PTC Therapeutics, Inc., a biopharmaceutical company, focuses on the discovery, development, and commercialization of medicines to patients 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 ferroptosis and inflammation platforms, including vatiquinone to treat Friedreich ataxia and utreloxastat for the treatment of amyotrophic lateral sclerosis. 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. PTC Therapeutics, Inc. was incorporated in 1998 and is headquartered in South Plainfield, New Jersey.

Amicus Therapeutics, Inc., a biotechnology company, focuses on discovering, developing, and delivering medicines for rare diseases. Its commercial product and product candidates include Galafold, an oral precision medicine for the treatment of adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene variant; and Pombiliti + Opfolda, for the treatment of late onset. It has collaboration and license agreements with the University of Pennsylvania to research and develop parvovirus gene therapy products; and GlaxoSmithKline. Amicus Therapeutics, Inc. was incorporated in 2002 and is headquartered in Princeton, New Jersey.

Pharming Group N.V., a biopharmaceutical company, develops and commercializes protein replacement therapies and precision medicines for the treatment of rare diseases in the United States, Europe, and internationally. The company offers RUCONEST, a recombinant C1 esterase inhibitor for the treatment of acute attacks in adult and adolescent patients with acute hereditary angioedema (HAE); and Joenja (leniolisib), an oral small molecule PI3K? inhibitor for the treatment of activated phosphoinositide 3-kinase delta syndrome. It also develops OTL-105, an investigational ex-vivo autologous hematopoietic stem cell gene therapy for the treatment of HAE. The company has a development collaboration and license agreement with Novartis; and a strategic collaboration agreement with Orchard Therapeutics plc for research, development, manufacturing, and commercialization of OTL-105. Pharming Group N.V. was incorporated in 1988 and is headquartered in Leiden, the Netherlands.

Aquestive Therapeutics, Inc. operates as a pharmaceutical company in the United States and internationally. The company markets Sympazan, an oral soluble film formulation of clobazam for the treatment of lennox-gastaut syndrome; Suboxone, a sublingual film formulation of buprenorphine and naloxone for the treatment of opioid dependence; Zuplenz, an oral soluble film formulation of ondansetron for the treatment of nausea and vomiting associated with chemotherapy and post-operative recovery; and Azstarys, a once-daily product for the treatment of attention deficit hyperactivity disorder. Its proprietary product candidates comprise Libervant, a buccal soluble film formulation of diazepam for the treatment of seizures; KYNMOBI, a sublingual film formulation of apomorphine for the treatment of episodic off-periods in Parkinson's disease; and Exservan, an oral soluble film formulation of riluzole for the treatment of amyotrophic lateral sclerosis. The company's proprietary pipeline of complex molecule product includes AQST-108, a sublingual film formulation delivering systemic epinephrine for the treatment of conditions other than anaphylaxis; and Anaphylm, an epinephrine sublingual film for the emergency treatment of allergic reactions, including anaphylaxis. In addition, it develops Adrenaverse, an epinephrine prodrug platform. The company was incorporated in 2004 and is headquartered in Warren, New Jersey.

Cumberland Pharmaceuticals Inc., a specialty pharmaceutical company, focuses on the acquisition, development, and commercialization of prescription products for hospital acute care, gastroenterology, and oncology in the United States and internationally. The company offers Acetadote, an injection for the treatment of acetaminophen poisoning; Caldolor, an injection for the treatment of pain and fever; Kristalose, a prescription laxative oral solution for the treatment of constipation; Omeclamox-Pak for the treatment of Helicobacter pylori infection and duodenal ulcer disease; Vaprisol, an injection for treating euvolemic and hypervolemic hyponatremia; Sancuso, an injection for the treatment of chemotherapy treatment; and Vibativ, an injection for the treatment of certain serious bacterial infections, including hospital-acquired and ventilator-associated bacterial pneumonia, as well as complicated skin and skin structure infections. It develops RediTrex injection for the treatment of active rheumatoid, juvenile idiopathic, and severe psoriatic arthritis, as well as disabling psoriasis. In addition, the company is developing ifetroban, a product candidate that is in phase II clinical trial for the treatment of aspirin-exacerbated respiratory disease, systemic sclerosis, and duchenne muscular dystrophy; and has completed phase II clinical trial for the treatment of hepatorenal syndrome and portal hypertension. Further, it develops a clinical program for the use of ifetroban to treat progressive fibrosing interstitial lung diseases. The company was incorporated in 1999 and is headquartered in Nashville, Tennessee.

Applied Therapeutics, Inc., a clinical-stage biopharmaceutical company, engages in the development of a pipeline of novel product candidates against validated molecular targets in indications of high unmet medical need in the United States. The company's lead product candidate is AT-007 (also called govorestat) that has completed phase 3 for the treatment of galactosemia in healthy volunteers and adults, in pediatric clinical study for the treatment of galactosemia in kids, for treating enzyme sorbitol dehydrogenase, and for the treatment of phosphomannomutase enzyme-CDG. It also develops AT-001 (also called caficrestat) that is in phase 3 clinical trials to treat diabetic cardiomyopathy, as well as for the treatment of diabetic peripheral neuropathy; and AT-003, which is in preclinical studies for the treatment diabetic retinopathy. The company has exclusive license and supply agreement with Mercury Pharma Group Limited to commercialize drug products containing AT-007. Applied Therapeutics, Inc. was incorporated in 2016 and is headquartered in New York, New York.

Grace Therapeutics, Inc. engages in the development and commercialization of pharmaceutical products for rare and orphan diseases in Canada. The company's lead product candidate is the GTX-104, an intravenous infusion to treat subarachnoid hemorrhage. It also develops GTX-102, an oral mucosal betamethasone spray for the treatment of ataxia-telangiectasia; and GTX-101, a topical bioadhesive film-forming bupivacaine spray for postherpetic neuralgia. The company was formerly known as Acasti Pharma Inc. and changed its name to Grace Therapeutics, Inc. in October 2024. The company was incorporated in 2002 and is headquartered in Princeton, New Jersey.

Aeterna Zentaris Inc., a specialty biopharmaceutical company, engages in developing and commercializing therapeutics and diagnostic tests in Canada, Switzerland, Ireland, Denmark, Germany, the United States, and internationally. Its lead product is Macrilen (macimorelin), an orally available peptidomimetic ghrelin receptor (GHSR-1a) agonist that stimulates the secretion of growth hormone by binding to the GHSR-1a for the diagnosis of patients with adult growth hormone deficiency and childhood-onset growth hormone deficiency, as well as endocrinology and oncology indications; AEZS-150, a delayed clearance parathyroid hormone fusion polypeptide that is in preclinical trial for the treatment of chronic hypoparathyroidism; and AEZS-130 that is in preclinical trial for the treatment of amyotrophic lateral sclerosis. The company also has a license and research agreement with University of Wuerzburg to develop, manufacture, and commercialize AIM biologicals for the treatment of neuromyelitis optica spectrum disorder; and for pre-clinical development towards the potential treatment of Parkinson's disease. In addition, it has a license agreement with Consilient Health Ltd. and NK MEDITECH Ltd. for the development and commercialization of macimorelin; a distribution and commercialization agreement with Er-Kim Pharmaceuticals Bulgaria Eood for the commercialization of macimorelin for the diagnosis of growth hormone deficiency in children and adults; as well as The University of Sheffield, the United Kingdom for the development, manufacture, and commercialization of parathyroid hormone fusion polypeptides for the treatment of chronic hypoparathyroidism. Aeterna Zentaris Inc. was incorporated in 1990 and is headquartered in Toronto, Canada.