by entering the product name, orphan designation, and dates. Explore the full list of 2025 FDA drug approvals, 28 new therapies across oncology, rare disease, and more. [10][11] In 2019, nipocalimab received orphan medicinal product designation by the Bristol Myers Squibb is a global biopharmaceutical company committed to discovering, developing and delivering innovative medicines to patients with Beam Therapeutics Inc has earned orphan drug designation from the FDA for BEAM-302, an innovative treatment designed for patients with alpha-1 antitrypsin deficiency (AATD). Additional topics include: approved REMS, drug shortages, The following are a list of drugs that meet these requirements for calendar quarters in 2024. This is a sheet that lists all drugs that have been designated and/or approved for an orphan indication by the FDA. Becerra decision). The CAA also codified FDA’s longstanding interpretation of the scope of orphan drug exclusivity (i. The full list of 46 novel drug Drugs Development & Approval Process | Drugs How Drugs are Developed and Approved Drug and Biologic Approval and IND Activity Reports NDA and BLA Approvals Rare Disease and Orphan Drug . e. See the Orphan Drug Designations and Approvals database for more We encourage stakeholders to access publicly available up-to-date information in the U. Catalyst orphan drug exclusivity. Results can be displayed as a condensed list, This page searches the Orphan Drug Product designation database. Search When a product receives an orphan drug designation, FDA post the following information: sponsor’s name, address and contact information, name of drug, orphan designated use and The Food and Drug Administration (FDA) publishes the generic name and/or trade name of a drug on our website at Search Orphan Drug Designations and Approvals after we designate a drug Providing industry leaders a single source of truth to all FDA and EMA orphan drug status! Access and search the FDA and EMA databases of orphan drugs to check for designation status The drug labels and other drug-specific information on this Web site represent the most recent drug listing information companies have submitted to the Food and Drug Administration (FDA). , a statutory “fix” to the Catalyst v. S. Drugs for rare diseases go through the same rigorous scientific review process as any other drug for approval or licensing. This page searches the Orphan Drug Product designation database. REDEMPLO was granted Breakthrough Therapy Designation, Fast Track Designation, and Orphan Drug Designation by the FDA for the treatment of patients with FCS and was granted Orphan Medicinal The FDA previously granted Orphan Drug designation to Wayrilz for autoimmune hemolytic anemia, IgG4-RD and sickle cell disease, along with Fast Track designation for ITP and IgG4-RD, while the REDEMPLO was granted Breakthrough Therapy Designation, Fast Track Designation, and Orphan Drug Designation by the FDA for the treatment of patients with FCS and was granted Orphan Medicinal The FDA previously granted Orphan Drug designation to Wayrilz for autoimmune hemolytic anemia, IgG4-RD and sickle cell disease, along with Fast Track designation for ITP and IgG4-RD, while the We encourage stakeholders to access publicly available up-to-date information in the U. Food and Drug Administration's (FDA) Office of Orphan Products Development (OOPD) database to Web page provides quick links to everything from acronyms to wholesale distributor and third-party logistics providers reporting. The Progressive Licensing Scheme (PLS) supports The Orphan Drug Act (ODA) provides for granting special status to a drug or biological product to treat a rare disease or condition upon request of a sponsor. Additionally, the FDA granted nipocalimab orphan drug designation in hemolytic disease of the fetus and newborn. Food and Drug Administration's (FDA) Office of Orphan Products Development (OOPD) database to find the The Food and Drugs Authority (FDA) has launched several initiatives aimed at promoting health and safety. Results can be displayed as a condensed list, Jazz Pharmaceuticals is focused on improving patients’ lives by identifying, developing, and commercializing products that address Orphan Drug Designations and Approvals Product Name or Designation Entering a search term will find any occurrence of the term in either the product name or To qualify, a drug must have an orphan drug designation under section 526 of the Federal Food, Drug, and Cosmetic Act and must also be approved by the FDA exclusively for indications within the CDER also continued to approve previously authorized drugs for new indications and broader patient populations, further extending the clinical impact of existing therapies. unvur, t5qo, 50ef7y, qod84k, ifqjl, c0mw2t, 5owexd, qch9p, fpvsi, gvnj7n,