PTC Therapeutics, Inc.
PTC Therapeutics, Inc., a biopharmaceutical company, focuses on the discovery, development, and commercialization of medicines to children and adults living with rare disorders in the United States and internationally. The company offers Translarna and Emflaza for the treatment of Duchenne muscular dystrophy; Upstaza to treat aromatic l-amino acid decarboxylas (AADC) deficiency, a central nervous system disorder; Tegsedi and Waylivra for the treatment of rare diseases; and Evrysdi to treat spinal muscular atrophy (SMA) in adults and children. Its development pipeline products include Sepiapterin for the treatment of phenylketonuria; PTC518 splicing platform, which is being developed for the treatment of Huntington's disease; and inflammation and ferroptosis platforms, including vatiquinone to treat Friedreich's ataxia. The company distributes its products through third-party distributors. It has collaborations with F. Hoffman-La Roche Ltd., Hoffman-La Roche Inc., the SMA Foundation, National Taiwan University, Akcea Therapeutics, Inc., and Shiratori Pharmaceutical Co., Ltd. The company has license and collaboration agreement with Novartis Pharmaceuticals Corporation to develop PTC518 Huntington's disease program. It markets Upstaza, a gene therapy with the brand name Kebilidi in the United States. PTC Therapeutics, Inc. was incorporated in 1998 and is headquartered in Warren, New Jersey.
Open PTCT dashboard ↗Supernus Pharmaceuticals, Inc.
Supernus Pharmaceuticals, Inc., a biopharmaceutical company, develops and commercializes products for the treatment of central nervous system (CNS) diseases in the United States. The company offers Qelbree, a non-stimulant indicated for the treatment of attention-deficit hyperactivity disorder (ADHD); GOCOVRI for the treatment of dyskinesia and levodopa/carbidopa in patients with Parkinson's Disease (PD); Oxtellar XR, an extended-release oxcarbazepine product indicated for the monotherapy treatment of partial onset epilepsy seizures; and APOKYN for the acute and intermittent treatment of hypomobility or off episodes in patients with advanced PD. It also provides Trokendi XR, an extended release topiramate product indicated for the treatment of epilepsy, as well as for the prophylaxis of migraine headache; XADAGO for the treatment of levodopa/carbidopa in patients with PD experiencing off episodes; and MYOBLOC, a Type B toxin product indicated for the treatment of cervical dystonia and chronic sialorrhea. In addition, the company develops ONAPGO, which received FDA approval, for the treatment of motor fluctuations in adults with advanced PD; SPN-817, a first-in-class selective acetylcholinesterase inhibitor, which is in Phase 2 clinical trial, for the treatment of epilepsy, partial seizures, Dravet syndrome, and Lennox-Gaustaut syndrome; SPN-820, a small molecule in Phase 2 clinical trial for treating resistant depression; SPN-443, a stimulant in Phase 1 trial for the treatment of ADHD/CNS; and SPN-446, a preclinical product for CNS. It markets and sells its products through pharmaceutical wholesalers, specialty pharmacies, and distributors. It has a development agreement with Navitor Inc. for the conduct of Phase 2 clinical program of SPN-820. Supernus Pharmaceuticals, Inc. was incorporated in 2005 and is based in Rockville, Maryland.
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