Orphazyme Funding, Investors & Competitors

Eli Lilly is a drug firm with a focus on neuroscience, cardiometabolic, cancer, and immunology. Lilly's key products include Verzenio and Jaypirca for cancer; Mounjaro, Zepbound, Foundayo, Jardiance, Trulicity, Humalog, and Humulin for cardiometabolic; and Taltz and Olumiant for immunology.

Johnson & Johnson is the world's largest and most diverse healthcare firm. It has two divisions: innovative medicine and medtech. These now represent all of the company's sales following the divestment of the consumer business, Kenvue, in 2023. After restructurings in 2023-24, the drug division focuses on three main therapeutic areas: immunology, oncology, and neurology. Geographically, just over half of total revenue is generated in the United States.

AbbVie is a pharmaceutical firm with a strong exposure to immunology (with Humira, Skyrizi, and Rinvoq) and oncology (with Imbruvica and Venclexta). The company was spun off from Abbott in early 2013. The 2020 acquisition of Allergan added several new products and drugs in aesthetics, including Botox. The 2024 acquisitions of Cerevel (neuroscience) and ImmunoGen (oncology) help supplement AbbVie's portfolio.

Roche is a Swiss biopharmaceutical and diagnostic company. The firm's bestselling pharmaceutical products include a variety of oncology therapies from acquired partner Genentech, and its diagnostics group was bolstered by the acquisition of Ventana in 2008. Oncology products account for 50% of pharmaceutical sales, and centralized and point-of-care diagnostics for more than half of diagnostic-related sales.

Merck makes pharmaceutical products to treat several conditions in a number of therapeutic areas, including cardiometabolic disease, cancer, and infections. Within cancer, the firm's immuno-oncology platform, led by Keytruda, is a major contributor to overall sales. The company also has a substantial vaccine business aimed at preventing pediatric diseases, as well as Gardasil for human papillomavirus. Additionally, Merck sells animal health-related drugs. From a geographical perspective, 47% of the company's sales are generated from US human health (pharmaceuticals and vaccines).

Novartis develops and manufactures innovative drugs. Key areas of drug development are oncology, immunology, neuroscience, and cardiovascular, renal, and metabolic. It also has an established medicines business, which includes off-patent franchises. The company sells its products globally, with the United States constituting close to one-third of total revenue.

AstraZeneca is a British-Swedish multinational pharmaceutical and biotechnology company headquartered on the Cambridge Biomedical Campus in Cambridge, England. It discovers, develops and sells prescription medicines and vaccines in oncology, cardiovascular, renal and metabolic disease, respiratory and immunology, and rare diseases, with medicines such as Tagrisso, Farxiga, Imfinzi, Calquence and Enhertu. The company earns revenue from branded drug sales to health systems worldwide, and in 2025 reported revenue of $58.7 billion, operating income of $13.7 billion and about 89,900 employees. It was formed on 6 April 1999 through the merger of Sweden's Astra AB and the UK's Zeneca Group, itself demerged from Imperial Chemical Industries in 1993. AstraZeneca is listed in London, Stockholm and New York and is led by chief executive Pascal Soriot. It has expanded through acquisitions including MedImmune, a majority stake in Acerta Pharma in 2016 and Alexion Pharmaceuticals in 2021.

Amgen is a leader in biotechnology-based human therapeutics. Flagship drugs include red blood cell boosters Epogen and Aranesp, immune system boosters Neupogen and Neulasta, and Enbrel and Otezla for inflammatory diseases. Amgen introduced its first cancer therapeutic, Vectibix, in 2006 and markets bone-strengthening drugs Prolia/Xgeva (approved 2010) and Evenity (2019). The acquisition of Onyx Pharmaceuticals bolstered the firm's therapeutic oncology portfolio with Kyprolis. Recent launches include Repatha (cholesterol-lowering), Aimovig (migraine), Lumakras (lung cancer), and Tezspire (asthma). The 2023 Horizon acquisition brought several rare-disease drugs, including thyroid eye disease drug Tepezza. Amgen also has a growing biosimilar portfolio.

Gilead Sciences develops and markets therapies to treat and prevent life-threatening infectious diseases, with the core of its portfolio focused on HIV and hepatitis B and C. Gilead's acquisition of Pharmasset brought rights to hepatitis C drug Sovaldi, which is also part of newer combination regimens that remain standards of care. Gilead is also growing its presence in the oncology market via acquisitions, led by CAR-T cell therapy Yescarta/Tecartus (from Kite) and breast and bladder cancer therapy Trodelvy (from Immunomedics).

With roughly one-third of the global branded diabetes treatment market, Novo Nordisk is the world's leading provider of diabetes care products. Based in Denmark, the company manufactures and markets a variety of human and modern insulins for patients with diabetes, as well as injectable and oral GLP-1 therapies, across its obesity and diabetes care segment. Novo also has a rare disease segment (contributing less than 10% of revenue) that specializes in protein therapies for hemophilia and other disorders.

Pfizer is one of the world's largest pharmaceutical firms, with annual sales of roughly $60 billion. While it historically sold many types of healthcare products and chemicals, now prescription drugs and vaccines account for the majority of sales. Top sellers include pneumococcal vaccine Prevnar 13 and cardiology drugs Vyndaqel and Eliquis. Pfizer sells these products globally, with international sales representing 40% of total sales. Within international sales, emerging markets are a major contributor.

Vertex Pharmaceuticals is a global biotechnology company that discovers and develops small-molecule drugs for the treatment of serious diseases. Its key drugs are Kalydeco, Orkambi, Symdeko, and Trikafta/Kaftrio, and Alyftrek for cystic fibrosis, where Vertex therapies remain the standard of care globally. Vertex has diversified its portfolio through Casgevy, a gene-editing therapy for beta thalassemia and sickle-cell disease, and Journavx, a non-opioid pain medication approved for the treatment of moderate-to-severe acute pain in adults. Additionally, Vertex is evaluating small-molecule inhibitors of APOL1-mediated kidney diseases. Vertex is also investigating cell therapies to deliver a potential functional cure for type 1 diabetes.

Bristol Myers Squibb discovers, develops, and markets drugs for various therapeutic areas, such as cardiovascular, cancer, and immune disorders. A key focus for Bristol is immuno-oncology, where the firm is a leader in drug development. Bristol derives close to 70% of total sales from the US, showing a higher dependence on the US market than most of its peer group.

Sanofi develops and markets drugs with a concentration in immunology, vaccines, and rare diseases. Although the company offers a diverse array of drugs, its highest-revenue product, Dupixent, accounts for over 30% of total sales. Profits for this immunology blockbuster drug are shared with Regeneron. About 45% of total revenue comes from the United States, 20% from Europe, and 6% from China.

Biosplice Therapeutics is a clinical-stage biotechnology firm headquartered in San Diego, California. The company develops small-molecule modulators of splicing factors to treat degenerative diseases, fibrosis, and cancers, with lead candidates in phase II trials for osteoarthritis and dry age-related macular degeneration.

Intas Pharmaceuticals is an Indian multinational pharmaceutical company headquartered in Ahmedabad, Gujarat, that develops, manufactures and markets generic and branded formulations, active ingredients and biosimilars. It was founded by Hasmukh Chudgar in 1977 and incorporated in 1985, and it is controlled by the Chudgar family, with Temasek and ChrysCapital as minority investors and the Abu Dhabi Investment Authority joining in 2022. It operates 22 manufacturing plants, 17 of them in India, and employs more than 25,000 people. Its Accord Healthcare subsidiary, which bought the Actavis UK and Ireland generics business in FY2017, gives it a large European business: in FY2022 Europe contributed about 39% of consolidated turnover, India 33% and the United States about 16%. ICRA reported consolidated operating income of Rs 18,416 crore for FY2022 and Rs 20,086 crore for FY2023, and ranked Intas seventh in the Indian pharmaceutical market in FY2022. Revenue comes from product sales, with specialty medicines such as oncology and biosimilars an increasing focus.

Biocon Biologics is an Indian biosimilars company headquartered in Bengaluru and a subsidiary of the listed group Biocon Limited. It develops, manufactures and sells biosimilar medicines in diabetes, oncology and immunology, including insulin, trastuzumab and pegfilgrastim products, in the United States, Europe, Japan, Canada, Australia and emerging markets. The business grew out of Biocon's insulin work that began in the early 2000s and was set up as a separate unit before outside investors including True North, Tata Capital Growth Fund, Goldman Sachs, ADQ and Serum Institute Life Sciences bought minority stakes between 2020 and 2023. In 2022 it acquired the global biosimilars business of its former partner Viatris, which gave it its own commercial network in developed markets. Revenue crossed USD 1 billion in the year to March 2024. In December 2025 Biocon Limited announced a plan to absorb the company and buy out the remaining minority holders through a share swap.

LEO Pharma is a Ballerup-headquartered pharmaceutical company focused on medical dermatology. It develops therapies for psoriasis, atopic dermatitis, actinic keratosis, and other skin ailments. Key products include calcipotriol/betamethasone dipropionate combinations, IL-17 inhibitors, and topical treatments. Research occurs at facilities in Denmark, the United States, the United Kingdom, and Ireland. The firm distributes in over 100 countries through direct subsidiaries and licensing agreements. Founded in 1908.