Regulatory bodies such as the U.S. Food and Drug Administration (FDA) and European Medicines Administration (EMA) have been offering the status of orphan drug designation (ODD) to new drugs and biologics.
Orphan Drugs Industry:
Regulatory bodies such as the U.S. Food and Drug Administration (FDA) and European Medicines Administration (EMA) have been offering the status of orphan drug designation (ODD) to new drugs and biologics. These drugs are used to treat rare diseases and conditions. These regulatory bodies have made it their priority to support the development such drugs. Biotech and drug discovery & development firms have been gaining approval for their drugs from these regulatory bodies. These drugs are focused on treatment of various conditions such as lecithin-cholesterol acyltransferase (LCAT) deficiency and soft tissue sarcoma. The ODD status offers various benefits such as financial incentives, support for clinical development, and market exclusivity for up to seven years in the U.S. The demand for orphan drugs will increase in the coming years. According to the report published by Allied Market Research, the global orphan drugs market is expected to reach $435.68 billion by 2030. Following are some of the activities taking place across the world.
New drugs are gaining approval from regulatory bodies and achieving the orphan drug status. These approvals are expected to help in treatment of rare diseases. ABIONYX Pharma gained the status of orphan-drug designation (ODD) for its Bio-HDL CER-001 by the U.S. FDA. The biotech firm has been instrumental in discovery and development of innovative drugs and therapies for patients and its new drug is used for treating lecithin-cholesterol acyltransferase (LCAT) deficiency. The firm highlighted that the designation is provided for partial LCAT deficiency that is associated with Fisheye Disease and an entire LCAT deficiency associated with renal symptoms and corneal opacities. The European Medicines Administration (EMA) already provided the ODD to this drug in July 2021.
The approval from FDA for new orphan drugs continues. C4 Therapeutics, Inc., the biopharmaceutical firm, gained the status of orphan-drug designation (ODD) for its CFT8634 by the U.S. FDA. This drug is used for treatment of soft tissue sarcoma. CFT8634 is a BiDAC™ degrader that targets BRD9. It carries out the treatment of cancers that are related to BRD9, including SMARCB1 deleted cancers and synovial sarcoma. BRD9 is considered “undruggable” target, as inhibitors of the bromodomain provide no effectiveness during the treatment. So, the potential of the targeted protein degrader CFT8634 presents huge opportunities when it comes to meet unmet requirements of patients suffering from soft tissue sarcoma and other types of cancers. Approval for such drugs will continue as regulatory bodies accelerate the approval process for drugs used in treatment of rare diseases.
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Top Leading Players:
- Abbvie Inc.
- Amgen Inc.
- Amryt Pharma Plc
- Bristol-Myers Squibb Company
- F. Hoffmann-La Roche AG
- GlaxoSmithkline PLC
- Johnson & Johnson (Janssen Global Services, LLC)
- Novartis AG (Sandoz)
- Pfizer, Inc
- Sanofi S.A.
Covid-19 scenario-
Moreover, the outbreak of the COVID-19 period has had a positive impact on the growth of the global orphan drugs market, owing to a significant rise in adoption of orphan drugs techniques in clinical trials and research activities in the healthcare industry in 2020. It is because researchers and clinicians across the world focused on analysing the effect of coronavirus on human immune system. A company named Cytek Biosciences took the initiative to provide technical assistance to researchers for their flow cytometry.