According to the official website of the FDA, a total of 481 drugs obtained FDA orphan drug certification in 2024. Previously, the highest value of this number was 477.

In fact, compared to over a decade ago, the number of drugs that have received FDA ODD has doubled in recent years. According to statistics, the total number of FDA ODDs since 1983 has been 7306. Among them, the number of drugs that have obtained FDA ODD has exceeded 100 since 2004 and has been increasing year by year since then. By 2017, the number of drugs that received FDA ODD had skyrocketed to 477. Between 2019 and 2023, the overall number of FDA ODDs obtained remained between 335 and 475. The number of drugs that have obtained FDA ODD in 2024 has broken through from the previous 2017 data, reaching 481. According to the latest data, although it has only been less than four months since 2025, there are already 96 drugs that have obtained FDA ODD.

This significant growth trend is closely related to the FDA's attention and investment in orphan drugs in recent years. In 1983, the Orphan Drug Act was officially enacted. The bill defines orphan drugs as a type of medication used to treat diseases that affect less than 200000 people in the United States, and stipulates that for drugs that have successfully obtained ODD, the benefits that drug project parties can enjoy include but are not limited to: 1) 7-year marketing exclusivity after the drug is approved for marketing; 2) Exemption from NDA/BLA application fees; 3) Clinical research expenses are eligible for a 25% tax reduction; 4) Possible exemption or reduction of application requirements for some clinical data.

This bill has to some extent alleviated the problem of the inability to achieve a balance between revenue and expenditure in the market for the research and production of orphan drugs. Meanwhile, due to the aforementioned preferential policies, ODD has also become an important milestone in the development of rare disease drugs.

At present, in addition to meeting the definition of orphan drugs, the following situations can also apply for orphan drug recognition:
 1) If a investigational drug (including biologics) can demonstrate its potential in treating, preventing, or diagnosing a rare disease in an orphan drug designation application, OOPD will grant the drug an ODD after reviewing the application materials. In general, clinical research data, animal model (/n-Vivo) data, and in vitro study (/n-Vivo) results may all serve as scientific evidence to demonstrate the potential effectiveness of new research.
 2) The innovation of drugs does not have a decisive effect on the granting of ODD. For a non innovative drug, if its active substances or main molecular structural characteristics are the same as those of previously approved drugs, and the proposed treatment or indications are also the same, but if the new drug shows clinical advantages over marketed drugs, it can also obtain orphan drug designation.
 3) For non rare diseases with a population of more than 200000 patients, if the investigational drug is suitable for the observed clinical subtype (Orphan Subset, with less than 200000 patients) of the non rare disease, and the properties of the drug (such as mechanism of action, toxicological characteristics, or existing clinical data) can prove that the drug is only suitable for this subtype and not for populations outside of it, then the drug can also be recognized as an orphan drug.

As for the 2024 ODD list, a significant trend is that pharmaceutical companies from countries such as China, South Korea, and France, in addition to the United States, are rapidly emerging in the field of orphan drug research and development capabilities. According to FDA information in 2024, among all drugs that successfully obtained FDA ODD, 300 were from American pharmaceutical companies, 68 were from Chinese pharmaceutical companies, 22 were from South Korea, and 11 were from France. Compared to the previous situation where only the United States was present and no other countries were seen, there has been a significant improvement. Especially in China, the number of FDA ODD drugs obtained in 2024 is second only to the United States.

Among them, an analysis of the list of drugs that domestic pharmaceutical companies successfully obtained FDA ODD in 2024 found that the indications for these drugs are mainly concentrated in CNS diseases, tumors, and hematological diseases. As for drug research and development companies (detailed list shown in the figure below), these companies are mainly distributed in Shanghai, Beijing, Suzhou, Wuhan, Hangzhou, and other cities.

Another point worth noting is that in the 2024 ODD list, cutting-edge technologies such as gene therapy, cell therapy, and antibody drugs dominate. For example, CRISPR gene editing technology has shown great potential in the treatment of multiple rare diseases. Specifically, the CRISPR gene editing therapy developed by Mass General Brigham for the treatment of multiple system smooth muscle dysfunction syndrome (MSMDS) caused by ACTA2-R179H mutations has become a major highlight of this year. This therapy provides patients with the possibility of a cure by precisely editing genes and repairing mutations.

Source: https://pharm.jgvogel.cn/c1498564.shtml