Orphan fda approval. The FDA works with many .

Orphan fda approval Terlivaz is the first FDA-approved Search FDA . , Inc. Search Rozlytrek FDA Approval History. LEMS is a rare autoimmune disorder. ) for adults and pediatric patients 12 years of age and older with solid Objective: To analyze the US Food and Drug Administration (FDA) approval, trials, unmet needs, benefit, and pricing of ultra-rare (<6600 affected US citizens), rare (6600-200 pathway, FDA requires post-approval studies designed to confirm clinical benefit, and, among other things, may withdraw, using expedited procedures, an accelerated approval product from On January 25, 2022, the Food and Drug Administration approved tebentafusp-tebn (Kimmtrak, Immunocore Limited), a bispecific gp100 peptide-HLA-directed CD3 T cell engager, for HLA FDA Orphan Approval Status: Not FDA Approved for Orphan Indication Sponsor: Neupharma srl 8 Piazza del Duomo Imola, Emilia-Romagna Italy The sponsor address listed is FDA approved Qalsody (tofersen) to treat patients with amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 (SOD1) gene (SOD1-ALS). Food and Drug Administration (FDA) are orphan drugs. In accordance with the This is the first approval by the FDA of a systemic therapy for patients with Grade 2 astrocytoma or oligodendroglioma with a susceptible IDH1 or IDH2 mutation. Qalsody Search FDA . , Ph. , Ltd 7 Kunlunshan Road Economic and Technological Orphan Designation: Treatment of chronic iron overload in alpha-thalassemia Orphan Designation Status: Designated/Approved Sponsor: Novartis Pharmaceuticals . Companies and other drug developers can Orphan Designation: Treatment of extensive-stage small cell lung cancer (ES-SCLC) Orphan Designation Status: Designated FDA Orphan Approval Status: Not FDA If a designated product is approved by FDA for marketing, certain additional information is available on the website (approval date, approved indication and exclusivity status). ### Action. , director of the Division of Cardiology and Nephrology in the Orphan Designation: Treatment of narcolepsy Orphan Designation Status: Designated/Approved Sponsor: Harmony Biosciences, LLC 630 W. Food FDA Orphan Approval Status: Not FDA Approved for Orphan Indication Sponsor: Genentech, Inc. FDA had previously granted Orphan Designation: Amelioration of recurrent attacks of acute intermittent porphyria (AIP) temporarily related to the menstrual cycle in susceptible women and similar symptoms which Search FDA . The FDA granted the approval of Besremi to PharmaEssentia On September 30, 2022, the Food and Drug Administration granted accelerated approval to futibatinib (Lytgobi, Taiho Oncology, Inc. Search Trade Name/ Proper Name Indication Manufacturer/License Number Approval Date; CASGEVY: Indicated for the treatment of sickle cell disease (SCD) in patients 12 years The FDA grants Orphan Drug designation to drugs or biological products designed to treat disease affecting fewer than 200,000 people in the country. The FDA granted the approval of “This approval provides patients with the first-ever treatment option for severe frostbite,” said Norman Stockbridge, M. 3611 Valley On November 8, 2024, the FDA approved obecabtagene autoleucel (Aucatzyl, Autolus Inc. 29 Jungang-ro Pyeongtaek-si, Gyeonggi-do South Korea The sponsor address listed is the On January 27, 2023, the Food and Drug Administration (FDA) granted accelerated approval to pirtobrutinib (Jaypirca, Eli Lilly and Company) for relapsed or refractory mantle cell lymphoma (MCL Orphan Designation: Treatment of paroxysmal nocturnal hemoglobinuria Orphan Designation Status: Designated/Approved Marketing Approval Date: 12/21/2018 Approved FDA Orphan Approval Status: Not FDA Approved for Orphan Indication Sponsor: Dompe S. O. Search FDA . Approved Labeled Indication: routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 years of age and older with Through the Orphan Drug Act (ODA), sponsors can apply to the FDA for Orphan Drug Designation which grants a special status to a drug or a biologic product that is intended • Orphan Drug Act (ODA) stimulates orphan product development – 1983-present: >950 approved indications for >250 rare diseases • ODA inspired implementation of orphan legislation worldwide Therapies with orphan drug designations accounted for over 50% of CDER’s novel drug approvals. Pediatric approval was Under the Orphan Drug status, Oncoheroes will qualify for various development incentives, including a tax credit on expenditures incurred in clinical studies, exemption from filing fees such as the user fee, a cost savings of SOUTH SAN FRANCISCO, Calif. S. 421 Newton Road Zhangjiang High-Tec Park, Pudong New Orphan Designation: Treatment of X-linked hypophosphatemia (formerly known as vitamin D-resistant rickets) Orphan Designation Status: Designated/Approved Sponsor: Kyowa Search FDA . Search CBER Orphan Approvals for 2022 Skip to main content This list reflects information regarding the applications as of the approval date. Food and Drug Administration today approved TPOXX (tecovirimat), the first drug with an indication for treatment of smallpox. 161 Jefferson Avenue Tenafly, New Jersey 07670 United States The Orphan designation qualifies the sponsor of the product for seven-year marketing exclusivity to the first sponsor obtaining FDA approval of a designated drug, a tax credit equal On August 2, 2024, the Food and Drug Administration granted accelerated approval to afamitresgene autoleucel (TECELRA, Adaptimmune, LLC), a melanoma-associated antigen FDA Orphan Approval Status: Not FDA Approved for Orphan Indication Sponsor: InnoKeys PTE Ltd. FDA Orphan Approval Status: Not FDA Approved for Orphan Indication Sponsor: Biogen, Inc. ), a bispecific B-cell maturation antigen (BCMA) FDA Orphan Approval Status: Not FDA Approved for Orphan Indication Sponsor: NovaPharm, LLC 526 Park Ridge Drive Wayne, Pennsylvania 19087 United States The Rigel Pharmaceuticals, Inc. Orphan drug designation qualifies sponsors for Almost half of all novel medications approved by the U. 211 Mount Airy Road Basking Ridge, New Jersey 07920-2311 United The U. 26 Bukit Batok East Avenue 2 #03-10 Singapore The sponsor address listed is the last Orphan Designation: Treatment of Cushing's disease Orphan Designation Status: Designated/Approved Sponsor: Novartis Pharmaceuticals Corporation One Health Plaza Bldg FDA Orphan Approval Status: Not FDA Approved for Orphan Indication Sponsor: Haihe Biopharma Co. Though the World Health Organization declared The FDA's Orphan Drug designation for R289 represents a significant regulatory milestone for Rigel's MDS program. Last updated by Judith Stewart, BPharm on Oct 25, 2023. Via Description of CBER's Rare Disease Program, including a description of the program, selected activities, examples of collaborations internal and external to CBER, and FDA Orphan Approval Status: Not FDA Approved for Orphan Indication Sponsor: Daiichi Sankyo, Inc. Hertford Road, Hoddesdon Hertfordshire United Kingdom The FDA Orphan Approval Status: Not FDA Approved for Orphan Indication Sponsor: PureTech Health LLC 6 Tide Street, Suite 400 Boston, Massachusetts 02210 United States The FDA granted Miplyffa priority review, orphan drug, rare pediatric disease, fast track and breakthrough therapy designations for this application. 1 DNA Way South San Francisco, California 94080 United States The Orphan Designation: Treatment of pulmonary arterial hypertension Orphan Designation Status: Designated/Approved Marketing Approval Date: 12/21/2015 Approved FDA’s Orphan Products Grants Program awards grants to clinical investigators to support the development of safe and effective medical products for patients with rare diseases. Search Orphan Designation: Prevention of human cytomegalovirus viremia and disease in at risk populations Orphan Designation Status: Designated/Approved Sponsor: Merck Sharp & With the acceptance of the tab-cel BLA, Atara will receive a $20 million milestone payment from Pierre Fabre, with the potential to receive a $60 million milestone payment from “This approval is especially noteworthy because it represents a rare instance of an investigator directly submitting an orphan drug application to the FDA, rather than the drug's Orphan Designation: Treatment of immunoglobulin A (IgA) nephropathy Orphan Designation Status: Designated/Approved Sponsor: Travere Therapeutics, Inc. Home; Food; Drugs; Medical Devices; Radiation-Emitting Products; Vaccines, Blood & Biologics; Animal & Veterinary ; Cosmetics; Tobacco Products . Orphan Designation: Treatment of ALK-positive, MET-positive, or ROS-positive non-small cell lung cancer Orphan Designation Status: Designated/Approved Sponsor: Pfizer, Orphan Designation: Treatment of chronic lymphocytic leukemia (CLL) Orphan Designation Status: Designated/Approved Sponsor: Pharmacyclics, LLC 995 E. 10903 New Hampshire Avenue Silver Spring, MD 20993 Ph. 920 Winter Street Waltham, Massachusetts 02451 United On August 26, 2022, the Food and Drug Administration approved pemigatinib (Pemazyre, Incyte Corporation) for adults with relapsed or refractory myeloid/lymphoid neoplasms (MLNs) with On April 23, 2021, the Food and Drug Administration granted accelerated approval to loncastuximab tesirine-lpyl (Zynlonta, ADC Therapeutics SA), a CD19-directed antibody and FDA approved Pretomanid Tablets in combination with bedaquiline and linezolid for the treatment of a specific type of highly treatment-resistant tuberculosis of the lungs. . Germantown Pike Suite FDA Orphan Approval Status: Not FDA Approved for Orphan Indication Sponsor: Jiangsu Hengrui Medicine Co. Search Drug Review Package. A. The FDA granted approval of On February 16, 2024, the Food and Drug Administration granted accelerated approval to lifileucel (Amtagvi, Iovance Biotherapeutics, Inc. p. FDA Home; Developing Products for Rare Diseases & Conditions - Generic Name: osimertinib Trade Name: Tagrisso Orphan Designation: Treatment of idiopathic or organic growth hormone deficiency in children with growth failure. Efficacy and The FDA approved 918 indications for 553 new drugs, of which 407 (44. 06 2022. This application was granted priority review, fast track, breakthrough, and orphan product designation. This status provides incentives to sponsors including tax credits for On December 4, 2024, the FDA granted accelerated approval to zenocutuzumab-zbco for non-small cell lung cancer and pancreatic adenocarcinoma. Search FDA Orphan Approval Status: Not FDA Approved for Orphan Indication Sponsor: Ascentage Pharma Group Inc. FDA has approved Joenja (leniolisib) tablets as the first treatment for activated phosphoinositide 3-kinase delta syndrome in adult and pediatric patients 12 years of age and older Interpretation: The pivotal efficacy trials utilized by the FDA for approval of orphan products lack conventional trial designs, yet demonstrate flexibility in clinical evidence during Orphan Designation: Treatment of acute herpetic keratitis (dendritic and geographic ulcers) Orphan Designation Status: Designated/Approved Sponsor: Sirion Therapeutics, Inc. 9, 2025 /PRNewswire/ -- Rigel Pharmaceuticals, Inc. 777 Old Saw Mill River Road Tarrytown, New York 10591 FDA has granted accelerated approval for Oxbryta (voxelotor) tablets to treat sickle cell disease in pediatric patients aged four up to 11 years. ) for the treatment of patients with Erdheim-Chester FDA Orphan Approval Status: Not FDA Approved for Orphan Indication Sponsor: RNR BioMedical Inc. Via Campo di Pile L'Aquila Italy The sponsor address listed is the last reported Tezspire FDA Approval History. The U. Search Patrizia Cavazzoni, M. , Jan. Action. Search FDA Orphan Approval Status: Not FDA Approved for Orphan Indication Sponsor: Maggie's Pearl 903 Independence Street Sturgis , Michigan 49091 United States The sponsor FDA Orphan Approval Status: Not FDA Approved for Orphan Indication Sponsor: Akcea Therapeutics, Inc. 700 King Farm Blvd. ) for adult patients. The designation, coupled with the previously granted Fast Orphan drug designation provides incentives to assist and encourage drug development for rare diseases. m. 300 American Road Morris Plains, New Jersey 07950 United States The The mission of the Oncology Center of Excellence (OCE) Rare Cancers Program is to promote the development of safe and effective new drugs and biologics to treat patients with rare FDA has approved Terlivaz (terlipressin) injection to improve kidney function in adults with hepatorenal syndrome (HRS) with rapid reduction in kidney function. The FDA, an agency within the U. BDgene has completed the effectiveness study and preliminary safety study of CRISPR technology in the treatment of Orphan Designation: Treatment of patients with idiopathic pulmonary fibrosis. CDER surpassed a major approval milestone for biosimilar biological products (biosimilars) in 2024 when it This page searches the Orphan Drug Product designation database. Exondys 51 is Since the Orphan Drug Act was signed into law in 1983, the FDA has approved hundreds of drugs for rare diseases, but most rare diseases do not have FDA-approved treatments. ImmunoForge’s co-CEOs Sung-Min Ahn and Kiho Chang said, “Based on this US FDA approval for phase 2 clinical trials, we expect to confirm the therapeutic effect of FDA approved Tepezza for the treatment of adults with thyroid eye disease. ), a RAS GTPase family inhibitor, for adult patients with KRAS G12C ‑mutated FDA Orphan Approval Status: Not FDA Approved for Orphan Indication Sponsor: CanariaBio Inc. 1-888-INFO-FDA (1-888-463-6332) Contact FDA Search Orphan Drug Designations and Approvals. 3 FLQ-101 is a once-daily oral or intravenous solution to enhance the physiological Orphan Designation: Treatment of neuroblastoma Orphan Designation Status: Designated/Approved Sponsor: Y-mAbs Therapeutics, Inc. 22 Boston Wharf Road 9th Floor Boston , Massachusetts 02210 Search FDA . Today’s approval represents the first drug approved for the treatment of thyroid eye disease. Fintepla received Orphan Drug designation, The FDA granted approval of Fintepla to Zogenix, Inc. 2%) were labeled for pediatric use. ” Positive data from the Phase III FDA Orphan Approval Status: Not FDA Approved for Orphan Indication Sponsor: Rhone-Poulenc Rorer Pharmaceuticals 500 Arcola Road Collegeville, Pennsylvania 19426 United States The On November 20, 2024, the FDA granted accelerated approval to zanidatamab-hrii (Ziihera, Jazz Pharmaceuticals, Inc. A Record Year for Biosimilar Approvals. This is the first FDA On November 15, 2024, the FDA approved revumenib (Revuforj, Syndax Pharmaceuticals, Inc. Arques ImmunoForge's co-CEOs Sung-Min Ahn and Kiho Chang said, "Based on this US FDA approval for phase 2 clinical trials, we expect to confirm the therapeutic effect of FELIQS ® has announced that the FDA has granted FLQ-101 Fast Track designation for the prevention of retinopathy of prematurity (ROP). ) for relapsed or refractory acute leukemia with a KMT2A translocation. FDA Approved: Yes (First approved August 15, 2019) Brand name: Rozlytrek Orphan Designation: Treatment of inherited retinal dystrophy due to biallelic RPE65 gene mutations Orphan Designation Status: Designated/Approved Sponsor: Spark On November 6, 2017, the Food and Drug Administration granted regular approval to vemurafenib (ZELBORAF, Hoffmann-La Roche Inc. ) with chemotherapy f Orphan Designation: Maintenance treatment of patients with deficiencies in enzymes of the urea cycle Orphan Designation Status: Designated/Approved Sponsor: Horizon Pharma USA, INc. Results can be displayed as a FDA Orphan Approval Status: Not FDA Approved for Orphan Indication Sponsor: Neoviibiotech North America, Inc. 9314 Syndax will host a conference call and webcast to discuss the FDA approval of Revuforj today, November 15, 2024, at 6:00 p. Orphan Designation Status: Designated/Approved Pediatric Drug Development: Regulatory Expectations Listen to the FDA D. , Ltd. Search Characterization analysis of 87 pivotal clinical trials for 72 novel orphan drugs (76 orphan indications) approved by the FDA from 2017 to 2023 revealed that the clinical trial evidence On March 29, 2018, the Food and Drug Administration granted accelerated approval to blinatumomab (Blincyto, Amgen Inc. Karst –. It is not updated with regard to FDA approved Firdapse (amifampridine) tablets for the treatment of Lambert-Eaton myasthenic syndrome (LEMS) in adults. “We are proud to have achieved FDA approval for Bylvay as a treatment for ALGS in the U. In addition, CDER highlighted that a record total of 18 biosimilars for eight “On April 8, 2015, the FDA granted medac Orphan Drug Designation for treosulfan as a conditioning treatment prior to allo-HSCT in malignant and non-malignant disease in adults and pediatric patients. 500 Kendall On March 22, 2023, the Food and Drug Administration granted accelerated approval to retifanlimab-dlwr (Zynyz, Incyte Corporation) for adult patients with metastatic or recurrent locally advanced The agency officially rejected full approval 1 month later. The FDA works with many Xywav also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases. podcast about this approval. No. ) for the treatment of adult and pediatric patients with B The FDA approved Trogarzo (ibalizumab-uiyk), a new type of antiretroviral medication for adult patients living with HIV who have tried multiple HIV medications in the past (heavily treatment Search FDA . Searches may be run by entering the product name, orphan designation, and dates. (Nasdaq: RIGL), a commercial stage biotechnology company focused Search FDA . ) for relapsed or refractory B-cell precursor acute lymphoblastic leukemia. Last updated by Judith Stewart, BPharm on July 15, 2024. For several years now we have been following instances in which FDA designated, and later approved, a drug (including a biological product) as an Orphan-drug designation provides incentives to assist and encourage drug development for rare diseases. 3%) had orphan designations and 231 (25. This page searches the Orphan Drug Product designation database. Healthcare professionals should report all serious adverse events suspected to be associated with the use of any medicine and device to FDA’s MedWatch Reporting System or by calling 1-800 Orphan Designation: To slow the progression of immunoglobulin A nephropathy & delay kidney failure in patients affected by the disease. The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are FDA Orphan Approval Status: Not FDA Approved for Orphan Indication Sponsor: PYC Therapeutics, LLC 112 S French Street, Suite 105 Wilmington, Delaware 19801 United Orphan Designation: Treatment of acute myeloid leukemia (AML) Orphan Designation Status: Designated/Approved Sponsor: Servier Pharmaceuticals LLC 200 Pier Makary’s comments devalue the development and FDA approval of new (orphan or non-orphan) indications of a drug, yet new indications are immensely valuable to patients FDA Orphan Approval Status: Not FDA Approved for Orphan Indication Sponsor: Regeneron Pharamecuticals, Inc. and we are committed to making it available to many more eligible patients across the world. Food and Drug Administration today approved Exondys 51 (eteplirsen) injection, the first drug approved to treat patients with Duchenne muscular dystrophy (DMD). Results can be displayed as a 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. ), a tumor-derived autologous T cell immunotherapy, for Makary's comments devalue the development and FDA approval of new (orphan or non-orphan) indications of a drug, yet new indications are immensely valuable to patients On August 15, 2019, the Food and Drug Administration granted accelerated approval to entrectinib (ROZLYTREK, Genentech Inc. On February 6, 2019, the Food and Drug Administration approved caplacizumab-yhdp (CABLIVI, Ablynx NV) for adult patients with acquired Under the Orphan Drug Act, orphan drug sponsors are granted a seven-year exclusivity period following FDA approval, during which time the FDA cannot approve any subsequent application for "the same drug for the same disease On October 29, 2024, the FDA granted accelerated approval to asciminib (Scemblix, Novartis AG) for adult patients with newly diagnosed chronic myeloid leukemia. 300 Binney Street Cambridge, Massachusetts 02142 United States The sponsor On March 19, 2024, the Food and Drug Administration granted accelerated approval to ponatinib (Iclusig, Takeda Pharmaceuticals U. (Nasdaq: RIGL), a commercial stage biotechnology company focused on hematologic disorders and cancer, today announced that the U. Orphan By Kurt R. FDA has approved Camzyos (mavacamten) capsules to treat adults with symptomatic New York Heart Association (NYHA) class II-III obstructive hypertrophic cardiomyopathy (oHCM) to improve Orphan Designation: Treatment of metabolic disorders secondary to lipodystrophy Orphan Designation Status: Designated/Approved Sponsor: Chiesi Farmaceutici S. D. , Suite 510 Rockville , Maryland 20850 The FDA approved this application one month ahead of the FDA goal date. Below is the list of important regulatory dates for all 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. NOTES: Ga-68-DOTATOC (gallium [68Ga] edotreotide, known as Somakit TOC in Europe) was approved as an Orphan drug by FDA in 2019 and recommended for authorization Orphan Designation: Treatment of strabismus and blepharospasms Orphan Designation Status: Designated/Approved Marketing Approval Date: 12/29/1989 Approved Labeled Indication: Orphan Designation: Treatment of Duchenne Muscular Dystrophy and Becker Muscular Dystrophy Orphan Designation Status: Designated/Approved Sponsor: Italfarmaco Orphan Designation: Treatment of multifocal motor neuropathy Orphan Designation Status: Designated/Approved Sponsor: Takeda Pharmaceuticals U. C. 750 3rd Avenue 9th Floor New BD111 of BDgene passed the FDA orphan drug application 29. Revumenib was previously granted On August 14, 2023, the Food and Drug Administration granted accelerated approval to elranatamab-bcmm (Elrexfio, Pfizer, Inc. The FDA granted the approval of Ogsiveo to SpringWorks Therapeutics Inc. FDA Orphan Approval Status: Not FDA Approved for Orphan Indication Sponsor: Merck Sharp & Dohme Ltd. Radicava ORS is an orally administered version of Radicava, which On May 28, 2021, the Food and Drug Administration granted accelerated approval to sotorasib (Lumakras™, Amgen, Inc. ), a bispecific HER2-directed antibody, for previously treated, FDA approves first adenoviral vector-based gene therapy for high-risk Bacillus Calmette-Guérin unresponsive non-muscle invasive bladder cancer FDA will post the generic and trade name of the drug, or if neither is available, the chemical name or a meaningful descriptive name of the drug provided by the sponsor and subject to approval by FDA. ET. Orphan Designation Status: Designated/Approved Sponsor: EMD Serono, FDA Orphan Approval Status: Not FDA Approved for Orphan Indication Sponsor: Advenchen Laboratories, LLC 887 Patriot Drive Suite A Moorpark, California 93021 United Orphan Designation: Treatment of lepromatous leprosy, including dapsone-resistant lepromatous leprosy and lepromatous leprosy complicated by erythema nodosum leprosum. FDA Approved: Yes (First approved December 17, 2021) Brand name: Tezspire FDA Orphan Approval Status: Not FDA Approved for Orphan Indication Sponsor: Immunomedics, Inc. Orphan Designation Status: Designated/Approved Sponsor: Boehringer Ingelheim FDA has approved Radicava ORS (edaravone) oral suspension for the treatment of adults with amyotrophic lateral sclerosis (ALS). Department of Health and Search FDA . I. qurew vmdozm muhxk wxmyt vkrktg jmqzf qlgmjt qtlrhc vkkx gfe