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Regulatory class of pharmaceutical drug
An orphan drug is a pharmaceutical agent that is developed to treat certain rare medical conditions. An orphan drug would not be profitable to produce
Orphan_drug
Law passed in the United States to facilitate development of orphan drugs
The Orphan Drug Act of 1983 is a law passed in the United States to facilitate development of orphan drugs—drugs for rare diseases such as Huntington's
Orphan_Drug_Act_of_1983
Disease affecting a small percentage of the population
financial incentives from governments or other agencies. Orphan drugs are medications targeting orphan diseases. Most rare diseases are genetic in origin and
Rare_disease
Disease
Because of preliminary data suggesting the drug may have activity, the U.S. FDA in 2013 granted tolcapone "orphan drug status" in studies aiming at the treatment
Wild-type transthyretin amyloid
Wild-type_transthyretin_amyloid
United States program for negotiating Medicare drug prices
onward, 20 drugs are to be added each year. In July 2025, the One Big Beautiful Bill Act expanded the program's orphan drug exclusion to cover drugs with multiple
Medicare Drug Price Negotiation Program
Medicare_Drug_Price_Negotiation_Program
Overview of the cost of medicines in the United States
approved orphan drugs. An orphan drug may cost as much as $400,000 annually. Monopolizing orphan drugs has proven to be a very profitable strategy for drug companies;
Prescription drug prices in the United States
Prescription_drug_prices_in_the_United_States
Industry involved with discovery, development, production and marketing of drugs
and Drug Administration. Archived from the original on 7 April 2020. Retrieved 31 May 2007. "The Orphan Drug Act (as amended)". U.S. Food and Drug Administration
Pharmaceutical_industry
Italian pharmaceutical company
pharmaceutical company in the world and assignee in the United States of an Orphan Drug Designation, subsequently receiving another seven. With the discovery
Leadiant_Biosciences
Type or designation of pharmaceuticals
increased to 200. Specialty drugs may also be designated as orphan drugs or ultra-orphan drugs under the U. S. Orphan Drug Act of 1983. This was enacted
Specialty drugs in the United States
Specialty_drugs_in_the_United_States
Topics referred to by the same term
up orphan in Wiktionary, the free dictionary. An orphan is one who has lost both parents. Orphan(s) or The Orphan(s) may also refer to: The Orphan (1920
Orphan_(disambiguation)
Term for a groundbreaking pharmaceutical drug
Advanced Therapies or orphan drugs, first-in-class status itself has no regulatory effect. By definition, a first-in-class drug does not have the safety
First-in-class_medication
Rare disease database
base dedicated to rare diseases as well as corresponding diagnosis, orphan drugs, clinical trials and expert networks. Orphanet was founded in France
Orphanet
Cholesterol 24-hydroxylase inhibitor
Takeda announced they were halting development of the drug. Soticlestat was designated as an orphan drug by the FDA in 2017 for the treatment for both Dravet
Soticlestat
U.S. nonprofit organization
development of orphan drugs, or drugs for treating rare diseases. They succeeded in getting the United States Congress to pass the Orphan Drug Act (ODA) in
National Organization for Rare Disorders
National_Organization_for_Rare_Disorders
American political advisor (born 1991)
Caitlin (January 10, 2025). "Pharma taps Holland and Knight ahead of orphan drug tax fight". Politico. Retrieved February 2, 2026. Cai, Sophia; Burns
Katie_Miller
French biotechnology company
Orphalan S.A. is an orphan drug development and commercialization company headquartered in Paris, France. It specializes in the development of treatments
Orphalan
Chemical compound
Ivacaftor Orphan Drug Designations and Approvals". U.S. Food and Drug Administration (FDA). 15 June 2017. Retrieved 25 October 2020. "Trikafta Orphan Drug Designations
Tezacaftor
Medication
States in October 2025. The US Food and Drug Administration granted the application for nerandomilast an orphan drug designation for the idiopathic pulmonary
Nerandomilast
2013 Canadian science fiction thriller television series
series Orphan Black: Echoes. Kevin Hanchard as Art Bell, a detective and Beth's police partner Michael Mando as Vic Schmidt, Sarah's abusive, drug-dealing
Orphan_Black
Blood pressure medication
outcomes do not differ by sex, age, or race. In October 2014, the US Food and Drug Administration (FDA) issued a black box warning that losartan can cause fetal
Losartan
Medication
The US Food and Drug Administration granted the application for lirafugratinib priority review, breakthrough therapy, and orphan drug designations. Lirafugratinib
Lirafugratinib
Medication and metabolite of cholesterol
FDA-Approved Drugs". U.S. Food and Drug Administration (FDA). Archived from the original on 25 January 2017. Retrieved 29 April 2020. "Actigall Orphan Drug Designation
Ursodeoxycholic_acid
Chemical compound
bleomycin-induced lung fibrosis, and COVID-19 caused ARDS. It was designated as an orphan drug by the FDA for the treatment of X-linked adrenoleukodystrophy. Lammel
Sobetirome
Medication
2026. The US Food and Drug Administration granted the application for zilurgisertib fast track, priority review, and orphan drug designations for this
Zilurgisertib
Medication
low-density lipoprotein (VLDL). The US Food and Drug Administration (FDA) granted the application of olezarsen orphan drug designation in February 2024. In August
Olezarsen
Substance used to diagnose, cure, treat, or prevent disease
pharmaceutical drug, medicinal product, medicinal drug, or simply drug) is a drug used to diagnose, cure, treat, or prevent disease. Drug therapy (pharmacotherapy)
Medication
Medication to treat symptoms of narcolepsy
raised the price of the drug dramatically after it acquired Orphan, and paid a $20M fine for off-label marketing of the drug in 2007. Clinical use of
Sodium_oxybate
American biomedical research
companies to develop orphan drugs by the FDA's orphan drug products programme is taking the companies' attention away from developing drugs that will benefit
Biomedical research in the United States
Biomedical_research_in_the_United_States
Chemical compound
phosphate (Firdapse) has orphan drug status in the EU for Lambert–Eaton myasthenic syndrome and Catalyst holds both an orphan designation and a breakthrough
Amifampridine
In 2018 it announced that its drug, OBI-3424, has been granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA) for the treatment
OBI_Pharma,_Inc.
Pharmaceutical company in Ghent, Belgium
diseases and cancer. Its work primarily focuses on the development of orphan drugs. In 2008, Argenx, based in Ghent, was founded by three scientists: Tim
Argenx
US biopharmaceutical company
development of amifampridine and its phosphate has brought attention to orphan drug policies that grant market exclusivity as an incentive for companies
Catalyst_Pharmaceuticals
Medication used to treat diabetes
granted the application for metformin orphan drug designation. The European Medicines Agency granted orphan drug status to metformin. Metformin and its
Metformin
Chemical compound
and Drug Administration (FDA) for the prevention of hemolytic disease of the fetus and newborn. Additionally, the FDA granted nipocalimab orphan drug designation
Nipocalimab
Enzyme inhibitor that inhibits urease
(struvite stones). In 1983 the US Food and Drug Administration approved acetohydroxamic acid (AHA) as an orphan drug for "prevention of so-called struvite
Acetohydroxamic_acid
Swedish-British pharmaceutical company
employees as of 2024. In April 2015, AstraZeneca's drug tremelimumab was approved as an orphan drug for the treatment of mesothelioma in the United States
AstraZeneca
Genetic disorder involving an imprinted genomic region
formulation of betahistine dihydrochloride received orphan drug designation from the US Food and Drug Administration (FDA) for the treatment of obesity
Prader–Willi_syndrome
Pharmaceutical Company
deficiency In clinic, phase 3 Orphan Drug designation, partnered with Takeda ARO-APOC3 Hypertriglyceridemia Phase 2, 3 Orphan Drug designation, Fast Track Designation
Arrowhead_Pharmaceuticals
Progressive neurological disease
Orphan Drug Designations and Approvals". www.accessdata.fda.gov. Archived from the original on April 27, 2021. Retrieved 2019-08-01. "Search Orphan Drug
Spinocerebellar_ataxia
Specially fomulated food in medical setting
and Drug Administration's 1988 Orphan Drug Act Amendments and subject to the general food and safety labeling requirements of the Federal Food, Drug, and
Medical_food
USFDA program and procedures
application Drug discovery FDA Fast Track Development Program Good Manufacturing Practice Inverse benefit law Lists of investigational drugs Orphan drug TOL101
Investigational_New_Drug
Combination drug
Sleepwalking. Xywav was granted orphan drug designation by the US Food and Drug Administration. Xywav is a prescription drug and a Schedule III controlled
Xywav
Monoclonal antibody
rights to the drug from Genmab. Daratumumab was granted breakthrough therapy drug status in 2013, for multiple myeloma. It was granted orphan drug status for
Daratumumab
Chemical compound
received orphan drug designation in the United States for mantle cell lymphoma and chronic lymphocytic leukemia, and was similarly designated as an orphan medicinal
Acalabrutinib
American biotech firm
2019, a drug named ARCT-810 which uses Lunar, received FDA orphan drug status for treating ornithine transcarbamylase deficiency. The orphan drug designation
Arcturus_Therapeutics
Chemical compound
Trifarotene was granted orphan drug designation for the treatment of congenital ichthyosis by both the U.S. Food and Drug Administration (FDA) and the
Trifarotene
Pharmaceutical drug used in cancer treatment
studies to ensure that the results are valid. Pembrolizumab was granted orphan drug designation for small-cell lung cancer in October 2017. In June 2018
Pembrolizumab
Chemical compound
Europea Union in December 2023. Zilucoplan was granted orphan drug designation by the US Food and Drug Administration (FDA) in August 2019, and by the EMA
Zilucoplan
Medication
source?] The US Food and Drug Administration (FDA) granted the application for efgartigimod alfa fast track and orphan drug designations. The FDA granted
Efgartigimod_alfa
Rare human genetic lysosomal storage disorder
of both treatments were similar. The US Food and Drug Administration (FDA) granted Galafold orphan drug status in 2004, and the European Commission followed
Fabry_disease
Medication
September 2026. The US Food and Drug Administration (FDA) granted the application for apitegromab fast track, orphan drug, and rare pediatric disease designations
Apitegromab
American businessman (1946–2017)
known as orphan diseases. Genzyme used biological processes to manufacture drugs that were not easily copied by generic drug manufacturers. The drugs were
Henri_Termeer
Pharmaceutical compound
rearrangements or NPM1 mutations. The U.S. Food and Drug Administration (FDA) has granted both Fast Track and Orphan Drug designations to Enzomenib. "Enzomenib -
Enzomenib
Chemical compound
Phase II trials showed mixed results; it then began to develop it as an orphan drug as an adjunct to electroconvulsive therapy for major depressive disorder
Pramiracetam
Deuterated cystic fibrosis drug (ivacaftor analogue)
US Food and Drug Administration (FDA) granted the application for vanzacaftor, tezacaftor, and deutivacaftor combination therapy orphan drug designation
Vanzacaftor/tezacaftor/deutivacaftor
Vanzacaftor/tezacaftor/deutivacaftor
Pharmaceutical drug
due to systolic dysfunction. Mavacamten was granted orphan drug designation by the US Food and Drug Administration (FDA). In April 2023, the Committee
Mavacamten
Disease affecting a very small percentage of the population
companies developing drugs that treat ultra-rare diseases. In 2018, the Scottish Government introduced a new definition of 'ultra-orphan medicines' that can
Ultra-rare_disease
Medication used to treat fungal infections
an orphan drug by both the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA). "Itraconazole Use During Pregnancy". Drugs.com
Itraconazole
Chemical compound
States in December 2025. The US Food and Drug Administration granted the application for aficamten orphan drug and breakthrough therapy designations. In
Aficamten
Chemical compound
lymphoid malignancies. In January 2017, it was granted orphan drug status by the U.S. Food and Drug Administration for advanced cholangiocarcinoma. It is
Silmitasertib
American biopharmaceutical company
treatment of advanced gastric cancer. It has been granted Orphan Drug status by the U.S. Food and Drug Administration (FDA). It has reported encouraging interim
ACT_Biotech_Inc
Swedish pharmaceutical company
2010 when Biovitrum acquired Swedish Orphan International Holding AB, a prominent European developer of orphan drugs. Following the acquisition, the combined
Swedish_Orphan_Biovitrum
Medication
receiving placebo. The US Food and Drug Administration (FDA) granted the application for omaveloxolone orphan drug, fast track, priority review, and rare
Omaveloxolone
Gene therapy
rejection. The US Food and Drug Administration (FDA) granted the application for exagamglogene autotemcel priority review, orphan drug, fast track, and regenerative
Exagamglogene_autotemcel
Pharmaceutical discovery procedure
large commercial success or public health effect can be expected, the orphan drug funding process ensures that people who experience those disorders can
Drug_discovery
Experimental drug
and secondary endpoints. Since ibutamoren is still an Investigational New Drug, it has not yet been approved to be marketed for consumption by humans in
Ibutamoren
Pharmaceutical drug
treatment of atypical hemolytic uremic syndrome, it designated it as an orphan drug. The FDA approval in 2011 was based on two small prospective trials of
Eculizumab
Child not being cared for despite having at least one living parent
social orphan is a child with no adults looking after them, even though one or more parents are still alive. Usually the parents are alcoholics, drug abusers
Social_orphan
Chemical compound
treatment of polycystic kidney disease. The FDA granted Jynarque an orphan drug designation in April 2012, for the treatment of autosomal dominant polycystic
Tolvaptan
Medication
medical use in the European Union in September 2023. The EMA granted orphan drug designation to epcoritamab in both February and June 2022. Epcoritamab
Epcoritamab
Chemical compound
indications. Dinaciclib is being developed by Merck & Co. It was granted orphan drug status by the FDA in 2011. Cyclin-dependent kinase inhibitor dinaciclib
Dinaciclib
Medication
doi:10.1002/hep.20822. PMID 16114043. "Imetelstat Orphan Drug Designations and Approvals". U.S. Food and Drug Administration (FDA). Archived from the original
Imetelstat
Medication used for spinal muscular atrophy
the first approved drug used in treating this disorder. Since the condition it treats is so rare, nusinersen has so-called "orphan drug" designation in the
Nusinersen
American pharmaceutical company
genetic diseases. In May 2020, CTX001 has received Orphan Drug Designation from the U.S. Food and Drug Administration for transfusion-dependent beta thalassemia
Vertex_Pharmaceuticals
Gene therapy medication
US Food and Drug Administration (FDA) granted onasemnogene abeparvovec fast track, breakthrough therapy, priority review, and orphan drug designations
Onasemnogene_abeparvovec
Medication
European Union in May 2024. The FDA approved tofersen for priority review, orphan drug, and fast track designations. Tofersen's effectiveness was evaluated
Tofersen
Switzerland based biotechnology company
was barred by provisions of the Orphan Drug Act from entering the US market, where Biogen’s treatment, Avonex, held orphan status. Bertarelli and Serono
Serono
Biopharmaceutical company in New Jersey, United States
granted their product, certepetide (also known as iRGD and CEND-1), orphan drug status in pancreatic cancer, followed by a Fast track (FDA) status in
Lisata_Therapeutics
Chemical compound
Biopharmaceuticals and was granted priority review and orphan drug designations by the United States Food and Drug Administration (FDA) for the treatment of fibrodysplasia
Palovarotene
Form of treatment for genetic disorders and other illnesses
amyloidosis in October 2018. The application for inotersen was granted orphan drug designation. It was developed by Ionis Pharmaceuticals and licensed to
Antisense_therapy
Monoclonal antibody
2026. The US Food and Drug Administration (FDA) granted the application for garetosmab breakthrough therapy, fast track, orphan drug, and priority review
Garetosmab
American biopharmaceutical company
Syndrome. In October 2017, Neurocrine announced that it had been granted orphan drug designation status from the FDA for valbenazine for the treatment of
Neurocrine_Biosciences
Combination cystic fibrosis medication
Food and Drug Administration (FDA) granted the application priority review, in addition to fast track, breakthrough therapy, and orphan drug designations
Elexacaftor/tezacaftor/ivacaftor
Elexacaftor/tezacaftor/ivacaftor
Pharmaceutical drug
patients. While sirolimus was considered for treatment of LAM, it received orphan drug designation status because LAM is a rare condition. The safety of LAM
Sirolimus
Medication for transthyretin amyloidosis
tafamidis an orphan medicine and the Food and Drug Administration also designated tafamidis meglumine as an orphan drug. Tafamidis was approved in the European
Tafamidis
Monoclonal antibody
application for talquetamab priority review, breakthrough therapy, and orphan drug designations. In the United States, Janssen received breakthrough therapy
Talquetamab
Chemical compound
cryptococcosis and tularaemia. OSU-03012 received an orphan drug designation in combination with the antifungal drug fluconazole for cryptococcosis of the brain
OSU-03012
Chemical compound
formulation of betahistine dihydrochloride received orphan drug designation from the US Food and Drug Administration (FDA) for the treatment of obesity
Betahistine
British-American surgeon and author
exposed loopholes in the Orphan Drug Act of 1983 that, they said, accounted for higher drug pricing. His article "The Orphan Drug Act: Restoring the Mission
Marty_Makary
Statistics† Open Access Rheumatology: Research and Reviews Open Access Surgery Orphan Drugs: Research and Reviews† Orthopedic Research and Reviews Pathology and
List of Dove Medical Press academic journals
List_of_Dove_Medical_Press_academic_journals
Monoclonal antibody
The US Food and Drug Administration (FDA) granted the application for tarlatamab priority review, breakthrough therapy, and orphan drug designations. Efficacy
Tarlatamab
Chemotherapy medication
Reports. 53 (1): 33–37. PMID 5772652. Investigational Sarcoma Drug GPX-150 Gets Orphan Drug Designation. 2016 Archived 24 January 2016 at the Wayback Machine
Doxorubicin
Chemical activating melatonin receptors
States for primary insomnia in 2010. The Food and Drug Administration (FDA) granted tasimelteon orphan drug designation status for blind individuals without
Melatonin_receptor_agonist
Medication
placebo group. The US Food and Drug Administration granted the application for plozasiran breakthrough therapy, orphan drug, and fast track designations
Plozasiran
Chemical compound
effects of hemodialysis and chemotherapy. In September 2022, the U.S. Food and Drug Administration (FDA) approved sodium thiosulfate under the trade name Pedmark to
Sodium_thiosulfate
Protein with a receptor structure but with unidentified ligand
an orphan receptor site is the PCP binding site in the NMDA receptor, a type of ligand-gated ion channel. This site is where the recreational drug PCP
Orphan_receptor
Medication
Australia. The US Food and Drug Administration (FDA) granted the application for levacetylleucine priority review, fast track, orphan drug, and rare pediatric
Levacetylleucine
Anti-cancer medication
for vorasidenib priority review, fast track, breakthrough therapy, and orphan drug designations. In July 2025, the Committee for Medicinal Products for
Vorasidenib
Chemical compound
and an orphan drug designation in 2019. Risdiplam is the international nonproprietary name (INN). Since 2019, Roche has been offering the drug globally
Risdiplam
Cancer drug
Abraxane where it is designated as an orphan drug as first-line treatment, in combination with gemcitabine, for the orphan disease "metastatic adenocarcinoma
Protein-bound_paclitaxel
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