FDA Approved Drugs for Genetic Disease

Drugs Approved in 2017

Haegarda (C1 Esterase Inhibitor Subcutaneous [Human]) ; CSL Behring; For the routine prophylaxis to prevent Hereditary Angioedema attacks, Approved June 2017

Brineura (cerliponase alfa); BioMarin; For the treatment of late infantile neuronal ceroid lipofuscinosis type 2 , Approved April 2017

Mepsevii (vestronidase alfa-vjbk) ; Ultragenyx; For the treatment of Mucopolysaccharidosis VII , Approved November 2017

Drugs Approved in 2016

Spinraza (nusinersen); Biogen; For the treatment of spinal muscular atrophy, Approved December 2016

Drugs Approved in 2015

Kanuma (sebelipase alfa); Alexion; For the treatment of Lysosomal Acid Lipase (LAL) deficiency, Approved December 2015

Strensiq (asfotase alfa); Alexion; For the treatment of hypophosphatasia, Approved October 2015

Xuriden (uridine triacetate); Wellstat Therapeutics; For the treatment of hereditary orotic aciduria, Approved September 2015

Drugs Approved in 2014

Alprolix [Coagulation Factor IX (Recombinant), Fc Fusion Protein]; Biogen Idec; For the treatment of hemophilia B, Approved March 2014

Cerdelga (eliglustat); Genzyme; For the treatment of certain adult patients with Gaucher disease type 1, Approved August 2014

Qudexy XR (topiramate); Upsher-Smith Laboratories; For the treatment of partial onset and primary generalized tonic-clonic seizures and Lennox-Gastaut Syndrome, Approved March 2014

Vimizim (elosulfase alfa); BioMarin; For the treatment of Mucopolysaccharidosis type IVA, Approved February 2014

Drugs Approved in 2013

Kineret, anakinra; Swedish Orphan Biovitrum; For the treatment of Cryopyrin-Associated Periodic Syndromes, Approved January 2013

Kynamro (mipomersen sodium); Genzyme; For the treatment of homozygous familial hypercholesterolemia, Approved January 2013

Ravicti (glycerol phenylbutyrate); Hyperion Therapeutics; For the treatment of pediatrics and adults with urea cycle disorders, Approved February 2013

Tretten (Coagulation Factor XIII A-Subunit [Recombinant]); Novo Nordisk; For the treatment of congenital factor XIII (FXIII) A-subunit deficiency, Approved December of 2013

Drugs Approved in 2012

Elelyso (taliglucerase alfa); Pfizer; For the treatment of Gaucher disease, Approved May 2012

Fycompa (perampanel); Eisai; For the treatment of partial-onset seizures with or without secondarily generalized seizures, Approved October 2012

Juxtapid (lomitapide); Aegerion Pharmaceuticals; For the treatment of homozygous familial hypercholesterolemia, Approved December of 2012

Kalydeco (ivacaftor); Vertex Pharmaceuticals; For the treatment of cystic fibrosis with the G551D mutation in the CFTR gene, Approved January of 2012

Oxtellar XR (oxcarbazepine extended release); Supernus Pharmaceuticals; For the adjunctive therapy of partial seizures in adults and in children 6 years to 17 years of age, Approved October 2012

Ultresa (pancrelipase) delayed-release capsules; Aptalis Pharma; For the treatment of exocrine pancreatic insufficiency due to cystic fibrosis or other conditions, Approved March 2012

Drugs Approved in 2011

Eylea (aflibercept); Regeneron Pharmaceuticals; For the treatment of neovascular (wet) age-related macular degeneration, Approved November 2011

Ferriprox (deferiprone); Apotex; For the treatment of transfusional iron overload due to thalassemia, Approved October 2011

Onfi (clobazam); Lundbeck; For the adjunctive treatment of seizures associated with Lennox-Gastaut syndrome, Approved October 2011

Potiga (ezogabine); Valeant Pharmaceuticals; For the treatment of partial-onset seizures, Approved June 2011

Drugs Approved in 2010

Cayston (aztreonam for inhalation solution); Gilead; For the treatment of cystic fibrosis patients with Pseudomonas aeruginosa, Approved February 2010

Vpriv (velaglucerase alfa for injection); Shire Pharmaceuticals; For the treatment of type 1 Gaucher disease, Approved March 2010

Drugs Approved in 2009

Berinert (C1 Esterase Inhibitor (Human)); CSL Behring; For the treatment of abdominal or facial attacks of hereditary angioedema, Approved October 2009

Kalbitor (ecallantide); Dyax; For the treatment of hereditary angioedema, Approved November 2009

Sabril (vigabatrin); Lundbeck; For the treatment of infantile spasms and complex partial seizures, Approved August 2009

Saphris (asenapine); Schering-Plough; For the treatment of schizophrenia and manic or mixed bipolar 1 episodes, Approved August of 2009

Zenpep (pancrelipase); Eurand; For the treatment of pancreatic insufficiency due to cystic fibrosis or other conditions, Approved August 2009

Drugs Approved in 2008

Banzel (rufinamide); Elan Pharmaceuticals; For the treatment of seizures associated with Lennox-Gastaut syndrome in pediatrics and adults, Approved November 2008

Stavzor (valproic acid delayed release); Banner Pharmacaps; For the treatment of bipolar manic disorder, seizures and migraine headaches, Approved July 2008

Vimpat (lacosamide); Schwarz Pharma; For the treatment of partial-onset seizures in adults with epilepsy, Approved October 2008

Drugs Approved in 2000

Kogenate FS (Antihemophilic Factor Recombinant); Bayer; For the treatment of hemophilia A, Approved June 2000

Neurontin (gabapentin) oral solution; Parke-Davis; Treatment of partial seizures in adults with epilepsy, Approved March 2000

Trileptal (oxcarbazepine) Tablets; Novartis; Approved for use as adjunctive therapy in the treatment of partial seizures in children (monotherapy for adults), Approved January 2000

Zonegran (zonisamide) Capsules; Dainippon Pharmaceutical; Adjunctive therapy for treatment of partial seizures in adults with epilepsy, Approved March 2000

Drugs Approved in 1999

Keppra; UCB; Levetiracetam tablets for adjunctive treatment of partial onset seizures in adults with epilepsy, Approved November 1999

Drugs Approved in 1998

Clonazepam; Watson Pharmaceuticals; Generic equivalent of Klonopin, Approved January 1998

Lamictal Chewable Dispersible Tablets; GlaxoSmithKline; Treatment for Epilepsy, Approved August 1998

Pulmozyme (dornase alfa); Genentech; Treatment for cystic fibrosis, Approved March 1998

Drugs Approved in 1997

BeneFIX (coagulation Factor IX (recombinant)); Genetics Institute; Treatment for hemophilia B, Approved February 1997

Carbatrol; Elan Pharmaceuticals; Treatment for epileptic seizures and trigeminal neuralgia, Approved September 1997

Galzin (zinc acetate); Teva Pharmaceutical; Treatment for Wilson’s disease, Approved January 1997

Tobi; PathoGenesis; Treatment for cystic fibrosis, Approved December 1997

Topamax (topiramate); Ortho-McNeil Pharmaceutical; Treatment for partial onset seizures, Approved January 1997

Drugs Approved in 1996

AlphaNine SD Coagulation Factor IX (Human); Alpha Therapeutic Corporation; Treatment for the prevention and control of bleeding in subjects with hemophilia, Approved July 1996

Depakote (divalproex sodium); Abbott Laboratories; Treatment for epilepsy, Approved June 1996

Pulmozyme (dornase alfa); Genentech; Treatment for cystic fibrosis patients with advanced disease, Approved December 1996

Saizen; Ares-Serono; Treatment for growth hormone deficiency and Turner Syndrome, Approved May 1996

Tegretol (carbamazepine); Ciba Pharmaceuticals; Treatment for young children with epilepsy, Approved March 1996

Tegretol XR (carbamazepine); Ciba-Geigy; Anti-epileptic treatment, Approved May 1996