Brazil Rare Diseases Market: By Drug Type (Biologics, Biosimilar, Small Molecule), By Therapeutic Area (Cancer, Blood-Related Disorders, Central Nervous System, Respiratory Disorders, Musculoskeletal Disorders, Cardiovascular Disorders & Others), By Age Group (Adult, Paediatric), By Route of Administration (Oral, Injectables & Others) and Country

Brazil rare diseases market size was valued at US$ 4,189.3 million in 2023 and is projected to grow at 11.8% CAGR from 2024-2030. WHO defines rare disease as often debilitating lifelong disease or disorder with a prevalence of 1 or less, per 1000 population. Rare diseases can be caused by a number of factors, including infections, allergies, genetics, and some cancers they can be debilitating and lifelong, and can have a high biopsychosocial and economic burden on patients, families, and health systems. They can be difficult to diagnose and treat, and can take years to be properly identified.

For instance, In Brazil according to WHO a rare disease is defined as one that affects fewer than 65 people per 100,000. The key market driving factors of market are increase in the prevalence of rare disease among public & health professionals & policy makers, favourable government policies boosting the market, growing number of new drug launches. For instance, FDA has approved 15 new drugs for rare diseases in 2024, including some that treat rare cancers. For instance, Dupixent (dupilimab), which was approved to treat eosinophilic esophagitis, increasing R&D activities for novel therapeutics drugs. Moreover, growth in research and development (R&D) for novel drug therapies for rare diseases some are gene therapies, stem cell therapies, and small nucleic acid therapies also drives the growth of market in the near future.

However, some restraints are lack of awareness regarding rare disease treatment, high cost of the drugs and therapy, limited research and medical expertise on rare diseases, difficulty for patients to access therapies and services. The key opportunities in Brazil rare disease market are the adoption of orphan–drug designation programs, demand for clinical trial design, adoption of AI in the field of drug repurposing . The emerging trends of Brazil rare disease market are demand for adoption of gene- targeted approaches and advancement in precision medicine.

Key Developments

• In March 2024, AstraZeneca declared that it has finalised a deal to buy Amolyt Pharma, a biotechnology business in the clinical stages that specialises in creating cutting-edge therapies for uncommon endocrine disorders.
• In January 2024, A definitive agreement has been reached between Sanofi and Inhibrx, Inc., a publicly traded clinical-stage biopharmaceutical company that focusses on creating a wide pipeline of innovative biologic treatment candidates.