Global orphan drug market The CDMO market has grown significantly as a result of numerous causes such as increased attention on rare illnesses and favorable government incentives such as tax breaks, fee waivers, and prolonged marketing exclusivity for orphan pharmaceuticals.
Orphan drugs are meant to treat specific medical diseases, affecting a tiny population, generally less than 200,000 people in the United States, with a comparable low frequency in other countries. CDMOs that specialize in orphan drugs provide a wide variety of services, including drug formulation, process development, clinical trial materials manufacture, and commercial-scale production. They collaborate closely with pharmaceutical and biotech businesses to bring orphan medications to the worldwide market.
Developing Orphan Drugs The CDMO Market: Meeting Unmet Medical Needs in Rare Diseases
The market for Orphan Drugs Contract Development and Manufacturing Organizations (CDMOs) is expected to expand significantly as expenditures in treatments for rare illnesses increase. The worldwide orphan drug CDMOs market, which is motivated by unmet medical requirements and regulatory incentives, is driving this expansion, making it an enticing area for CDMOs. For instance,
- In February 2023, AskBio, which is a subsidiary of Bayer AG, announced today that the European Commission (EC) has granted orphan drug designation to AB-1003, also known as LION-101*. This designation is for the purpose of treating limb-girdle muscular dystrophy (LGMD). AB-1003 represents a groundbreaking investigational gene therapy based on recombinant adeno-associated virus (AAV) and is currently in development as a one-time intravenous (IV) infusion.
Over 30 million individuals in the United States are plagued by over 7,000 rare diseases, many of which are life-threatening and have no effective medical therapies. Because of their complicated biology and limited understanding of their natural history, developing medicines, biologics, and medical devices for these uncommon illnesses is difficult. Given the crucial nature of orphan pharmaceuticals and their sometimes complicated production procedures, ensuring high quality and regulatory compliance is critical. To achieve these standards, orphan medicine production is usually outsourced to CDMOs that provide quality control systems. Orphan pharmaceuticals often serve tiny patient populations, necessitating the production of smaller amounts for clinical and commercial usage. Consequently, producing orphan drugs demands flexibility in both batch size and process design, making outsourcing a favored choice.
Major Factors Driving the Rapid Growth of the Orphan Drugs CDMO Market
The rapid rise of the orphan medicines CDMO industry is highly influenced by a number of key variables, including an increasing patient population seeking rare illness therapies. To begin with, fast advances in medical diagnosis and increased awareness have resulted in a significant rise in the accuracy of rare illness diagnoses, resulting in a greater need for orphan medications. Furthermore, governments throughout the world have enacted legislation that gives pharmaceutical firms with different incentives, such as prolonged market exclusivity and tax breaks, in order to encourage them to find therapies for rare illnesses. Furthermore, as the global population grows, even uncommon diseases can impact a large number of people, adding to the rise of the orphan medicines CDMOs market. Moreover, the emergence of precision medicine and genomics has allowed for more precise rare disease diagnosis and treatment, further intensifying the need for customized orphan drugs.
Collaborations among patient advocacy organizations, pharmaceutical firms, and research institutes have resulted in greater research funding and improved medication development efforts. The globalization of healthcare services has increased patient access to rare illness therapies, hence extending the potential orphan medications CDMOs market.
For instance,
- In September 2022, Sanofi received FDA clearance for Xenpozyme, an enzyme replacement therapy for acid sphingomyelinase deficiency (ASMD) without central nervous system involvement. Xenpozyme is the FDA-approved drug sanctioned for addressing non-CNS ASMD symptoms in both children and adults in the United States.
Key Market Challenges Affecting the Growth of the Orphan Drugs CDMOs Market
Global orphan medicines CDMOs market has great growth potential, but it also confronts obstacles such as restricted availability of essential components (API), lengthy formulation development procedures, and the requirement for trustworthy small-scale production.
North America holds the largest share of the global orphan drug CDMOs market.
The Global orphan medication CDMOs market is expanding rapidly, with a prominent presence in North America, where many pharmaceutical and biotech businesses are situated. Many significant reasons contribute to the area’s supremacy, including a growing patient population, favorable regulatory policies, and the existence of top market competitors in the region.
During the projection period, the Asia-Pacific area is also expected to develop rapidly. Rising awareness of rare illnesses, an increase in the number of clinical studies, and increased acceptance of orphan pharmaceuticals are some of the primary factors that may fuel the growth of the orphan drugs CDMOs market in this area.
Competitive Landscape Analysis: Global Orphan Drugs CDMOs Market
Some of the key players operating in the global orphan drugs CDMOs market are Novartis AG, F. Hoffmann-La Roche Ltd, Celgene, Bristol-Myers Squibb Company, Sanofi, Bayer Healthcare, Doppel and LLS Health, among others.
Leading Market Players Adopt Aggressive and Efficient Growth Strategies to Establish Their Foothold in Orphan Drugs CDMOs Market
To get a larger market share, the main competitors in the global orphan medicines CDMOs market are pursuing both organic and inorganic growth methods such as partnerships and collaborations, new product launches, and acquisitions of other firms.
For instance,
- In December 2020, AGC Biologics announced its cooperation with Laboratoire Pierre Fabre for the manufacturing of ER-004, an intra-amniotic medication that is intended to change the treatment of a rare and incapacitating genetic illness. AGC Biologics’ function is to create GMP material to assist the next step of clinical studies.
The global orphan drugs CDMO market is expected to gain consistent momentum in the coming years due to an increase in the number of orphan diseases, favorable government policies such as greater duration of exclusivity in marketing for orphan drugs, and aggressive and efficient organic and inorganic growth strategies employed by the major market players.
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