BioMarin Pharmaceutical Inc. engages in the development and commercialization of therapies for people with serious and life-threatening rare diseases and medical conditions. Its commercial products include Vimizim, an enzyme replacement therapy for the treatment of mucopolysaccharidosis (MPS) IV type A, a lysosomal storage disorder; Naglazyme, a recombinant form of N-acetylgalactosamine 4-sulfatase for patients with MPS VI; and Kuvan, a proprietary synthetic oral form of 6R-BH4 that is used to treat patients with phenylketonuria (PKU), an inherited metabolic disease. The company's commercial products also comprise Palynziq, a PEGylated recombinant phenylalanine ammonia lyase enzyme, which is delivered through subcutaneous injection to reduce blood Phe concentrations; Brineura, a recombinant human tripeptidyl peptidase 1 for the treatment of patients with ceroid lipofuscinosis type 2, a form of Batten disease; Voxzogo, a once daily injection analog of c-type natriuretic peptide for the treatment of achondroplasia; and Aldurazyme, a purified protein designed to be identical to a naturally occurring form of the human enzyme alpha-L-iduronidase. In addition, it develops Roctavian, an adeno associated virus vector, for the treatment of patients with severe hemophilia A. The company serves specialty pharmacies, hospitals, and non-U.S. government agencies, as well as distributors and pharmaceutical wholesalers in the United States, Europe, Latin America, and internationally. It has license and collaboration agreements with Sarepta Therapeutics; Ares Trading S.A.; Catalyst Pharmaceutical Partners, Inc.; and CAMP4 Therapeutics Corporation. The company was incorporated in 1996 and is headquartered in San Rafael, California.

Neurocrine Biosciences, Inc. discovers, develops, and markets pharmaceuticals for neurological, neuroendocrine, and neuropsychiatric disorders in the United States and internationally. The company's products include INGREZZA for tardive dyskinesia and chorea associated with Huntington's disease; ALKINDI for adrenal insufficiency; Efmody capsules for classic congenital adrenal hyperplasia; Orilissa tablets for endometriosis; and Oriahnn capsules to treat uterine fibroids. Its product candidates in clinical development include valbenazine to treat dyskinetic cerebral palsy in pediatrics and adults; NBI-921352 to treat developmental and epileptic encephalopathy syndrome in pediatrics and adults; NBI-827104 to treat epileptic encephalopathy with continuous spike-and-wave during sleep; NBI-1076986 to treat movement disorders; crinecerfront to treat congenital adrenal hyperplasia in adults and children; EFMODY to treat congenital adrenal hyperplasia and adrenal insufficiency in adults; valbenazine for the adjunctive treatment of schizophrenia; NBI-1065845 for the treatment of inadequate response to treatment in major depressive disorder; luvadaxistat to treat cognitive impairment related to schizophrenia; NBI-1117568 for the treatment of schizophrenia; NBI-1070770 to treat major depressive disorder; NBI-1117570 for the treatment of symptoms of psychosis and cognition in neurological and neuropsychiatric conditions; and NBI-1117569, NBI-1117567, and NBI-1065890 to treat CNS indications. The company also has license and collaboration agreements with Heptares Therapeutics Limited; Takeda Pharmaceutical Company Limited; Idorsia Pharmaceuticals Ltd; Xenon Pharmaceuticals Inc.; Voyager Therapeutics, Inc.; BIAL – Portela & Ca, S.A.; Mitsubishi Tanabe Pharma Corporation; and AbbVie Inc. The company was incorporated in 1992 and is headquartered in San Diego, California.

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.

Crinetics Pharmaceuticals, Inc., a clinical-stage pharmaceutical company, focuses on the discovery, development, and commercialization of novel therapeutics for rare endocrine diseases and endocrine-related tumors. The company's lead product candidate is paltusotine, an oral selective nonpeptide somatostatin receptor type 2 agonist, which is in Phase 3 trial for the treatment of acromegaly; and Phase 2 trial for treating carcinoid syndrome associated with neuroendocrine tumors. It is also developing CRN04894, an investigational oral nonpeptide product candidate to antagonize the adrenocorticotrophic hormone (ACTH) receptor that has completed a Phase 1 study for the treatment of diseases caused by excess ACTH, including congenital adrenal hyperplasia and Cushing's disease. In addition, the company is developing antagonists of the parathyroid hormone (PTH) receptor for the treatment of primary hyperparathyroidism and humoral hypercalcemia of malignancy, and other diseases of excess PTH; identified investigational orally available somatostatin receptor type 3 targeted nonpeptide agonists for the treatment of autosomal dominant polycystic kidney disease; and developing thyroid-stimulating hormone receptor antagonists for the treatment of graves' disease and thyroid eye disease, as well as Oral GLP-1 and GIP nonpeptides for the treatment of diabetes and obesity. Crinetics Pharmaceuticals, Inc. was incorporated in 2008 and is headquartered in San Diego, California.

Xeris Biopharma Holdings, Inc., a biopharmaceutical company, engages in developing and commercializing therapies in Illinois. The company offers Gvoke, a ready-to-use liquid-stable glucagon for the treatment of severe hypoglycemia pediatric and adult patients; Keveyis, a therapy for the treatment of hyperkalemic, hypokalemic, and related variants of primary periodic paralysis; and Recorlev, a cortisol synthesis inhibitor proved for the treatment of endogenous hypercortisolemia in adult patients with Cushing's syndrome. It is also developing XP-8121, a once-weekly subcutaneous injection of levothyroxine that is in phase I clinical trial for the treatment of hypothyroidism; and non-aqueous XeriSol and XeriJect technologies for various therapies. The company was incorporated in 2005 and is headquartered in Chicago, Illinois.

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.

Eagle Pharmaceuticals, Inc., a pharmaceutical company, focuses on developing and commercializing product candidates to treat diseases of the central nervous system or metabolic critical care, and oncology in the United States. The company offers Ryanodex for malignant hyperthermia; and Belrapzo and Bendeka for chronic lymphocytic leukemia and indolent B-cell non-Hodgkin's lymphoma. Its product candidates also include EP-4104, a dantrolene sodium to treat organophosphate exposure; PEMFEXY, a ready-to-use/dilute liquid form of pemetrexed for non-small cell lung cancer and mesothelioma; EA-114 (fulvestrant) for HR+/HER- breast cancer; and Vasopressin injection, which is indicated to enhance blood pressure in adults with vasodilatory shock. The company has license and collaboration agreements with Combioxin, SA for the development and commercialization rights to CAL02, an antitoxin agent for the treatment of severe pneumonia in combination with traditional antibacterial drugs; and AOP Orphan Pharmaceuticals GmbH for the commercial rights of Landiolol, a novel therapeutic product candidate for the short-term reduction of ventricular rate in patients with supraventricular tachycardia, including atrial fibrillation and atrial flutter. It has a strategic collaboration with Tyme Technologies, Inc. for the development of SM-88 to treat breast cancer (HR+/HER2-) and high-risk metastatic sarcomas. Eagle Pharmaceuticals, Inc. was incorporated in 2007 and is headquartered in Woodcliff Lake, New Jersey.

Spruce Biosciences, Inc., a biopharmaceutical company, focuses on developing and commercializing novel therapies for rare endocrine disorders. The company engages in developing tildacerfont, a non-steroidal therapy to enhance disease control and reduce steroid burden for patients suffering from congenital adrenal hyperplasia (CAH), which is in Phase 2b clinical trial; and to evaluate glucocorticoid reduction in adult patients with classic CAH that is Phase 2b clinical trial. It is also developing tildacerfont for the treatment of pediatric classic congenital adrenal hyperplasia in children that is in Phase 2 clinical trial; and for females with polycystic ovary syndrome, which is in Phase 2 clinical trial. Spruce Biosciences, Inc. has a license agreement with Eli Lilly and Company to research, develop, and commercialize compounds for various pharmaceutical uses; and collaboration and license agreement with Kaken Pharmaceutical Co. Ltd. to develop, manufacture, and commercialize tildacerfont for the treatment of CAH in Japan. The company was incorporated in 2014 and is headquartered in South San Francisco, California.