BioMarin Pharmaceutical Inc. competitors & alternatives

Pharmaceuticals

Sarepta Therapeutics, Inc. operates as a biopharmaceutical firm with products already available on the market. Its primary objective is the research and advancement of genetic treatment modalities, encompassing RNA-targeted therapeutics and gene therapies, specifically for the management of rare diseases. The company's commercial offerings include EXONDYS 51, an injectable medication prescribed for Duchenne muscular dystrophy (DMD) patients whose genetic mutation in the dystrophin gene is responsive to exon 51 skipping. Similarly, VYONDYS 53 is provided for individuals with DMD who can benefit from exon 53 skipping. Sarepta is also actively developing several investigational therapies: AMONDYS 45, a product candidate that utilizes phosphorodiamidate morpholino oligomer chemistry and exon-skipping technology to bypass exon 45 of the dystrophin gene. SRP-5051, a peptide-conjugated PMO engineered to bind to exon 51 of dystrophin pre-mRNA. SRP-9001, a gene therapy initiative focused on micro-dystrophin for DMD. SRP-9003, a gene therapy program targeting limb-girdle muscular dystrophies. The company has established collaborative relationships with various partners, including F. Hoffman-La Roche Ltd, Nationwide Children's Hospital, Lysogene, Duke University, Genethon, and StrideBio. Sarepta was incorporated in 1980 and maintains its corporate headquarters in Cambridge, Massachusetts.

Pharmaceuticals

BioCryst Pharmaceuticals, Inc. is a biotechnology company focused on discovering and developing novel, orally administered, small-molecule therapeutics. The company currently markets two key products: Peramivir injection: An intravenous neuraminidase inhibitor, sold under the brand names RAPIVAB, RAPIACTA, and PERAMIFLU, used for the treatment of acute uncomplicated influenza. ORLADEYO: An oral serine protease inhibitor designed to manage hereditary angioedema. BioCryst's development pipeline includes several promising candidates: BCX9930: An oral factor D inhibitor in Phase II clinical trials for complement-mediated diseases. BCX9250: An oral activin receptor-like kinase-2 inhibitor undergoing Phase I clinical evaluation for fibrodysplasia ossificans progressiva. Galidesivir: An RNA dependent-RNA polymerase inhibitor, also in Phase I, aimed at treating a range of RNA viruses, including Marburg, Yellow Fever, Ebola, and Zika. The company engages in strategic collaborations and in-license agreements with numerous partners. These include pharmaceutical entities like Torii Pharmaceutical Co., Ltd., Seqirus UK Limited, Shionogi & Co., Ltd., Green Cross Corporation, and Mundipharma International Holdings Limited. Their alliances also extend to governmental and academic organizations such as the National Institute of Allergy and Infectious Diseases, the Biomedical Advanced Research and Development Authority, the U.S. Department of Health and Human Services, The University of Alabama at Birmingham, Albert Einstein College of Medicine of Yeshiva University, and Industrial Research, Ltd. Established in 1986, BioCryst Pharmaceuticals, Inc. is headquartered in Durham, North Carolina.

Pharmaceuticals

Mirum Pharmaceuticals, Inc. is a biopharmaceutical firm dedicated to discovering and commercializing groundbreaking therapies for serious rare and orphan conditions. Its lead experimental drug, LIVMARLI, an oral treatment, is currently undergoing trials for progressive familial intrahepatic cholestasis (PFIC), Alagille syndrome, and biliary atresia. Furthermore, the company is advancing Volixibat to address intrahepatic cholestasis of pregnancy and primary sclerosing cholangitis. Founded in 2018, Mirum Pharmaceuticals, Inc. operates from its headquarters in Foster City, California.

Ionis Pharmaceuticals, Inc.0JDI · PharmaceuticalsCarlsbad, United States

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Pharmaceuticals

Ionis Pharmaceuticals, Inc. is a U.S.-based biotechnology company specializing in the discovery and development of RNA-targeted therapeutic agents. The company's commercialized portfolio features SPINRAZA, a treatment for spinal muscular atrophy (SMA) in both pediatric and adult patients. It also offers TEGSEDI, an injectable therapy for polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults, and WAYLIVRA, approved for familial chylomicronemia syndrome and familial partial lipodystrophy. Beyond its marketed therapies, Ionis is actively progressing numerous drug candidates through pivotal Phase 3 clinical trials. These late-stage programs include Eplontersen, a monthly self-administered subcutaneous injection targeting all types of TTR amyloidosis; Olezarsen, designed for patients with severe hypertriglyceridemia (SHTG); Donidalorsen, for hereditary angioedema; ION363, aimed at amyotrophic lateral sclerosis; Pelacarsen, for individuals with established cardiovascular disease and elevated lipoprotein(a); and Tofersen, developed to inhibit superoxide dismutase 1 production. Furthermore, Ionis is exploring treatments across a broader spectrum of therapeutic areas, such as metabolic, infectious, renal, ophthalmic diseases, and cancer. The company maintains a significant strategic collaboration with Biogen Inc. and holds various collaboration and licensing agreements with other prominent pharmaceutical partners, including AstraZeneca, Bayer AG, GlaxoSmithKline plc, Novartis AG, Roche, Janssen Biotech, Inc., and Flamingo Therapeutics, Inc. Founded in 1989, Ionis Pharmaceuticals, Inc. operates from its headquarters in Carlsbad, California.

Pharmaceuticals

Catalyst Pharmaceuticals, Inc., founded in 2002 and based in Coral Gables, Florida, operates as a commercial-stage biopharmaceutical enterprise. This company is dedicated to discovering, developing, and marketing therapeutic solutions for individuals in the United States who are afflicted with uncommon, severe, and long-term neuromuscular and neurological disorders. Among its key offerings is Firdapse, an amifampridine phosphate tablet formulation approved for managing Lambert-Eaton Myasthenic Syndrome (LEMS). Catalyst also provides Ruzurgi, a specific treatment option for pediatric patients diagnosed with LEMS. Beyond its existing products, Catalyst is actively investigating additional applications for Firdapse, including its potential use in treating MuSK antibody positive myasthenia gravis, spinal muscular atrophy type 3, and hereditary neuropathy with liability to pressure palsies. The company engages in various strategic collaborations, such as licensing agreements with BioMarin Pharmaceutical Inc., and a joint development and commercialization agreement with Endo Ventures Limited concerning a generic version of Sabril tablets. Previously known as Catalyst Pharmaceutical Partners, Inc., the company adopted its current name in May 2015.

CANBRIDGE-B01228 · PharmaceuticalsChina

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Pharmaceuticals

CANbridge Pharmaceuticals Inc. is a biopharmaceutical company focused on the global research, development, and commercialization of treatments for rare disorders and various cancer types. The company's marketed products include Hunterase, an enzyme replacement therapy for Hunter syndrome; NERLYNX (Neratinlib), a powerful, irreversible tyrosine kinase inhibitor targeting HER2-positive breast cancer; and Caphosol Mouth Rinse, used to alleviate temporary and persistent dry mouth and throat. Its robust development pipeline features several investigational therapies: CAN008, an artificially engineered antibody-like fully human fusion protein being developed for glioblastoma multiforme; CAN108, an orally administered reversible inhibitor of the ileal bile acid transporter, designed to treat rare cholestatic liver diseases; CAN106, a humanized monoclonal antibody against complement C5 for complement-mediated conditions; CAN103 and CAN104, both recombinant human enzyme replacement therapies (ERTs); CAN105, a bispecific antibody; and gene therapy programs CAN201 and CAN202, both utilizing adeno-associated virus (AAV) sL65 capsid technology. Established in 2012, CANbridge Pharmaceuticals Inc. is based in Beijing, China.

Pharmaceuticals

Amarin Corporation plc is a pharmaceutical firm dedicated to the research, development, and commercialization of therapeutic solutions for cardiovascular diseases. Its operations span several international regions, including the United States, Germany, Canada, Lebanon, and the United Arab Emirates. The company's flagship product is VASCEPA, an omega-3 fatty acid formulation available by prescription only. This medication acts as an adjunctive therapy to diet, designed to decrease elevated triglyceride levels in adult patients suffering from severe hypertriglyceridemia. Amarin primarily distributes its offerings through wholesale channels and specialized pharmacy providers. Furthermore, the company has entered into a collaboration with Mochida Pharmaceutical Co., Ltd. to advance the development and market introduction of drug products and new indications derived from Vascepa's active pharmaceutical ingredient, which encompasses omega-3 acid and eicosapentaenoic acid. Established in 1989 as Ethical Holdings plc, the organization rebranded to Amarin Corporation plc in 1999. Its corporate headquarters are located in Dublin, Ireland.

ACADIA Pharmaceuticals Inc.ACAD · PharmaceuticalsSan Diego, United States

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Pharmaceuticals

Acadia Pharmaceuticals Inc. operates as a biopharmaceutical company, primarily dedicated to discovering, developing, and commercializing small molecule therapeutics. Their core focus lies in addressing critical unmet medical needs within the realm of central nervous system (CNS) disorders. The company's marketed product, NUPLAZID (pimavanserin), is prescribed for the management of hallucinations and delusions associated with Parkinson's disease psychosis. Beyond its commercialized offering, ACADIA maintains a robust development pipeline, featuring several promising candidates: Pimavanserin: This compound is currently undergoing Phase 3 clinical trials for additional indications, specifically for Alzheimer's disease psychosis and the negative symptoms of schizophrenia. Trofinetide: A novel synthetic compound, Trofinetide is also in Phase 3 development, aimed at treating Rett syndrome. ACP-044: This orally administered, first-in-class non-opioid analgesic is progressing through Phase 2 studies for both acute and chronic pain. ACP-319: Identified as a positive allosteric modulator of the muscarinic receptor, ACP-319 is in Phase 1 development, exploring its potential for schizophrenia and improving cognition in Alzheimer's patients. Established in 1993, ACADIA Pharmaceuticals Inc. is headquartered in San Diego, California.

Pharmaceuticals

Ligand Pharmaceuticals Incorporated operates as a biopharmaceutical entity, primarily engaged in sourcing and advancing technologies to empower pharmaceutical companies globally in their efforts to discover and develop novel therapeutic solutions. Its diverse portfolio of commercialized products addresses a wide range of medical conditions. For hematologic malignancies, these include Kyprolis and Evomela, both targeting multiple myeloma, as well as Rylaze, a treatment for acute lymphoblastic leukemia and lymphoblastic lymphoma in both adult and pediatric patients. In the realm of infectious diseases, Ligand offers Veklury for moderate to severe COVID-19, Vaxneuvance for preventing invasive Streptococcus pneumoniae disease, and Pneumosil, a pneumococcal conjugate vaccine designed to protect children from pneumonia. Bone health solutions comprise Teriparatide injection for osteoporosis and Duavee for postmenopausal osteoporosis. Other key offerings include Zulresso, a Captisol-enabled formulation of brexanolone for postpartum depression; Nexterone, a Captisol-enabled amiodarone; and Noxafil-IV, a Captisol-enabled posaconazole for intravenous administration. The company’s comprehensive product lineup further extends to the Aziyo portfolio, featuring pericardial repair and CanGaroo envelope extracellular matrix products; Exemptia for autoimmune disorders; Vivitra for breast cancer; Bryxta and Zybev for a variety of indications; and Minnebro for managing hypertension. Beyond its own commercialized products, Ligand Pharmaceuticals collaborates with partners on numerous programs currently undergoing clinical development, targeting conditions such as cancer, seizure disorders, diabetes, cardiovascular ailments, muscle wasting, hepatic and renal diseases, and various other serious illnesses. The company also directly supplies Captisol materials, a proprietary technology frequently integrated into many of its drug formulations. Established in 1987, Ligand Pharmaceuticals is headquartered in Emeryville, California.

Pharming Group N.V.PHAR · PharmaceuticalsLeiden, Netherlands

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Pharmaceuticals

Pharming Group N.V. is a biopharmaceutical company dedicated to the creation and commercialization of protein replacement therapies and precision medicines. The firm concentrates its efforts on addressing rare diseases and critical unmet medical needs across the United States, Europe, and other international markets. Its primary commercialized product is Ruconest, a recombinant human C1 esterase inhibitor utilized for the acute treatment of hereditary angioedema. Pharming's development pipeline also includes rhC1INH, which is being investigated for conditions such as pre-eclampsia, acute kidney injury, and COVID-19. Additionally, the company is developing leniolisib, a phosphoinositide 3-kinase delta (PI3K delta) inhibitor designed for patients with activated PI3K delta syndrome, as well as an alpha-glucosidase therapy targeting Pompe and Fabry diseases. The company has established significant collaborations, including a development and licensing agreement with Novartis. It also maintains a strategic partnership with Orchard Therapeutics plc for the research, development, manufacturing, and commercialization of OTL-105, an investigational ex-vivo autologous hematopoietic stem cell gene therapy intended for hereditary angioedema. Pharming Group N.V. is headquartered in Leiden, the Netherlands.

Biomedica Management CorpPharmaceuticalsUnited States

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Pharmaceuticals

Biomedica Management Corp is a pharmaceutical company that specializes in the commercialization, distribution, and in-licensing of rare disease and innovative therapies in emerging and underserved markets. It provides biopharmaceutical partners with comprehensive services including market access strategies, regulatory navigation, and reimbursement solutions to expand patient access to breakthrough treatments.

Pharmaceuticals

Jaguar Health, Inc., a commercial-stage pharmaceutical firm, specializes in developing prescription therapies for both human and animal patients afflicted by gastrointestinal ailments, particularly chronic and severe diarrhea. Its operations are structured into two distinct divisions: Human Health and Animal Health. Presently, the company markets Mytesi, an approved medication providing symptomatic relief for noninfectious diarrhea experienced by adults with HIV/AIDS undergoing antiretroviral therapy. Beyond its commercial product, Jaguar Health is actively advancing Crofelemer, the active pharmaceutical ingredient in Mytesi, for a broad spectrum of additional indications. These include managing diarrhea associated with cancer therapy, seeking orphan drug designation for symptomatic relief in infants and children with congenital diarrheal disorders, and for adult and pediatric patients suffering from short bowel syndrome with intestinal failure. Further potential applications encompass supportive care for diarrhea linked to inflammatory bowel diseases, diarrhea-predominant irritable bowel syndrome (IBS-D), and idiopathic or functional diarrhea. Their pipeline also features lechlemer, a next-generation anti-secretory agent being developed to combat cholera. In the animal health sector, they are developing Canalevia, an oral, plant-derived drug candidate designed to address chemotherapy-induced diarrhea and exercise-induced diarrhea in canines. Established in 2013, Jaguar Health, Inc. maintains its headquarters in San Francisco, California.