Treatments

The following database contains products that have been granted orphan status, or have been market approval in the EU or the USA.

You can filter the content by clicking on the titles: Product, Indication, Sponsor and Date. Clicking once, will filter the column alphabetically from A – Z and a second click on the same title will filter from Z – A.

Become a Partner.

Country Product Indication Sponsor Date
Dantrolene sodium suspension for injection Malignant hyperthermia Eagle Pharmaceuticals, Inc.;50 Tice Blvd, Suite 315;Woodcliff Lake, New Jersey, 07677 August 16, 2013
Small molecule inhibitor of histone methyltransferase DOT1L Acute myeloid leukemia Epizyme Inc.;400 Technology Square, 4th Floor;Cambridge, Massachusetts, 02139 August 15, 2013
ADXS11-001 Anal cancer Advaxis, Inc.;305 College Road East;Princeton, New Jersey, 08540 August 12, 2013
Bispecific antibody (monoclonal antibody) HER2-expressing advanced adenocarcinoma of the stomach and gastroesophageal junction Merrimack Pharmaceuticals, inc.;One Kendall Square, Suite B7201;Cambridge, Massachusetts, 02139 August 8, 2013
Bispecific antibody (monoclonmal antibody) HER2-expressing adenocarcinoma of the esophagus Merrimack Pharmaceuticals, Inc.;1 Kendall Square, Suite B7201;Cambridge, Massachusetts, 02139 August 8, 2013
Conjugate of a dengue virus specific small chemical ligand and an amphiphilic PEG based polymer Dengue fever (includes dengue hemorrhagic fever and dengue shock syndrome) NanoViricides Incorporated;135 Wood Street, Suite 205;West Haven, Connecticut, 06516 August 6, 2013
Human Hemin Prevention of ischemia reperfusion injury in patients undergoing solid organ transplantation Borders Technology Management Ltd;2 Broomlee Mains Court, Peeblesshire, EH467JG;Scotland August 6, 2013
L. reuteri Necrotizing enterocolitis Infant Bacterial Therapeutics;Kungsbroplan 3A, P. O. Box 3242;Stockholm August 1, 2013
(S)-3-(1-(9H-purin-6-ylamino)ethyl)-8-chloro-2-phenylisoquinolin-1(2H)-one Follicular lymphoma Verastem, Inc.;117 Kendrick Street, Suite 500;Needham, Massachusetts, 02494 August 1, 2013
Angiotensin (1-7)[A(1-7)] Duchenne muscular dystrophy Tarix Pharmaceuticals Ltd.;12 Bow Street;Cambridge, Massachusetts, 02138 July 25, 2013
(5R)-5-(4-{[2-fluorophenyl)methyl]oxy}phenyl)-L-prolinamide, hydrochloride Trigeminal neuralgia Biogen, Inc.;300 Binney Street;Cambridge, Massachusetts, 02142 July 24, 2013
Bezafibrate Barth syndrome Barth Sydrome Foundation, Inc.;P. O. Box 618;Larchmont, New York, 10538 July 24, 2013
N-methyl-4-({4-[({3-methyl(methylsulfonyl)amino]pyrazin-2-yl}methyl)amino]-5-(trifluoromethyl)pyrimidin-2-yl}amino)benzamide hydrochloride Malignant pleural mesothelioma Verastem, Inc.;117 Kendrick Street, Suite 500;Needham, Massachusetts, 02494 July 18, 2013
Alisertib Small cell lung cancer Millennium Pharmaceuticals, Inc.;40 Landsdowne Street;Cambridge, Massachusetts, 02139 July 12, 2013
Pertuzumab Stomach Cancer Genentech, Inc.;1 DNA Way;South San Francisco, California, 94080 July 12, 2013
Denileukin diftitox Cutaneous T-cell lymphoma Eisai Inc.;155 Tice Blvd;Woodcliff Lake, New Jersey, 07677 July 12, 2013
Allopregnanolone Neimann-Pick disease, type C La Jolla Pharmaceutical Company, Inc.;10182 Telesis Court, 6th Floor, Suite 600;San Diego, California, 92121 July 12, 2013
(S)-4-(5-chloro-2-(isopropylamino)pyridin-4-yl)-N-(1-(3-chlorophenyl)-2-hydroxyethyl)-1H-pyrrole-2-carboxamide hydrochloride Metastatic Melanoma BioMed Valley Discoveries, Inc.;4520 Main Street, 16th Floor;Kansas City, Missouri, 64111 June 24, 2013
Flunarizine hydrochloride Alternating hemiplegia Marathon Pharmaceuticals, LLC;1033 Skokie Blvd., Suite 600;Northbrook, IL, 60062 June 24, 2013
Humanized 3F8-IgG1 monoclonal antibody Neuroblastoma Memorial Sloan-Kettering Cancer Center;1275 York Avenue;New York, New York, 10065 June 24, 2013
Flunarizine Alternating hemiplegia of childhood Marathon Pharmaceuticals, LLC;1033 Skokie Blvd., Suite 600;Northbrook, Illinois, 60062 June 24, 2013
Eculizumab Neuromyelitis optica Alexion Pharmaceuticals, Inc.;100 College Street;New Haven, Connecticut, 06510 June 24, 2013
Cyclo(-y-aminobutyryl-L-phenylalanyl-L-tryptophanyl-D-tryptophanyl-L-lysyl-L-threonly-L-phenylalanyl-N-3-carboxypropyl)-glycine amide, acetate salt Acromegaly Cortendo AB;900 Northbrook Drive;Trevose, Pennsylvania, 19053 June 24, 2013
IL-12 secreting dendritic cells loaded with autologous tumor lysate Glioma Activartis Biotech GmbH;1090 Vienna, Zimmermannplatz 10; June 24, 2013
Multi-peptide cancer vaccine Multiple myeloma OncoPep, Inc.;520 Boston Street;North Andover, Massachusetts, 01845 June 23, 2013