Orphan Drugs Market: By Drug class (Biologics and Small Molecules) By Disease Type (Oncological Diseases, Metabolic Diseases, Hematologic and Immunologic Diseases, Infectious Diseases, and Neurologic Diseases) By Distribution channel (Hospitals Pharmacies, Retail Pharmacies, Online stores) Geography. 

The orphan drugs market was valued at USD 174.11 million in 2023 and is poised to grow at a CAGR of 9.5% from 2024 to 2030. Orphan drugs are developed to treat rare medical conditions affecting less than 1 in 2,000 populations. Orphan drugs are used to treat patients with chronic severe diseases for which no treatment, or at least adequate one, has so far been available such as Agammaglobulinemia, Good pasture syndrome, Granulomatosis with Polyangiitis (GPA formerly Wagener Granulomatosis), Leucocyte adhesion deficiency, Pediatric Burton Agammaglobulinemia, Pediatric service combined Immunodeficiency, Schnitzler Syndrome and others. The orphan drugs market is growing significantly due to the rise in patient population, favorable reimbursement policies in developed and developing countries, and an increase in R&D for the launch of newer therapeutics by the market players. Moreover, pharmaceutical companies can price orphan drugs at premium levels due to the small patient population and the specialized nature of rare diseases, creating a proficient revenue potential.

In addition, regulatory incentives like decreased regulatory fees and tax credits further propel companies to invest in orphan drug development. Additionally, the rise in research and development efforts focused on addressing unmet medical needs, collaborations between academic institutions and market players, and fundraising from public and private organizations are anticipated to propel the orphan drugs market over the forecast years. However, the high cost of treatment, the stringent regulatory framework for product approval, and high R&D expenditure remain substantial barriers that hamper the growth of the orphan drugs market. However, the key opportunities associated with these drugs are enhanced affordability and patient access to these critical treatments, geographical expansion by the market players, and government incentives for R&D activities, which create opportunities for the players in the orphan drugs market.

Orphan Drugs Market Recent Key Developments:

• In March 2023, ADX-2191 molecule developed by Aldeyra Therapeutics, Inc received First FDA-Approved Drug for Patients Suffering from Primary Vitreoretinal Lymphoma.
• In September 2023, Ojjaara (momelotinib) received FDA approval for the myelofibrosis patients with anaemia condition.