US Orphan Drugs Market, Drugs Sales, Price, Dosage & Clinical Trials Insight 2028 Report Highlights:
- US Orphan Drugs Market Opportunity: > USD 150 Billion By 2028
- Orphan Drugs Dosage, Price & Treatment Cost: > 350 Orphan Drug
- Annual & Quarterly Sales Insight (2019 – Q1’2023):> 100 Orphan Drugs
- US Orphan Drugs Reimbursement Scenario: Medicare, Medicaid, Private Insurers
- Active Clinical Trials Insight By Company, Indication & Phase: 1000 Orphan Drugs
- Marketed Orphan Drugs Clinical Insight By Company & Indication: > 400 Orphan Drugs
- Competitive Landscape: 75 Companies
In recent years, drug developers have focused their research and development resources in the market of rare or orphan diseases. Affecting a limited number of patients, orphan drugs are developed specifically for rare diseases. To widen the competitive landscape for the orphan drug market which previously was did not witness much growth, the US orphan drug designations has emerged as a powerful catalysts resulting in new technological advancements in the drug research and development field.
Prior to implementation of orphan drug programs, these diseases were believed to be commercially unviable by pharmaceutical companies due to their small patient base. However, the US Orphan Drug Act revolutionized this landscape, by offering incentives to companies willing to invest in the development of treatments for orphan conditions. This has resulted in several pharmaceutical companies developing drugs that could potentially in future be orphan drug designation.
There has been a spur in the development of effective treatments for conditions which were before left with no treatment options. For instance, Recurrent Respiratory Papillomatosis (RRP) is a rare disease caused by HPV-6 and HPV-11. While surgical treatment is the standard of care approach for RRR, it often grows back due to the remnants of HPV infection. In May 2023, Inovio was granted an orphan drug designation for its investigational DNA medicine candidate, INO-3107 which is designed to generate a targeted T cell response against HPV-6 and HPV-11 for the treatment of recurrent respiratory papillomatosis.
As a driving force, the US orphan drug designation is behind the market’s growth. Pharmaceutical companies developing orphan drugs are granted exclusive marketing rights for a period of 7 years, during which no other drug with the same active ingredient and indication can gain market approval for the same rare disease. This market exclusivity provides a unique opportunity for pharmaceutical companies to recover their investments, incentivizing drug development and driving a surge in innovative treatments for orphan diseases.
Furthermore, collaborative arrangements have always been a strong part of the unending growth of US’s pharmaceutical industry. With orphan drug sponsors getting seven years of market exclusivity with FDA approvals of their orphan drugs, other companies are left with no options but to be put on hold for their respective therapeutic candidate. Therefore, companies opt for collaborative approach their benefits both the parties.
Recently, Caeregen Therapeutics and Wacker Biotech joined together for a collaborative agreement to produce a novel regenerative medicine, CTR-107 for the treatment of Familial Exudative Vitreoretinopathy (FEVR), a genetic disorder of retinal blood vessel formation, resulting in partial or complete vision loss. As the Contract Development Manufacturing Organization (CDMO), for Caeregen Therapeutics, Wacker Biotch produces drug substance for the company. Being a potential breakthrough program for a rare disease, CTR-107 has also received Rare Pediatric Disease designation from the US FDA for the same indication.
In recent years, awareness for rare diseases has been growing which has resulted in advancements in science and technology, resulting in growing understanding of the underlying mechanisms of orphan diseases. Also, the incentives offered by the US orphan drug status have also been fueling the growth of orphan drug market. Companies benefit from determing their own price decisions, in addition to becoming a sole market owner for 7 years. These factors influence the competition and pricing making the US orphan drug market a sustainable area even with limited patient population.
Synlogic was granted the orphan designation for its drug SYNB1934 being developed for the treatment of phenylketonuria (PKU). As the company initiates the phase III clinical trial, with US orphan drug status, Synlogic will benefit from receiving financial incentives as well as regulatory support from FDA to design appropriate trial design for SYNB1934, accelerating the clinical study as well as the market entry for the drug.
Overall, the commercial potential of the US FDA orphan drug status in the US and international markets are immense. With growing investments, innovative therapies, improving patient access and companies addressing the unmet medical needs, the orphan drug market of US is propelling the growth in rare disease research. Moreover, FDA orphan drug designations serve as a gold standard for international regulatory bodies, expediting their approval in those regions. As the pharmaceutical industry continues to prioritize rare disease research, the commercial potential of FDA orphan drug status will remain a driving force in shaping the future of innovative treatments.